Małopłytkowość
Leczenie
Małopłytkowość definiowana jest jako liczba płytek krwi poniżej 150 × 10³/µl, a jej leczenie zależy od etiologii, stopnia nasilenia oraz obecności objawów krwawienia. W łagodnych przypadkach (płytki >50 × 10³/µl) bez krwawień zaleca się jedynie monitorowanie, natomiast w ciężkich (płytki <20-30 × 10³/µl) lub przy aktywnym krwawieniu konieczne jest leczenie. Pierwszym krokiem jest eliminacja przyczyny podstawowej, np. odstawienie leków wywołujących małopłytkowość lub leczenie infekcji. W pierwotnej małopłytkowości immunologicznej (ITP) leczenie pierwszego rzutu obejmuje kortykosteroidy (prednizon 0,5-2,0 mg/kg/dobę lub deksametazon 40 mg/d przez 4 dni) oraz dożylne immunoglobuliny (IVIG), które szybko podnoszą liczbę płytek, choć efekt jest przejściowy. Alternatywnie stosuje się immunoglobulinę anty-D u pacjentów Rh-dodatnich. W przypadku braku odpowiedzi lub nawrotów stosuje się agoniści receptora trombopoetyny (romiplostim, eltrombopag, awatrombopag), rituximab, splenektomię lub fostamatinib.
- Leczenie małopłytkowości – wprowadzenie
- Leczenie przyczynowe małopłytkowości
- Leczenie małopłytkowości immunologicznej (ITP)
- Leczenie w przypadkach nagłych i ciężkiej małopłytkowości
- Leczenie specyficznych typów małopłytkowości
- Małopłytkowość indukowana heparyną (HIT)
- Małopłytkowość u pacjentów z przewlekłą chorobą wątroby
- Małopłytkowość wywołana chemioterapią (CIT)
- Leczenie małopłytkowości u dzieci
- Strategia obserwacji („Watchful waiting”)
- Nowe kierunki w leczeniu małopłytkowości
- Zasady postępowania w małopłytkowości
Leczenie małopłytkowości – wprowadzenie
Małopłytkowość (trombocytopenia) to stan, w którym liczba płytek krwi spada poniżej 150 × 10³/µl. Leczenie małopłytkowości zależy od jej przyczyny, nasilenia objawów, liczby płytek krwi oraz ogólnego stanu pacjenta. Celem terapii jest zwiększenie liczby płytek krwi do bezpiecznego poziomu, który minimalizuje ryzyko krwawienia, a jednocześnie pozwala pacjentom prowadzić normalne życie w oczekiwaniu na spontaniczną lub wywołaną leczeniem remisję12.
W przypadku łagodnej małopłytkowości (liczba płytek >50 × 10³/µl) bez objawów krwawienia często wystarczające jest jedynie monitorowanie stanu pacjenta. Natomiast w przypadkach ciężkiej małopłytkowości (liczba płytek <20-30 × 10³/µl) lub aktywnego krwawienia, konieczne jest wdrożenie odpowiedniego leczenia34.
Leczenie przyczynowe małopłytkowości
Pierwszym krokiem w leczeniu małopłytkowości jest identyfikacja i wyeliminowanie przyczyny podstawowej5. W wielu przypadkach może to prowadzić do normalizacji liczby płytek krwi6:
- Jeśli małopłytkowość jest spowodowana lekami (np. heparyna), odstawienie leku zwykle prowadzi do poprawy liczby płytek78
- W przypadku małopłytkowości wywołanej przez infekcje (np. Helicobacter pylori, wirusowe zapalenie wątroby), leczenie zakażenia może poprawić liczbę płytek9
- Przy wtórnej małopłytkowości immunologicznej związanej z chorobami autoimmunologicznymi (np. toczeń rumieniowaty układowy), leczenie choroby podstawowej jest zalecane10
Leczenie małopłytkowości immunologicznej (ITP)
Leczenie pierwszego rzutu
W przypadku pierwotnej małopłytkowości immunologicznej (ITP), główne opcje leczenia pierwszego rzutu obejmują1112:
- Kortykosteroidy – pozostają podstawowym lekiem pierwszego wyboru dla większości pacjentów z nowo zdiagnozowanym ITP:
- Prednizon (0,5-2,0 mg/kg/dobę) lub deksametazon (40 mg dziennie przez 4 dni)13
- Działają poprzez hamowanie produkcji przeciwciał i zmniejszenie niszczenia płytek14
- Aktualne wytyczne ASH zalecają krótsze kursy (do 6 tygodni) zamiast długotrwałego stosowania15
- Efekty uboczne mogą obejmować: drażliwość, problemy żołądkowe, przyrost masy ciała, nadciśnienie, trądzik, zaburzenia snu16
- Dożylne immunoglobuliny (IVIG):
- Szybko podnoszą liczbę płytek (w ciągu 24-48 godzin)17
- Skuteczne u ponad 80% pacjentów, ale efekt jest przejściowy18
- Stosowane często w nagłych przypadkach lub gdy konieczny jest szybki wzrost liczby płytek19
- Działania niepożądane mogą obejmować: gorączkę, dreszcze, bóle głowy, bóle mięśni i stawów, pokrzywkę, wysypkę20
- Immunoglobulina anty-D (WinRho):
Leczenie drugiego rzutu
Dla pacjentów, którzy nie odpowiadają na leczenie pierwszego rzutu lub mają nawroty, dostępne są następujące opcje terapeutyczne24:
- Agoniści receptora trombopoetyny (TPO-RA):
- Rituximab:
- Chimeryczne przeciwciało monoklonalne anty-CD20 działające na limfocyty B31
- Podawany jako infuzja dożylna, zwykle co tydzień przez 4 tygodnie32
- Może osłabiać skuteczność szczepionek33
- Działania niepożądane mogą obejmować: reakcje na infuzję, bóle stawów, wysypkę, gorączkę, zwiększone ryzyko infekcji34
- Splenektomia (usunięcie śledziony):
- Fostamatinib (Tavalisse):
Inne leki immunosupresyjne
W przypadku opornej ITP można rozważyć następujące leki immunosupresyjne41:
- Mykofenolan mofetylu – użyteczny w ITP, niedawno lepiej zbadany42
- Azatiopryna – lek cytostatyczny hamujący produkcję przeciwciał43
- Cyklosporyna A – lek immunosupresyjny44
- Cyklofosfamid – lek cytostatyczny45
- Danazol – syntetyczny androgen46
- Dapson – lek przeciwbakteryjny i przeciwzapalny47
Leczenie w przypadkach nagłych i ciężkiej małopłytkowości
W przypadku zagrażającego życiu krwawienia lub bardzo niskiej liczby płytek, stosuje się4849:
- Terapię kombinowaną:
- Jednoczesne podawanie kortykosteroidów w wysokich dawkach (np. metyloprednizolon 1g dożylnie dziennie przez 3 dni)50
- IVIG (1 g/kg dziennie przez 1-2 dni)51
- Transfuzje płytek w przypadku aktywnego krwawienia52
- Transfuzje płytek krwi:
- Leki przeciwfibrynolityczne:
- Wymiana osocza (plazmafereza):
Leczenie specyficznych typów małopłytkowości
Małopłytkowość indukowana heparyną (HIT)
Małopłytkowość indukowana heparyną wymaga natychmiastowego leczenia60:
- Natychmiastowe odstawienie wszystkich produktów heparynowych61
- Wdrożenie alternatywnego antykoagulantu (nie heparyny)62:
- Bezpośrednie inhibitory trombiny: argatroban, biwalirudyna63
- Pośrednie inhibitory czynnika Xa: fondaparinux64
- Doustne bezpośrednie antykoagulanty (DOAC): riwaroksaban, apiksaban65
- Unikanie transfuzji płytek, które mogą nasilać efekt trombogenny66
- IVIG w wysokich dawkach jako terapia wspomagająca67
Małopłytkowość u pacjentów z przewlekłą chorobą wątroby
U pacjentów z małopłytkowością spowodowaną przewlekłą chorobą wątroby, którzy mają zaplanowany zabieg, dostępne są następujące opcje68:
- Awatrombopag (Doptelet) – zatwierdzony przez FDA w 2018 r.69
- Lusutrombopag (Mulpleta) – zatwierdzony przez FDA w 2018 r.70
- Oba leki są agonistami receptora trombopoetyny i mogą być podawane jako krótki kurs przed zabiegiem71
Małopłytkowość wywołana chemioterapią (CIT)
W przypadku małopłytkowości wywołanej chemioterapią, stosuje się72:
- Redukcję intensywności dawki chemioterapii lub wydłużenie odstępów między cyklami73
- Transfuzje płytek krwi przy liczbie płytek <20 × 10³/µl lub krwawieniu74
- Czynniki wzrostu stymulujące produkcję płytek (np. interleukina-11)75
- Rekombinowaną ludzką trombopoetynę (rhTPO) lub agonistów receptora trombopoetyny76
| Grupa leków | Przykłady | Mechanizm działania | Główne zastosowanie | Typowe działania niepożądane |
|---|---|---|---|---|
| Kortykosteroidy | Prednizon, deksametazon, metyloprednizolon | Hamowanie produkcji przeciwciał i zmniejszenie niszczenia płytek | Leczenie pierwszego rzutu w ITP | Drażliwość, problemy żołądkowe, przyrost masy ciała, nadciśnienie, trądzik |
| Immunoglobuliny | IVIG, Anty-D | Blokowanie niszczenia płytek przez przeciwciała | Szybkie podniesienie liczby płytek w nagłych przypadkach | Gorączka, dreszcze, bóle głowy, reakcje alergiczne |
| Agoniści receptora trombopoetyny (TPO-RA) | Romiplostim, eltrombopag, awatrombopag, lusutrombopag | Stymulacja produkcji płytek w szpiku kostnym | Leczenie drugiego rzutu w przewlekłej ITP | Bóle głowy, ryzyko zakrzepicy, podwyższona liczba płytek |
| Inhibitory kinazy | Fostamatinib | Hamowanie niszczenia płytek przez makrofagi | Oporny ITP, gdy inne leczenie zawodzi | Biegunka, nadciśnienie, nudności |
| Przeciwciała monoklonalne | Rituximab | Redukcja limfocytów B produkujących przeciwciała | Leczenie drugiego rzutu w ITP | Reakcje na infuzję, zwiększone ryzyko infekcji |
| Inne leki immunosupresyjne | Azatiopryna, mykofenolan mofetylu, cyklosporyna | Hamowanie reakcji immunologicznych | Leczenie trzeciego rzutu w opornej ITP | Supresja szpiku, zwiększone ryzyko infekcji i nowotworów |
Leczenie małopłytkowości u dzieci
Leczenie ITP u dzieci różni się od leczenia dorosłych. Większość przypadków ITP u dzieci ustępuje samoistnie w ciągu kilku miesięcy, bez konieczności leczenia77:
- Obserwacja jest preferowaną strategią dla dzieci bez objawów lub z minimalnymi objawami krwawienia78
- Leczenie jest wskazane w przypadku znaczących objawów krwawienia lub bardzo niskiej liczby płytek79
- Krótkie kursy kortykosteroidów są preferowane w stosunku do IVIG lub anty-D jako leczenie pierwszego rzutu80
- Dla dzieci opornych na leczenie pierwszej linii, agoniści receptora trombopoetyny (TPO-RA) są preferowani w stosunku do rituximabu lub splenektomii81
- Splenektomia jest rzadko wykonywana u dzieci i jest rozważana tylko w przypadkach ciężkiej małopłytkowości opornej na leczenie82
Strategia obserwacji („Watchful waiting”)
W niektórych przypadkach, szczególnie przy łagodnej małopłytkowości (liczba płytek >30 × 10³/µl) bez objawów krwawienia, można zastosować strategię uważnej obserwacji83:
- Regularne monitorowanie liczby płytek84
- Obserwacja pod kątem nowych objawów krwawienia85
- Unikanie leków wpływających na funkcję płytek (np. aspiryna, ibuprofen)86
- Ograniczenie aktywności fizycznej, która może powodować urazy87
Nowe kierunki w leczeniu małopłytkowości
Obecnie trwają badania nad nowymi metodami leczenia małopłytkowości88:
- Inhibitory kinazy Brutona (BTK)89
- Inhibitory receptora Fc noworodkowego (FcRn)90
- Przeciwciała anty-CD3891
- Inhibitory desialilacji płytek92
- Inhibitory klasycznej drogi dopełniacza93
- Ludzkie płytki krwi uzyskane z indukowanych pluripotencjalnych komórek macierzystych94
Zasady postępowania w małopłytkowości
Leczenie małopłytkowości powinno być zindywidualizowane, biorąc pod uwagę95:
- Przyczynę małopłytkowości96
- Nasilenie małopłytkowości (liczba płytek krwi)97
- Obecność objawów krwawienia98
- Choroby współistniejące99
- Preferencje pacjenta100
- Ryzyko działań niepożądanych leczenia101
Głównym celem leczenia jest utrzymanie liczby płytek na poziomie bezpiecznym, co zmniejsza ryzyko poważnego krwawienia, a jednocześnie minimalizuje działania niepożądane terapii i poprawia jakość życia pacjenta102.
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Materiały źródłowe
- #1 Immune Thrombocytopenia (ITP) Treatment & Management: Approach Considerations, Thrombopoietin Receptor Agonists, Treatment in Childrenhttps://emedicine.medscape.com/article/202158-treatment
The goal of medical care for immune thrombocytopenia (ITP) is to increase the platelet count to a safe level, permitting patients to live normal lives while awaiting spontaneous or treatment-induced remission. ITP has no cure, and relapses may occur years after seemingly successful medical or surgical management. […] The treatment paradigm is shifting, with expanding experience with earlier use of thrombopoietin receptor analogs (TPO-RA; TPO-mimetics) in chronic ITP. However, this is not yet the standard of care. […] Corticosteroids (ie, oral prednisone or high-dose dexamethasone) remain the drugs of choice for the initial management of acute ITP, especially in the setting of life-threatening thrombocytopenia or when response in the platelet count is needed in an urgent or emergent setting.
- #2 Thrombocytopenia (low platelet count) – Diagnosis and treatment – Mayo Clinichttps://www.mayoclinic.org/diseases-conditions/thrombocytopenia/diagnosis-treatment/drc-20378298
Thrombocytopenia can last for days or years. People with mild thrombocytopenia might not need treatment. For people who do need treatment for thrombocytopenia, treatment depends on its cause and how severe it is. […] If your thrombocytopenia is caused by an underlying condition or a medication, addressing that cause might cure it. For example, if you have heparin-induced thrombocytopenia, your doctor can prescribe a different blood-thinning drug. […] Other treatments might involve: […] Blood or platelet transfusions. If your platelet level becomes too low, your doctor can replace lost blood with transfusions of packed red blood cells or platelets. […] Medications. If your condition is related to an immune system problem, your doctor might prescribe drugs to boost your platelet count. The first-choice drug might be a corticosteroid. If that doesn’t work, stronger medications can be used to suppress your immune system. […] Surgery. If other treatments don’t help, your doctor might recommend surgery to remove your spleen (splenectomy). […] Plasma exchange. Thrombotic thrombocytopenic purpura can result in a medical emergency requiring plasma exchange.
- #3 Thrombocytopenia | AAFPhttps://www.aafp.org/pubs/afp/issues/2012/0315/p612.html
Thrombocytopenia is defined as a platelet count of less than 150 103 per L. […] Treatment is based on the etiology and, in some cases, treating the secondary cause results in normalization of platelet counts. […] Corticosteroids are first-line treatment for immune thrombocytopenic purpura. […] Treatment is generally restricted to those with severe thrombocytopenia and is rarely indicated when platelet counts are greater than 50 103 per L unless there is evidence of active bleeding. Corticosteroids are considered first-line treatment and increase platelet counts usually within one week of initiation. […] Secondary immune thrombocytopenic purpura is associated with other underlying conditions, such as autoimmune disorders (e.g., systemic lupus erythematosus, antiphospholipid syndrome, Graves disease, sarcoidosis), lymphoproliferative disorders, and infections (e.g., human immunodeficiency virus, Epstein-Barr virus, cytomegalovirus, varicella-zoster virus, hepatitis C virus, Helicobacter pylori).
- #4 Thrombocytopenia: Evaluation and Management | AAFPhttps://www.aafp.org/pubs/afp/issues/2022/0900/thrombocytopenia.html
Thrombocytopenia is a platelet count of less than 150 103 per L and can occur from decreased platelet production, increased destruction, splenic sequestration, or dilution or clumping. […] Transfusion of platelets is recommended when patients have active hemorrhage or when platelet counts are less than 10 103 per L, in addition to treatment (when possible) of underlying causative conditions. […] Initial treatment for immune thrombocytopenia is corticosteroid therapy or intravenous immune globulin (human) infusion. Recurrent or unresponsive cases are treated with thrombopoietin receptor agonists or immunomodulators (e.g., rituximab). […] Treatment of heparin-induced thrombocytopenia is discontinuation of heparin and immediate anticoagulation with a nonheparin anticoagulant to prevent thromboembolic complications.
- #5 Thrombocytopenia – StatPearls – NCBI Bookshelfhttps://www.ncbi.nlm.nih.gov/books/NBK542208/
Thrombocytopenic emergencies that require immediate action includes conditions of suspected HIT, TTP, HUS, drug-induced ITP, pregnancy with severe thrombocytopenia, bleeding with severe thrombocytopenia, urgently needed an invasive procedure in the presence of severe thrombocytopenia, leukopenia, and aplastic anemia. […] In patients with bleeding and severe thrombocytopenia, treatment includes platelet transfusion. […] Management includes identifying the underlying cause and treating it. […] Primary immune thrombocytopenia: This condition is a diagnosis of exclusion. First line treatment includes glucocorticoids and intravenous immune globulins; these agents inhibit autoantibody production and platelet degradation. Second line treatment includes rituximab, immunosuppressive drugs, and splenectomy. Third line agents are thrombopoietin receptor agonists, which stimulate platelet production.
- #6 Thrombocytopenia | AAFPhttps://www.aafp.org/pubs/afp/issues/2012/0315/p612.html
Thrombocytopenia is defined as a platelet count of less than 150 103 per L. […] Treatment is based on the etiology and, in some cases, treating the secondary cause results in normalization of platelet counts. […] Corticosteroids are first-line treatment for immune thrombocytopenic purpura. […] Treatment is generally restricted to those with severe thrombocytopenia and is rarely indicated when platelet counts are greater than 50 103 per L unless there is evidence of active bleeding. Corticosteroids are considered first-line treatment and increase platelet counts usually within one week of initiation. […] Secondary immune thrombocytopenic purpura is associated with other underlying conditions, such as autoimmune disorders (e.g., systemic lupus erythematosus, antiphospholipid syndrome, Graves disease, sarcoidosis), lymphoproliferative disorders, and infections (e.g., human immunodeficiency virus, Epstein-Barr virus, cytomegalovirus, varicella-zoster virus, hepatitis C virus, Helicobacter pylori).
- #7 Thrombocytopenia – StatPearls – NCBI Bookshelfhttps://www.ncbi.nlm.nih.gov/books/NBK542208/
Drug-induced thrombocytopenia: Withholding the causative drug usually results in improvement of platelet counts in cases of drug-induced thrombocytopenia. The mainstay of treatment in HIT is to withdraw all heparin products and to initiate anti-thrombin and anti-Xa activity anticoagulant agents. Dicoumarol agents added once platelet count reaches normal. […] TTP gets treated with plasma exchange. […] In patients with secondary ITP managing the underlying condition is recommended, like, in SLE, SLE treatment is with immunosuppressive agents, and in patients with H. pylori-associated thrombocytopenia, eradication of H.pylori increases platelet count.
- #8 Thrombocytopenia | AAFPhttps://www.aafp.org/pubs/afp/issues/2012/0315/p612.html
If drug-induced thrombocytopenia is suspected, the causative medication should be discontinued and the patient’s platelet count should be repeated in one week. […] Patients with unexplained severe thrombocytopenia, decreasing platelet counts, additional hematologic abnormalities, or associated bleeding complications should be referred to a hematologist.
- #9 Thrombocytopenia – StatPearls – NCBI Bookshelfhttps://www.ncbi.nlm.nih.gov/books/NBK542208/
Drug-induced thrombocytopenia: Withholding the causative drug usually results in improvement of platelet counts in cases of drug-induced thrombocytopenia. The mainstay of treatment in HIT is to withdraw all heparin products and to initiate anti-thrombin and anti-Xa activity anticoagulant agents. Dicoumarol agents added once platelet count reaches normal. […] TTP gets treated with plasma exchange. […] In patients with secondary ITP managing the underlying condition is recommended, like, in SLE, SLE treatment is with immunosuppressive agents, and in patients with H. pylori-associated thrombocytopenia, eradication of H.pylori increases platelet count.
- #10 Thrombocytopenia – StatPearls – NCBI Bookshelfhttps://www.ncbi.nlm.nih.gov/books/NBK542208/
Drug-induced thrombocytopenia: Withholding the causative drug usually results in improvement of platelet counts in cases of drug-induced thrombocytopenia. The mainstay of treatment in HIT is to withdraw all heparin products and to initiate anti-thrombin and anti-Xa activity anticoagulant agents. Dicoumarol agents added once platelet count reaches normal. […] TTP gets treated with plasma exchange. […] In patients with secondary ITP managing the underlying condition is recommended, like, in SLE, SLE treatment is with immunosuppressive agents, and in patients with H. pylori-associated thrombocytopenia, eradication of H.pylori increases platelet count.
- #11 Immune Thrombocytopenia (ITP) Treatment & Management: Approach Considerations, Thrombopoietin Receptor Agonists, Treatment in Childrenhttps://emedicine.medscape.com/article/202158-treatment
The goal of medical care for immune thrombocytopenia (ITP) is to increase the platelet count to a safe level, permitting patients to live normal lives while awaiting spontaneous or treatment-induced remission. ITP has no cure, and relapses may occur years after seemingly successful medical or surgical management. […] The treatment paradigm is shifting, with expanding experience with earlier use of thrombopoietin receptor analogs (TPO-RA; TPO-mimetics) in chronic ITP. However, this is not yet the standard of care. […] Corticosteroids (ie, oral prednisone or high-dose dexamethasone) remain the drugs of choice for the initial management of acute ITP, especially in the setting of life-threatening thrombocytopenia or when response in the platelet count is needed in an urgent or emergent setting.
- #12 How we treat primary immune thrombocytopenia in adults | Journal of Hematology & Oncology | Full Texthttps://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01401-z
The main goals of ITP treatment are to prevent bleeding and maintain the platelet count above a safe level to minimize the bleeding tendency. […] The updated ASH guidelines recommend the initiation of disease-specific treatments if the platelet counts are <30 Ã 10^9/L, while it is a recommendation based on very low certainty in the evidence of effects. [...] Corticosteroids remain the initial cornerstone therapy for ITP patients without relative contraindications. [...] IVIg is another commonly used initial therapy that can raise the platelet counts rapidly in more than 80% of newly diagnosed ITP patients; however, it is relatively expensive and the response is usually transient. [...] Anti-RhD Ig has been proposed as an alternative to IVIg in Rh-positive patients with intact spleens. [...] Neither corticosteroids, IVIg, nor anti-RhD Ig is able to modify the natural course of ITP by preventing its chronic evolution.
- #13 Immune Thrombocytopenia | Treatment & Management | Point of Carehttps://www.statpearls.com/point-of-care/23385
Second-line treatments for adults include TPO-RAs (ie, eltrombopag, romiplostim, or avatrombopag), rituximab, and splenectomy. […] Splenectomy is typically reserved for children with severe thrombocytopenia and significant hemorrhagic symptoms that require multiple pharmacological interventions. […] In 2018, the FDA approved the spleen tyrosine kinase inhibitor fostamatinib as an option for ITP refractory to second-line treatments. […] The ASH guideline panel recommends either prednisone 0.5-2.0 mg/kg per day or dexamethasone 40 mg daily for 4 days as initial corticosteroid therapy for adults newly diagnosed with ITP.
- #14 Thrombocytopenia > Fact Sheets > Yale Medicinehttps://www.yalemedicine.org/conditions/thrombocytopenia
In general, immune thrombocytopenia purpura treatments include: Corticosteroids, such as prednisone or dexamethasone, may be an effective first-line treatment […] Immunoglobulin therapy (IVIG), an intravenous therapy […] Thrombopoietin receptor agonists, which help to stimulate platelet production […] Rituximab, an immune suppressor, may help to improve platelet levels in patients who dont respond to the above therapies. […] Splenectomy, or surgical removal of the spleen, if immune modulation therapies in addition to the above therapies fail. […] In general, supportive care to stop bleeding from any cause of thrombocytopenia includes: Aminocaproic acid or transexamic acid, which promotes clotting in the mucosa (a mucus membrane that lines certain cavities in the body), such as in the nose, mouth, gastrointestinal, or genitourinary tracts, may be given to patients experiencing life-threatening bleeding
- #15 Updated Recommendations for the Treatment of Immune Thrombocytopenia â Hematology & Oncologyhttps://www.hematologyandoncology.net/archives/august-2020/updated-recommendations-for-the-treatment-of-immune-thrombocytopenia/
JCThe decision to start treatment in adults with ITP is guided by the platelet count and symptoms of bleeding. […] The current 2019 guidelines from the American Society of Hematology (ASH) recommend treatment in adults with newly diagnosed disease who are having clinically important bleeding. […] The goal of therapy is to stabilize the platelet count in a safe range, not necessarily to normalize the count. […] Corticosteroids remain the frontline therapy of choice in adults with newly diagnosed ITP, but now a shorter duration of therapyâ6 weeks or fewerâis recommended in place of the previously recommended longer courses plus taper. […] Intravenous immunoglobulin (IVIG) and anti-D therapy are still frontline options for patients in whom corticosteroids are contraindicated. […] The recent ASH guidelines also moved thrombopoietin receptor agonists into a more up-front setting according to the response rates at 1 month and the durability of the responses.
- #16 Idiopathic Thrombocytopenic Purpura (ITP) | Children’s Hospital of Philadelphiahttps://www.chop.edu/conditions-diseases/idiopathic-thrombocytopenic-purpura-itp
Specific treatment for ITP will be determined by your child’s physician based on: […] Not all children with ITP require treatment. Close monitoring of your child’s platelets and prevention of serious bleeding complications may be the course of action chosen until the body is able to correct the disorder on its own. Many children with ITP are able to spontaneously recover in about 2 to 4 days. […] When treatment is necessary, the two most common forms of treatment are: […] Steroids, which help prevent bleeding by decreasing the rate of platelet destruction. Steroids, if effective, will result in an increase in platelet counts seen within two to three weeks. Side effects may include irritability, stomach irritation, weight gain, hypertension and acne. […] Intravenous gamma globulin (IVGG), a protein that contains many antibodies and also slows the destruction of platelets. IVGG works more quickly than steroids (within 24 to 48 hours).
- #17 Idiopathic Thrombocytopenic Purpura (ITP) | Children’s Hospital of Philadelphiahttps://www.chop.edu/conditions-diseases/idiopathic-thrombocytopenic-purpura-itp
Specific treatment for ITP will be determined by your child’s physician based on: […] Not all children with ITP require treatment. Close monitoring of your child’s platelets and prevention of serious bleeding complications may be the course of action chosen until the body is able to correct the disorder on its own. Many children with ITP are able to spontaneously recover in about 2 to 4 days. […] When treatment is necessary, the two most common forms of treatment are: […] Steroids, which help prevent bleeding by decreasing the rate of platelet destruction. Steroids, if effective, will result in an increase in platelet counts seen within two to three weeks. Side effects may include irritability, stomach irritation, weight gain, hypertension and acne. […] Intravenous gamma globulin (IVGG), a protein that contains many antibodies and also slows the destruction of platelets. IVGG works more quickly than steroids (within 24 to 48 hours).
- #18 How we treat primary immune thrombocytopenia in adults | Journal of Hematology & Oncology | Full Texthttps://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01401-z
The main goals of ITP treatment are to prevent bleeding and maintain the platelet count above a safe level to minimize the bleeding tendency. […] The updated ASH guidelines recommend the initiation of disease-specific treatments if the platelet counts are <30 Ã 10^9/L, while it is a recommendation based on very low certainty in the evidence of effects. [...] Corticosteroids remain the initial cornerstone therapy for ITP patients without relative contraindications. [...] IVIg is another commonly used initial therapy that can raise the platelet counts rapidly in more than 80% of newly diagnosed ITP patients; however, it is relatively expensive and the response is usually transient. [...] Anti-RhD Ig has been proposed as an alternative to IVIg in Rh-positive patients with intact spleens. [...] Neither corticosteroids, IVIg, nor anti-RhD Ig is able to modify the natural course of ITP by preventing its chronic evolution.
- #19 Immune Thrombocytopenia (ITP) Treatment & Management: Approach Considerations, Thrombopoietin Receptor Agonists, Treatment in Childrenhttps://emedicine.medscape.com/article/202158-treatment
TPO-RA and rituximab are second-line options for patients with ITP lasting 3 months or more and in those who are corticosteroid dependent or have no response to corticosteroids. […] Intravenous immunoglobulin (IVIG) can be the drug of second choice (after corticosteroids) for some patients, such as when a rapid increase in platelet count is important. […] The treatment of chronic, refractory ITP may introduce risks of toxicity from medications that are comparable in severity to the risks of untreated thrombocytopenia. […] Splenectomy is an option for chronic ITP, as it is associated with long-term treatment-free remissions, but it is very rarely indicated in pediatric patients. […] For many years, the only treatment options after corticosteroids, IV RhIG, IVIG, and rituximab were cyclophosphamide, azathioprine, and danazol.
- #20 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Steroids temporarily calm the immune system to slow the rate at which platelets are destroyed. They can be taken by mouth. They do have side effects, including irritability, trouble sleeping, upset stomach, weight gain, acne, high blood pressure, and high blood sugar. […] Intravenous immunoglobulin (IVIG), also known as intravenous gamma globulin: IVIG contains antibodies that help slow the rate at which platelets are destroyed. It is given intravenously, through a needle inserted into a vein, over the course of three to six hours. It has temporary side effects, including fever, chills, headache, muscle and joint pain, hives, rash, and allergic reactions. […] Thrombopoietin (TPO) receptor agonists: This family of medications increases platelet production. They include eltrombopag, a daily oral medication; romiplostim, a weekly injection under the skin; and avatrombopag, a daily oral medication. These, too, can have side effects, including headaches, an elevated platelet count, and a risk of blood clots.
- #21 How we treat primary immune thrombocytopenia in adults | Journal of Hematology & Oncology | Full Texthttps://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01401-z
The main goals of ITP treatment are to prevent bleeding and maintain the platelet count above a safe level to minimize the bleeding tendency. […] The updated ASH guidelines recommend the initiation of disease-specific treatments if the platelet counts are <30 Ã 10^9/L, while it is a recommendation based on very low certainty in the evidence of effects. [...] Corticosteroids remain the initial cornerstone therapy for ITP patients without relative contraindications. [...] IVIg is another commonly used initial therapy that can raise the platelet counts rapidly in more than 80% of newly diagnosed ITP patients; however, it is relatively expensive and the response is usually transient. [...] Anti-RhD Ig has been proposed as an alternative to IVIg in Rh-positive patients with intact spleens. [...] Neither corticosteroids, IVIg, nor anti-RhD Ig is able to modify the natural course of ITP by preventing its chronic evolution.
- #22 Immune Thrombocytopenia (ITP) – Hematology and Oncology – Merck Manual Professional Editionhttps://www.merckmanuals.com/professional/hematology-and-oncology/thrombocytopenia-and-platelet-dysfunction/immune-thrombocytopenia-itp
Splenectomy can achieve a complete remission in about two thirds of patients who relapse after initial corticosteroid therapy. […] If thrombocytopenia can be controlled with second-line medical therapies, splenectomy is often not necessary. […] Second-line medical therapies include thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, or other immunosuppressive drugs. […] In children or adults with immune thrombocytopenia and life-threatening bleeding, rapid phagocytic blockade is attempted by giving IVIG 1 g/kg once a day for 1 to 2 days or, in Rh-positive patients, a single dose of IV anti-D immune globulin 75 mcg/kg. […] High-dose methylprednisolone (1 g IV once a day for 3 days) is easier to administer than IVIG or IV anti-D immune globulin but may not be as effective.
- #23 Pediatric immune thrombocytopenia (ITP) treatment – Kim – Annals of Bloodhttps://aob.amegroups.org/article/view/6316/html
The use of anti-D has decreased since it was first approved for use in ITP in 1995. […] A major contributor to the decreased utilization of anti-D was the FDA black box warning focused on severe intravascular hemolysis issued in 2010. […] Emergency care is crucial for severe uncontrolled bleeding, multiple treatment modalities may be applied together including platelet transfusion, IV steroids, IVIG and/or anti-D. […] Second line agents are indicated for children who do not respond or relapse after first line agents. […] Increasingly TPO-RAs are being utilized as the initial second line agent for pediatric ITP patients who do not respond to upfront therapies. […] Eltrombopag was approved for use in children with chronic ITP in 2015 following two multicenter, double-blind, placebo controlled trials.
- #24 Recent advances in treatments of adult immune thrombocytopeniahttps://pmc.ncbi.nlm.nih.gov/articles/PMC9057657/
Most of ITP patients respond to primary treatment such as glucocorticoids or IVIG, but a significant number of patients eventually require secondary treatment because the maintain of long-term responses are difficult. Such patients may consider medical therapy (TPO-RA, rituximab) or surgical option (splenectomy). […] TPO-RAs are small molecule that stimulates thrombopoietin to increase the production of platelets by megakaryocytes in bone marrow. […] Because B-cell plays an important role in pathogenesis of ITP, rituximab, chimeric monoclonal antibody against CD20 on the surface of B-lymphocyte has been proposed for many years to treatment of ITP. […] Splenectomy is an effective treatment as the spleen is the major site of platelet destruction as well as a site of anti-platelet antibody production.
- #25 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Steroids temporarily calm the immune system to slow the rate at which platelets are destroyed. They can be taken by mouth. They do have side effects, including irritability, trouble sleeping, upset stomach, weight gain, acne, high blood pressure, and high blood sugar. […] Intravenous immunoglobulin (IVIG), also known as intravenous gamma globulin: IVIG contains antibodies that help slow the rate at which platelets are destroyed. It is given intravenously, through a needle inserted into a vein, over the course of three to six hours. It has temporary side effects, including fever, chills, headache, muscle and joint pain, hives, rash, and allergic reactions. […] Thrombopoietin (TPO) receptor agonists: This family of medications increases platelet production. They include eltrombopag, a daily oral medication; romiplostim, a weekly injection under the skin; and avatrombopag, a daily oral medication. These, too, can have side effects, including headaches, an elevated platelet count, and a risk of blood clots.
- #26 Immune Thrombocytopenia (ITP) Treatment | Nplate® (romiplostim)https://www.nplate.com/
Nplate is a prescription medicine used to treat low blood platelet counts (thrombocytopenia) in: adults with immune thrombocytopenia (ITP) when certain medicines or surgery to remove your spleen have not worked well enough. […] Nplate is used to try to keep your platelet count about 50,000 per microliter in order to lower the risk for bleeding. […] Nplate can cause serious side effects, including: Worsening of a precancerous blood condition to a blood cancer (leukemia): Nplate is not for use in people with a precancerous condition called myelodysplastic syndromes (MDS), or for any condition other than immune thrombocytopenia (ITP). […] Nplate is not used to make your platelet count normal.
- #27 Immune Thrombocytopenia (ITP) Treatment & Management: Approach Considerations, Thrombopoietin Receptor Agonists, Treatment in Childrenhttps://emedicine.medscape.com/article/202158-treatment
In the past decade, thrombopoietin receptor agonists (TPO-RAs) have entered clinical practice as second-line agents, with robust evidence supporting their efficacy. […] Eltrombopag was studied in a phase III double-blind trial in adults with previously treated ITP lasting more than 6 months and with platelet counts lower than 30,000/L. […] Eltrombopag has a boxed warning for the risk of severe and potentially life-threatening hepatotoxicity. […] Avatrombopag, an oral TPO receptor agonist, was approved by the FDA in 2019 for adults with chronic ITP who have had an insufficient response to a previous treatment. […] For pediatric patients requiring treatment, ASH suggests a short course of corticosteroids for first-line treatment rather than IVIG or anti-D immunoglobulin. […] For children or adolescents with ITP who have significant ongoing bleeding despite treatment with IVIG, RhIG, or conventional doses of corticosteroids, ASH suggests a TPO-RA rather than rituximab or splenectomy as second-line treatment.
- #28 Immune Thrombocytopenia (ITP) Diagnosis & Treatment | DOPTELET® (avatrombopag)https://doptelet.com/what-is-itp/itp-diagnosis-and-treatment/
If immunosuppressants aren’t working for you, and your platelet counts don’t stay lifted, your doctor may consider a thrombopoietin receptor agonist (TPO-RA), such as Doptelet. […] Doptelet is a TPO-RA, not an immunosuppressant. […] TPO-RAs are a type of medication that mimics your body’s natural process of creating platelets. […] Doptelet can help boost the pathway responsible for making platelets, which adds to the platelets your body is already making. […] Doptelet is a prescription medicine used to help treat low blood platelet counts in adults with chronic immune thrombocytopenia (ITP) when other treatments have not worked well enough. […] For patients with chronic ITP, the most common side effects are headache, fatigue, bruising, nosebleeds, upper respiratory tract infection, joint pain, bleeding gums, purple or red spots on your skin (petechiae), and runny nose.
- #29 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
With the introduction of TPO-RA, rituximab and fostamatinib, therapeutic options for treating ITP have evolved from our prior standard care of corticosteroids and splenectomy. […] The TPO-RA have markedly changed the approach to treating ITP. In clinical trials they have shown response rates as high as 93%, onset of action often in 7 to 14 days, minimal side effects, a sustained duration of effect and improved HRQoL. […] Our general practice is to start a TPO-RA and then gradually taper the patient off the remaining corticosteroids over the next 4 weeks. Most patients can be subsequently maintained on the TPO-RA alone. […] For all patients on TPO-RA our goal is a platelet count of 50,000-250,000/L; as discussed further below, patients with platelet counts consistently over 200,000/L should be assessed for remission by planned dose reduction.
- #30 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Steroids temporarily calm the immune system to slow the rate at which platelets are destroyed. They can be taken by mouth. They do have side effects, including irritability, trouble sleeping, upset stomach, weight gain, acne, high blood pressure, and high blood sugar. […] Intravenous immunoglobulin (IVIG), also known as intravenous gamma globulin: IVIG contains antibodies that help slow the rate at which platelets are destroyed. It is given intravenously, through a needle inserted into a vein, over the course of three to six hours. It has temporary side effects, including fever, chills, headache, muscle and joint pain, hives, rash, and allergic reactions. […] Thrombopoietin (TPO) receptor agonists: This family of medications increases platelet production. They include eltrombopag, a daily oral medication; romiplostim, a weekly injection under the skin; and avatrombopag, a daily oral medication. These, too, can have side effects, including headaches, an elevated platelet count, and a risk of blood clots.
- #31 Treatment of Immune Thrombocytopenia: Contextualization from a Historical Perspectivehttps://www.mdpi.com/2038-8330/16/3/39
Current guidelines recommend starting treatment below 20â30 Ã 10^9/L platelets, regardless of the presence of bleeding. […] The increase in platelet counts with glucocorticoids, adrenocorticotropic hormone (ACTH), or other immunosuppressive agents has been described since 1951. […] The introduction of IVIGs by Imbach et al. in 1981 was a milestone in the treatment of symptomatic ITP in children. […] Anti-D immunoglobulin consists of IgG selectively derived from plasma of donors immunized against the D antigen. […] Thrombopoietin (TPO) is the most potent regulatory molecule of megakaryopoiesis. […] Fostamatinib is an oral inhibitor of splenic tyrosine kinase (SYK) involved in the FcR signaling pathway. […] Rituximab (RTX) is a genetically engineered chimeric mouse/human monoclonal antibody representing a glycosylated immunoglobulin with human IgG1 constant regions and murine light-chain and heavy-chain variable region sequences.
- #32 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Rituximab calms the immune attack on platelets by suppressing B cells, the white blood cells that produce antibodies. It is given as an IV infusion, typically weekly for four weeks, and works for about six to 12 months. It has temporary side effects, including immune reactions to the infusion, joint pain, rash, fever, and an increased risk of infections. […] Mycophenolate and sirolimus: These oral medications help prevent attacks on platelets by inhibiting activity of white blood cells called lymphocytes (B and T cells). Each has different side effects that your care team can discuss with you. […] Hydroxychloroquine is sometimes prescribed if ITP is accompanied by other autoimmune disorders. […] As needed, our team may offer hormone therapy if patients have heavy menstrual bleeding and anti-fibrinolytic medications (aminocaproic acid, tranexamic acid) to reduce nosebleeds, gum bleeding, or heavy menstrual bleeding.
- #33 Immune thrombocytopenia (ITP) – Diagnosis and treatment – Mayo Clinichttps://www.mayoclinic.org/diseases-conditions/idiopathic-thrombocytopenic-purpura/diagnosis-treatment/drc-20352330
Other drugs. Rituximab (Rituxan, Ruxience, Truxima) helps increase the platelet count by reducing the immune system response damaging them. But this medicine also can keep vaccinations from working well. Later surgery to remove the spleen might increase the need for the protection against illness that vaccinations give. […] Fostamatinib (Tavalisse) is a newer drug approved for people with long-lasting ITP who haven’t responded to other treatments.
- #34 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Rituximab calms the immune attack on platelets by suppressing B cells, the white blood cells that produce antibodies. It is given as an IV infusion, typically weekly for four weeks, and works for about six to 12 months. It has temporary side effects, including immune reactions to the infusion, joint pain, rash, fever, and an increased risk of infections. […] Mycophenolate and sirolimus: These oral medications help prevent attacks on platelets by inhibiting activity of white blood cells called lymphocytes (B and T cells). Each has different side effects that your care team can discuss with you. […] Hydroxychloroquine is sometimes prescribed if ITP is accompanied by other autoimmune disorders. […] As needed, our team may offer hormone therapy if patients have heavy menstrual bleeding and anti-fibrinolytic medications (aminocaproic acid, tranexamic acid) to reduce nosebleeds, gum bleeding, or heavy menstrual bleeding.
- #35 The Immune Thrombocytopenia Treatment Landscape: A Focus on Patient Perspectives and Unmet Clinical Needs – European Medical Journalhttps://www.emjreviews.com/hematology/symposium/the-immune-thrombocytopenia-treatment-landscape-a-focus-on-patient-perspectives-and-unmet-clinical-needs-s060424/
Corticosteroids are the current first-line management for ITP, but most patients will require subsequent therapy. […] Second-line treatment options include immunomodulation with the anti-CD20 monoclonal antibody (mAb) rituximab or spleen-associated tyrosine kinase (Syk) inhibitors, notably fostamatinib; splenectomy to remove the site of platelet destruction; or increasing platelet production with thrombopoietin receptor agonists (TPO-RAs). […] However, Ghanima highlighted the limited durable response and remission rates achievable with most currently available second-line therapies as a key concern when choosing treatment. […] In terms of sustained responses off-treatment, splenectomy is the strategy that supplies the highest remission rate: 69% at 1 year compared to 24% for rituximab.
- #36 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
A key aspect in caring for ITP patients is to have a mutual understanding of the terms response, remission and cure. […] My overall treatment recommendations in ITP are as follows: Many ITP patients do not need treatment; Initial treatment is corticosteroids, preferably prednisone, and if severe bleeding, IVIG; Splenectomy works but should be delayed at least 12 to 24 months. […] TPO-RA are highly effective; low rate of adverse effects; improve Health-related Quality of Life; may not need to be forever; over half come off therapy within 2 years. […] A large number of very promising new ITP treatments (Bruton kinase inhibitors, neonatal Fc receptor inhibitors, anti-CD 38 antibodies) are being developed and patients should be offered access to these trials.
- #37 Thrombocytopenia: Symptoms, Stages & Treatmenthttps://my.clevelandclinic.org/health/diseases/14430-thrombocytopenia
Splenectomy: This is surgery to remove your spleen. Your surgeon may do this if tests show your spleen is trapping large numbers of platelets. People who have splenectomies have an increased risk of developing infections. They may receive vaccinations to prevent infections. […] If your provider recommends treatment, you may need ongoing treatment to maintain a normal platelet level. Your provider will monitor your overall health and platelet levels. […] Your healthcare provider can explain what’s causing the low platelet count and discuss treatment options. They’ll also discuss lifestyle changes that may boost your platelet levels.
- #38 New Strategies for the Treatment of Immune Thrombocytopenia â Hematology & Oncologyhttps://www.hematologyandoncology.net/archives/march-2022/new-strategies-for-the-treatment-of-immune-thrombocytopenia/
Although fostamatinib is one of the agents that is recommended as a second-line treatment, I think that it has been inadequately explored as a first-line treatment. Fostamatinib is unique in ITP treatment because it is the only treatment based on a SYK inhibitor approach. Unlike other agents, which predominantly involve immunosuppression by interfering with the lymphocyte production of antibody (eg, corticosteroids), by reducing the lymphocyte burden (eg, rituximab or cytotoxic drugs), or by increasing thrombopoiesis, SYK inhibitors are believed to work by interfering with the phagocytosis of antibody-coded platelets in the process of ITP. Older studies looked at the use of fostamatinib in patients with chronic phase ITP who had failed to respond to at least 2 prior treatment strategies, but some of the newer studies are showing that an earlier introduction of fostamatinib in ITP may lead to a higher response rate.
- #39 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
In some patients with an inadequate response to TPO-RA, as mentioned above, a low dose of prednisone may synergize greatly and provide an adequate platelet count. […] Fostamatinib is a newer agent with a unique mechanism of action. It inhibits syk kinase thereby reducing macrophage destruction of platelets. […] Mycophenolate mofetil is a useful agent in ITP but has not been well studied until recently. […] Dapsone has a long history of use in chronic ITP. It is yet another steroid sparing option in responsive ITP patients. […] Danazol is another steroid sparing medication like dapsone and MMF which is helpful in some ITP patients. […] We rarely recommend splenectomy in adult patients except as a last measure or because of patient preference. […] Most of the time we are talking about response and remission. Most ITP patients do well and have a response to some therapy and many will come off therapy completely.
- #40 List of 18 Thrombocytopenia Medications Comparedhttps://www.drugs.com/condition/thrombocytopenia.html
Fostamatinib (Tavalisse) prevents antibody-mediated destruction of platelets and may be given to people with long-lasting ITP who haven’t responded to other treatments. […] Other medications, such as rituximab (Rituxan, Ruxience, Truxima), help to increase platelets by reducing the immune system response that is damaging them.
- #41 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
In some patients with an inadequate response to TPO-RA, as mentioned above, a low dose of prednisone may synergize greatly and provide an adequate platelet count. […] Fostamatinib is a newer agent with a unique mechanism of action. It inhibits syk kinase thereby reducing macrophage destruction of platelets. […] Mycophenolate mofetil is a useful agent in ITP but has not been well studied until recently. […] Dapsone has a long history of use in chronic ITP. It is yet another steroid sparing option in responsive ITP patients. […] Danazol is another steroid sparing medication like dapsone and MMF which is helpful in some ITP patients. […] We rarely recommend splenectomy in adult patients except as a last measure or because of patient preference. […] Most of the time we are talking about response and remission. Most ITP patients do well and have a response to some therapy and many will come off therapy completely.
- #42 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
In some patients with an inadequate response to TPO-RA, as mentioned above, a low dose of prednisone may synergize greatly and provide an adequate platelet count. […] Fostamatinib is a newer agent with a unique mechanism of action. It inhibits syk kinase thereby reducing macrophage destruction of platelets. […] Mycophenolate mofetil is a useful agent in ITP but has not been well studied until recently. […] Dapsone has a long history of use in chronic ITP. It is yet another steroid sparing option in responsive ITP patients. […] Danazol is another steroid sparing medication like dapsone and MMF which is helpful in some ITP patients. […] We rarely recommend splenectomy in adult patients except as a last measure or because of patient preference. […] Most of the time we are talking about response and remission. Most ITP patients do well and have a response to some therapy and many will come off therapy completely.
- #43 Conventional Treatmentshttps://pdsa.org/treatments/conventional
Many doctors consider corticosteroids (steroids) for their initial approach for newly diagnosed patients. […] Immunosuppressants, including azathioprine (Imuran), cyclosporine (Sandimmune), diphenylsulfone (Dapsone), Sirolimus (Rapamycin), and mycophenolate mofetil (Cellcept), are considered a second or third-line treatment for ITP. […] Intravenous immunoglobulin (IVIG) is a type of antibody treatment used as a front line therapy to temporarily elevate platelet counts. […] The synthetic androgen (male sex hormone) danazol (Danocrine) is sometimes used to treat ITP when other treatments have failed. […] A new approach to treating ITP is the use of a spleen tyrosine kinase (SYK) inhibitor. […] Second-line ITP treatment approach using platelet growth factors or thrombopoietin (TPO) receptor agonists.
- #44 New Strategies for the Treatment of Immune Thrombocytopenia â Hematology & Oncologyhttps://www.hematologyandoncology.net/archives/march-2022/new-strategies-for-the-treatment-of-immune-thrombocytopenia/
The initial treatment of adults with newly diagnosed ITP typically consists of the early administration of a corticosteroid (dexamethasone or prednisone), with anti-D concentrate or intravenous gamma globulin used for rapid hemostasis to prevent or reverse bleeding in the context of marked thrombocytopenia. Corticosteroids can achieve an initial platelet count response in approximately 70% of patients within the first 6 months of treatment. For the remaining 30% of patients, in whom this regimen is ineffective or inadequate, we move to agents that the US Food and Drug Administration (FDA) has approved for use in persistent or chronic ITP. Second-line agents include the monoclonal anti-CD20 antibody rituximab; the thrombopoietin (TPO) receptor agonists eltrombopag (Promacta, Novartis), avatrombopag (Doptolet, Dova), and romiplostim (Nplate, Amgen); and the spleen tyrosine kinase (SYK) inhibitor fostamatinib (Tavalisse, Rigel). Additional second-line agents for which evidence is more limited are azathioprine, cyclosporin A, cyclophosphamide, danazol, dapsone, mycophenolate mofetil, and vinca alkaloids. Another option for second-line treatment is splenectomy.
- #45 Conventional Treatmentshttps://pdsa.org/treatments/conventional
There are many treatments for ITP. All treatment options have different risks and benefits, and some can be toxic causing serious side effects. It is important to understand both the success rate and potential side effects before beginning a treatment. […] The general types of conventional treatments include: […] Anti-RhoD (WinRho) is an intravenous (IV) drug infusion used to elevate platelet counts temporarily, and can be repeated over a period of time for extended relief. […] B-cell depletion by a monoclonal antibody (anti-CD20, rituximab [Rituxan]) is a widely used treatment choice, but has not been approved by the Food and Drug Administration (FDA) to treat ITP. […] Various chemotherapy drugs, including vincristine (Oncovin) and cyclophosphamide (Cytoxan) have been used as a second or third-line treatment choice for chronic ITP patients.
- #46 Conventional Treatmentshttps://pdsa.org/treatments/conventional
Many doctors consider corticosteroids (steroids) for their initial approach for newly diagnosed patients. […] Immunosuppressants, including azathioprine (Imuran), cyclosporine (Sandimmune), diphenylsulfone (Dapsone), Sirolimus (Rapamycin), and mycophenolate mofetil (Cellcept), are considered a second or third-line treatment for ITP. […] Intravenous immunoglobulin (IVIG) is a type of antibody treatment used as a front line therapy to temporarily elevate platelet counts. […] The synthetic androgen (male sex hormone) danazol (Danocrine) is sometimes used to treat ITP when other treatments have failed. […] A new approach to treating ITP is the use of a spleen tyrosine kinase (SYK) inhibitor. […] Second-line ITP treatment approach using platelet growth factors or thrombopoietin (TPO) receptor agonists.
- #47 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
In some patients with an inadequate response to TPO-RA, as mentioned above, a low dose of prednisone may synergize greatly and provide an adequate platelet count. […] Fostamatinib is a newer agent with a unique mechanism of action. It inhibits syk kinase thereby reducing macrophage destruction of platelets. […] Mycophenolate mofetil is a useful agent in ITP but has not been well studied until recently. […] Dapsone has a long history of use in chronic ITP. It is yet another steroid sparing option in responsive ITP patients. […] Danazol is another steroid sparing medication like dapsone and MMF which is helpful in some ITP patients. […] We rarely recommend splenectomy in adult patients except as a last measure or because of patient preference. […] Most of the time we are talking about response and remission. Most ITP patients do well and have a response to some therapy and many will come off therapy completely.
- #48 Immune thrombocytopenia (ITP) – Diagnosis and treatment – Mayo Clinichttps://www.mayoclinic.org/diseases-conditions/idiopathic-thrombocytopenic-purpura/diagnosis-treatment/drc-20352330
People with mild immune thrombocytopenia might need only regular platelet checks. Children usually improve without treatment. Most adults with ITP will need treatment at some point. The condition often gets worse or lasts long, also known as chronic. […] Treatment might include medicines to increase platelet count or surgery to remove the spleen, known as a splenectomy. A health care provider can talk about the pros and cons of treatment options. Some people find the side effects of treatment are worse than the disease. […] If medicine doesn’t make ITP better, surgery to remove the spleen might be the next step. When it works, this surgery quickly ends the attacks on platelets and improves platelet count. […] Rarely, ITP can cause a lot of bleeding. Emergency care usually includes getting blood, also known as transfusion, that contains many platelets. Steroids and immune globulin given through a tube in a vein also might help.
- #49 Immune thrombocytopenia – Symptoms, diagnosis and treatment | BMJ Best Practicehttps://bestpractice.bmj.com/topics/en-gb/138
Immune thrombocytopenia (ITP) in children typically presents with a preceding viral illness and an abrupt onset. There is a female preponderance among adults, who may present with thrombocytopenia with or without bleeding. […] Treatment is based on platelet count and bleeding symptoms. Patients with life-threatening bleeding, regardless of platelet count, can be considered for combination therapy with corticosteroids, intravenous immunoglobulin (IVIG), and platelet transfusion. […] Initial treatment options for newly diagnosed ITP include observation, or a corticosteroid and/or IVIG depending on platelet count and bleeding symptoms. Intravenous anti-D immunoglobulin can be considered in patients who are rhesus-positive and non-splenectomised. […] Subsequent treatment with thrombopoietin receptor agonists, rituximab, or fostamatinib (adults only) can be considered in patients who are unresponsive to, or intolerant of, initial treatment. Splenectomy may also be an option from 12 months after diagnosis. […] Salvage therapy with oral immunosuppressants, such as mycophenolate, azathioprine, or dapsone, can be considered in patients who do not respond to multiple subsequent treatments.
- #50 Immune Thrombocytopenia (ITP) – Hematology and Oncology – Merck Manual Professional Editionhttps://www.merckmanuals.com/professional/hematology-and-oncology/thrombocytopenia-and-platelet-dysfunction/immune-thrombocytopenia-itp
Splenectomy can achieve a complete remission in about two thirds of patients who relapse after initial corticosteroid therapy. […] If thrombocytopenia can be controlled with second-line medical therapies, splenectomy is often not necessary. […] Second-line medical therapies include thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, or other immunosuppressive drugs. […] In children or adults with immune thrombocytopenia and life-threatening bleeding, rapid phagocytic blockade is attempted by giving IVIG 1 g/kg once a day for 1 to 2 days or, in Rh-positive patients, a single dose of IV anti-D immune globulin 75 mcg/kg. […] High-dose methylprednisolone (1 g IV once a day for 3 days) is easier to administer than IVIG or IV anti-D immune globulin but may not be as effective.
- #51 Immune Thrombocytopenia (ITP) – Hematology and Oncology – Merck Manual Professional Editionhttps://www.merckmanuals.com/professional/hematology-and-oncology/thrombocytopenia-and-platelet-dysfunction/immune-thrombocytopenia-itp
Splenectomy can achieve a complete remission in about two thirds of patients who relapse after initial corticosteroid therapy. […] If thrombocytopenia can be controlled with second-line medical therapies, splenectomy is often not necessary. […] Second-line medical therapies include thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, or other immunosuppressive drugs. […] In children or adults with immune thrombocytopenia and life-threatening bleeding, rapid phagocytic blockade is attempted by giving IVIG 1 g/kg once a day for 1 to 2 days or, in Rh-positive patients, a single dose of IV anti-D immune globulin 75 mcg/kg. […] High-dose methylprednisolone (1 g IV once a day for 3 days) is easier to administer than IVIG or IV anti-D immune globulin but may not be as effective.
- #52 Immune Thrombocytopenia (ITP) – Hematology and Oncology – Merck Manual Professional Editionhttps://www.merckmanuals.com/professional/hematology-and-oncology/thrombocytopenia-and-platelet-dysfunction/immune-thrombocytopenia-itp
Patients with ITP and life-threatening bleeding are also given platelet transfusions. […] Treatment of children with immune thrombocytopenia is usually supportive because most children spontaneously recover. […] If mucosal bleeding occurs, corticosteroids or IVIG may be given. […] However, if thrombocytopenia is severe and symptomatic for 6 months, then TPO-RA (eg, romiplostim, eltrombopag) should be considered.
- #53 Thrombocytopenia: Evaluation and Management | AAFPhttps://www.aafp.org/pubs/afp/issues/2022/0900/thrombocytopenia.html
Prophylactic platelet transfusions are recommended for patients with platelet counts less than 10 103 per L or at higher counts with signs of bleeding. […] Patients often require hospitalization, but newly diagnosed ITP with platelet counts of 20 103 per L or greater in patients who are asymptomatic or with minimal mucosal bleeding can be managed in the outpatient setting, provided that definitive hematology follow-up is performed within 24 to 72 hours. […] Initial therapy is corticosteroids or intravenous immune globulin (human) infusion. Recurrent or unresponsive cases are treated with thrombopoietin receptor agonists or immunomodulators (e.g., rituximab). […] Platelet transfusions are the standard of care to manage severe thrombocytopenia and for preprocedural prophylaxis to achieve a platelet count of at least 50 103 per L.
- #54 A Guide To Thrombocytopenia Treatment – Hospital Procedures Consultantshttps://hospitalprocedures.org/a-guide-to-thrombocytopenia-treatment/
Thrombocytopenia treatment depends on what has caused the condition. It is thus crucial to know why a patient has thrombocytopenia. Knowing the root cause will ensure the effectiveness of the thrombocytopenia therapeutic procedures. […] Thrombocytopenia treatment primarily involves addressing the underlying cause of the low platelet count. Other treatment options include: […] Administering steroids can prevent excessive bleeding due to thrombocytopenia. They can reduce antibody production against platelets which can allow the platelet levels to increase over the course of two to four weeks. […] A splenectomy is thus a viable bedside treatment option that entails the surgical removal of the spleen. […] A platelet transfusion may be necessary to prevent excessive bleeding and manage thrombocytopenia, particularly before major surgeries and procedures such as: […] The effects of a transfusion are usually temporary, and the platelet count typically only rises for around three to four days. However, this is enough for a patient to safely undergo different procedures as it can reduce the risk of excessive blood loss.
- #55 Thrombocytopenia (low platelets) | Lymphoma Actionhttps://lymphoma-action.org.uk/about-lymphoma-side-effects-treatment/thrombocytopenia-low-platelets
A platelet transfusion involves having platelets from a healthy donor through a drip into one of your veins. Platelet transfusions aim to increase your platelet count enough to stop any bleeding or to reduce your risk of bleeding. […] If your thrombocytopenia is caused by autoantibodies destroying your platelets, platelet transfusions usually do not help. Instead, the treatment for this type of thrombocytopenia aims to reduce the number of autoantibodies being made, or stop your platelets being destroyed. […] If you are bleeding, or you need to have a procedure that puts you at risk of bleeding, you might need other treatments to help your blood clot, such as: tranexamic acid, which helps prevent bleeding by stopping blood clots from being broken down; an injection of vitamin K, which your body needs to make proteins that help blood clot (clotting factors); fresh frozen plasma (the liquid part of blood, which contains clotting factors) from a healthy donor given through a drip into one of your veins.
- #56 Thrombocytopenia (low platelets) | Lymphoma Actionhttps://lymphoma-action.org.uk/about-lymphoma-side-effects-treatment/thrombocytopenia-low-platelets
A platelet transfusion involves having platelets from a healthy donor through a drip into one of your veins. Platelet transfusions aim to increase your platelet count enough to stop any bleeding or to reduce your risk of bleeding. […] If your thrombocytopenia is caused by autoantibodies destroying your platelets, platelet transfusions usually do not help. Instead, the treatment for this type of thrombocytopenia aims to reduce the number of autoantibodies being made, or stop your platelets being destroyed. […] If you are bleeding, or you need to have a procedure that puts you at risk of bleeding, you might need other treatments to help your blood clot, such as: tranexamic acid, which helps prevent bleeding by stopping blood clots from being broken down; an injection of vitamin K, which your body needs to make proteins that help blood clot (clotting factors); fresh frozen plasma (the liquid part of blood, which contains clotting factors) from a healthy donor given through a drip into one of your veins.
- #57 Immune Thrombocytopenia (ITP) | Boston Children’s Hospitalhttps://www.childrenshospital.org/conditions/immune-thrombocytopenia-itp
Rituximab calms the immune attack on platelets by suppressing B cells, the white blood cells that produce antibodies. It is given as an IV infusion, typically weekly for four weeks, and works for about six to 12 months. It has temporary side effects, including immune reactions to the infusion, joint pain, rash, fever, and an increased risk of infections. […] Mycophenolate and sirolimus: These oral medications help prevent attacks on platelets by inhibiting activity of white blood cells called lymphocytes (B and T cells). Each has different side effects that your care team can discuss with you. […] Hydroxychloroquine is sometimes prescribed if ITP is accompanied by other autoimmune disorders. […] As needed, our team may offer hormone therapy if patients have heavy menstrual bleeding and anti-fibrinolytic medications (aminocaproic acid, tranexamic acid) to reduce nosebleeds, gum bleeding, or heavy menstrual bleeding.
- #58 Thrombocytopenia > Fact Sheets > Yale Medicinehttps://www.yalemedicine.org/conditions/thrombocytopenia
Platelet transfusion is given when a patient with reduced platelet production is experiencing bleeding. Patients undergoing surgery may need platelet transfusions before and after the procedure to lower the risk of bleeding. […] Plasma exchange is often used for patients with bleeding caused by thrombotic thrombocytic purpura (TTP), a rare, life-threatening type of thrombocytopenia.
- #59 Severe Thrombocytopenia: Symptoms, Causes, Treatment, Morehttps://www.healthline.com/health/severe-thrombocytopenia
Sometimes, surgical spleen removal is an option for people with severe thrombocytopenia. […] Therapeutic plasma exchange, or plasmapheresis, is a treatment similar to kidney dialysis. It removes your blood from your body, separates your red and white blood cells from your plasma, and returns your blood to your body with replacement plasma. […] Treatment depends on the underlying cause of severe thrombocytopenia but can include medications, blood transfusions, and surgery.
- #60 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #61 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #62 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #63 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #64 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #65 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #66 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
Heparin-induced thrombocytopenia (HIT) requires immediate treatment once clinically suspected. Treatment is often initiated before confirmatory testing. The first step is to discontinue all heparin products immediately and avoid any further exposure. […] Warfarin may cause microthrombosis in patients with HIT. Such patients typically present with an international normalized ratio (INR) greater than 4, which corresponds to severe protein C depletion. If warfarin is going to be used, it should not be started until therapeutic anticoagulation is achieved with a non-heparin anticoagulant and substantial platelet recovery has occurred; preferably, the platelet count should exceed 150 109/L. […] Platelet transfusions should be avoided in HIT, as they may increase the thrombogenic effect. […] Patients with HIT are at high risk for thrombotic events and should be treated with alternative anticoagulants. These include the following: Parenteral direct thrombin inhibitors – Argatroban, bivalirudin; Indirect factor Xa inhibitors – Fondaparinux, danaparaoid (not available in the United States); Direct oral anticoagulants (DOACs) – Direct factor Xa inhibitors (rivaroxaban, apixaban, edoxaban, betrixaban) and a direct thrombin inhibitor (dabigatran).
- #67 Heparin-Induced Thrombocytopenia Treatment & Management: Approach Considerations, Long-term Monitoringhttps://emedicine.medscape.com/article/1357846-treatment
For patients in need of urgent reversal, because of possible need for invasive procedures or high risk of bleeding, parenteral anticoagulation with argatroban or bivalirudin is preferred. […] For patients with suspected or confirmed HIT who are clinically stable and at average risk of bleeding, guidelines from the American Society of Hematology (ASH) suggest that fondaparinux and the DOACs are reasonable options. […] High-dose intravenous immunoglobulin (IVIG) has been used as an adjunct to anticoagulation in patients with HIT. At a usual dose of 1 g/kg for 2 days, IVIG is typically effective in interrupting platelet activation by HIT antibodies and results in a rapid increase in the platelet count. […] For patients with heparin-induced thrombocytopenia (HIT) who have only isolated thrombocytopenia, therapeutic doses of alternative anticoagulants should be continued until the platelet counts recover to a stable plateau.
- #68https://journals.lww.com/jcge/fulltext/2020/07000/treatment_options_for_thrombocytopenia_in_patients.6.aspx
Thrombocytopenia is a consequence of portal hypertension and is the most common hematological manifestation of chronic liver disease (CLD) (ie, cirrhosis). […] Fortunately, we now have effective and accurate treatment modalities to raise platelet counts before scheduled procedures, known as thrombopoietin receptor agonists. Two drugs in this therapeutic class (avatrombopag and lusutrombopag) are now approved for the treatment of thrombocytopenia in adults with CLD undergoing a procedure and have revolutionized how this is managed. […] In recent years, TPO receptor agonists, which have been used routinely for the treatment of thrombocytopenia associated with other conditions, are being used in CLD. […] The most recently published eltrombopag data in CLD is from the ELEVATE study (Eltrombopag Evaluated for Its Ability to Overcome Thrombocytopenia and Enable Procedures), a phase 3, international, double-blind, placebo-controlled trial.
- #69https://journals.lww.com/jcge/fulltext/2020/07000/treatment_options_for_thrombocytopenia_in_patients.6.aspx
In 2018, 2 oral, selective TPO agonists, avatrombopag and lusutrombopag, were approved by the FDA for thrombocytopenia in adults with CLD undergoing a procedure. […] Overall, the safety profile of avatrombopag in both studies was similar to the placebo, with a comparable overall incidence of treatment-emergent AEs in avatrombopag- and placebo-treated patients in both the low and high baseline platelet count cohorts. […] As the data presented demonstrate, both avatrombopag and lusutrombopag can safely and effectively increase platelets in thrombocytopenic CLD patients undergoing a scheduled procedure. […] The approval of lusutrombopag was based on the results of L-PLUS 1, a Japanese, phase 3, double-blind, parallel-group study of 96 patients with CLD and thrombocytopenia (platelet counts 50,000/L) undergoing invasive procedures. […] Overall, 47.7% (51/107) of patients in the lusutrombopag group and 48.6% (52/107) in the placebo group had at least one treatment-emergent AE.
- #70https://journals.lww.com/jcge/fulltext/2020/07000/treatment_options_for_thrombocytopenia_in_patients.6.aspx
In 2018, 2 oral, selective TPO agonists, avatrombopag and lusutrombopag, were approved by the FDA for thrombocytopenia in adults with CLD undergoing a procedure. […] Overall, the safety profile of avatrombopag in both studies was similar to the placebo, with a comparable overall incidence of treatment-emergent AEs in avatrombopag- and placebo-treated patients in both the low and high baseline platelet count cohorts. […] As the data presented demonstrate, both avatrombopag and lusutrombopag can safely and effectively increase platelets in thrombocytopenic CLD patients undergoing a scheduled procedure. […] The approval of lusutrombopag was based on the results of L-PLUS 1, a Japanese, phase 3, double-blind, parallel-group study of 96 patients with CLD and thrombocytopenia (platelet counts 50,000/L) undergoing invasive procedures. […] Overall, 47.7% (51/107) of patients in the lusutrombopag group and 48.6% (52/107) in the placebo group had at least one treatment-emergent AE.
- #71 Thrombocytopenia: Evaluation and Management | AAFPhttps://www.aafp.org/pubs/afp/issues/2022/0900/thrombocytopenia.html
Certain thrombopoietin receptor agonists (i.e., avatrombopag [Doptelet] and lusutrombopag [Mulpleta]) have been used to raise platelet counts for patients undergoing invasive procedures. However, these medications require five to seven days to increase platelet counts, limiting their use for urgent procedures. […] In addition to patients undergoing procedures, platelet transfusions are often used in patients with severe thrombocytopenia to treat or prevent acute hemorrhage. Based on multiple trials involving patients with hematologic disorders and those undergoing hematopoietic stem cell transplantation, the American Society of Clinical Oncology clinical practice guideline update and the Cochrane Database of Systematic Reviews recommend prophylactic platelet transfusion when platelet counts are less than 10 103 per L, or at higher counts with signs of bleeding.
- #72https://haematologica.org/article/view/haematol.2021.279512
Chemotherapy-induced thrombocytopenia (CIT) is a common complication of the treatment of non-hematologic malignancies. […] In patients with such counts, it is important to exclude other causes of thrombocytopenia (medications, infection, thrombotic microangiopathy, post-transfusion purpura, coagulopathy and immune thrombocytopenia). If these are not present, the common approach is to reduce chemotherapy dose intensity or switch to other agents. […] Thrombopoietic growth factors (recombinant human thrombopoietin, pegylated human megakaryocyte growth and development factor, romiplostim, eltrombopag, avatrombopag and hetrombopag) improve pretreatment and nadir platelet counts, reduce the need for platelet transfusions, and enable chemotherapy dose intensity to be maintained. […] National Comprehensive Cancer Network guidelines permit their use but their widespread adoption awaits adequate phase III randomized, placebo-controlled studies demonstrating maintenance of relative dose intensity, reduction of platelet transfusions and bleeding, and possibly improved survival.
- #73 Low platelet count (thrombocytopenia) | Canadian Cancer Societyhttps://cancer.ca/en/treatments/side-effects/low-platelet-count
If cancer causes low platelet counts, every attempt is made to treat the cancer. However, the treatment itself may cause thrombocytopenia. When chemotherapy causes low platelet counts, the dose may have to be lowered or the length of time between chemotherapy cycles may have to be longer. […] If your platelet count is less than 20,000/mm 3 and you are bleeding or have a lot of bruising, you may need a platelet transfusion. Transfused platelets only last about 3 days, so some people may need more than one transfusion. […] The healthcare team may also give medicines to help the body make more platelets. These are called growth factors. Interleukin-11 is an example of a growth factor. Growth factors may help prevent severe thrombocytopenia.
- #74 Low platelet count (thrombocytopenia) | Canadian Cancer Societyhttps://cancer.ca/en/treatments/side-effects/low-platelet-count
If cancer causes low platelet counts, every attempt is made to treat the cancer. However, the treatment itself may cause thrombocytopenia. When chemotherapy causes low platelet counts, the dose may have to be lowered or the length of time between chemotherapy cycles may have to be longer. […] If your platelet count is less than 20,000/mm 3 and you are bleeding or have a lot of bruising, you may need a platelet transfusion. Transfused platelets only last about 3 days, so some people may need more than one transfusion. […] The healthcare team may also give medicines to help the body make more platelets. These are called growth factors. Interleukin-11 is an example of a growth factor. Growth factors may help prevent severe thrombocytopenia.
- #75 Low platelet count (thrombocytopenia) | Canadian Cancer Societyhttps://cancer.ca/en/treatments/side-effects/low-platelet-count
If cancer causes low platelet counts, every attempt is made to treat the cancer. However, the treatment itself may cause thrombocytopenia. When chemotherapy causes low platelet counts, the dose may have to be lowered or the length of time between chemotherapy cycles may have to be longer. […] If your platelet count is less than 20,000/mm 3 and you are bleeding or have a lot of bruising, you may need a platelet transfusion. Transfused platelets only last about 3 days, so some people may need more than one transfusion. […] The healthcare team may also give medicines to help the body make more platelets. These are called growth factors. Interleukin-11 is an example of a growth factor. Growth factors may help prevent severe thrombocytopenia.
- #76https://haematologica.org/article/view/haematol.2021.279512
The clinicians response to thrombocytopenia in a cancer patient is varied. Reduction of relative dose intensity (RDI) of chemotherapy or radiation is common; less effective regimens may be chosen; treatment may even be precluded. […] Platelet transfusion is often the only immediately available treatment. There is increasing interest in using recombinant human thrombopoietin (rhTPO) or thrombopoietin receptor agonists (TPO-RA) such as romiplostim, eltrombopag, avatrombopag, lusutrombopag, and hetrombopag to enhance platelet production and platelet counts. […] Overall, it is reasonable when confronted with CIT first to assess the underlying need for chemotherapy and the goals of treatment for that particular patient. […] Platelet transfusion support can be used if maintenance of dose intensity is vital for response or survival. […] Despite the lack of Food and Drug Administration approval for these agents, rhTPO and TPO-RA might be considered in patients for whom thrombocytopenia prevents maintenance of dose intensity crucial for remission or survival, and who cannot be supported by platelet transfusions.
- #77 Idiopathic Thrombocytopenic Purpura (ITP) | Children’s Hospital of Philadelphiahttps://www.chop.edu/conditions-diseases/idiopathic-thrombocytopenic-purpura-itp
Specific treatment for ITP will be determined by your child’s physician based on: […] Not all children with ITP require treatment. Close monitoring of your child’s platelets and prevention of serious bleeding complications may be the course of action chosen until the body is able to correct the disorder on its own. Many children with ITP are able to spontaneously recover in about 2 to 4 days. […] When treatment is necessary, the two most common forms of treatment are: […] Steroids, which help prevent bleeding by decreasing the rate of platelet destruction. Steroids, if effective, will result in an increase in platelet counts seen within two to three weeks. Side effects may include irritability, stomach irritation, weight gain, hypertension and acne. […] Intravenous gamma globulin (IVGG), a protein that contains many antibodies and also slows the destruction of platelets. IVGG works more quickly than steroids (within 24 to 48 hours).
- #78 Immune Thrombocytopenia | Treatment & Management | Point of Carehttps://www.statpearls.com/point-of-care/23385
Severe ITP, when platelet counts are below 20,000/L, warrants medical treatment. Immunosuppressive therapy is the cornerstone of management. […] The ASH 2019 ITP guidelines recommend adult and pediatric algorithms based on the need for treatment and inpatient management. […] ASH guidelines recommend corticosteroids over observation in adults with newly diagnosed ITP and a platelet count of less than 30,000/L if they are asymptomatic or have minor mucocutaneous bleeding. […] ASH suggests observation instead of corticosteroids in children with newly diagnosed ITP and no or minor bleeding. […] ASH also recommends observation over intravenous immunoglobulin (IVIG) and anti-D immunoglobulin for these children. […] For adults with ITP, the ASH guideline panel recommends second-line therapies if they are dependent on corticosteroids, unresponsive to corticosteroids for at least 3 months, or considered to have chronic ITP.
- #79 Pediatric immune thrombocytopenia (ITP) treatment – Kim – Annals of Bloodhttps://aob.amegroups.org/article/view/6316/html
Importantly, severe life threatening side effects are reported for IVIG and anti-D. […] The majority of children with newly diagnosed ITP are asymptomatic or develop only mild bleeding symptoms. […] Treatment is indicated if parents do not feel they can appropriately limit their child’s activity, such as in the case of an active toddler or adolescent engaging in sports. […] Children with severe or emergent bleeding such as hematuria, hematochezia, or other internal hemorrhage such as intracranial bleeding require immediate and often multi-modal treatment. […] Ultimately, the decision to treat pediatric ITP patients is an individualized one and shared decision making between the patient, caregiver and physician is critical. […] Anti-D is another frontline option in children who require treatment.
- #80 Pediatric immune thrombocytopenia (ITP) treatment – Kim – Annals of Bloodhttps://aob.amegroups.org/article/view/6316/html
Pediatric immune thrombocytopenia (ITP) is a heterogeneous autoimmune condition with variability in etiology, bleeding phenotype, need for treatment and response to therapy, as well as duration of disease. […] When treatment is required, first line therapies are relatively standardized and aim to rapidly diminish bleeding risk. […] ITP management continues to evolve: as research expands our understanding of the molecular underpinnings of ITP, providers are increasingly able to refine and individualize treatment regimens. […] For children requiring therapy but without life threatening bleeding, corticosteroids are the recommended first line therapy over IVIG or anti-D. […] Corticosteroids are suggested over IVIG or anti-D by the 2019 American Society of Hematology ITP guidelines, due to low cost, universal availability, ease of outpatient administration, no exposure to multiple blood donors and overall minimal side effects associated with short courses of steroids.
- #81 Immune Thrombocytopenia (ITP) Treatment & Management: Approach Considerations, Thrombopoietin Receptor Agonists, Treatment in Childrenhttps://emedicine.medscape.com/article/202158-treatment
In the past decade, thrombopoietin receptor agonists (TPO-RAs) have entered clinical practice as second-line agents, with robust evidence supporting their efficacy. […] Eltrombopag was studied in a phase III double-blind trial in adults with previously treated ITP lasting more than 6 months and with platelet counts lower than 30,000/L. […] Eltrombopag has a boxed warning for the risk of severe and potentially life-threatening hepatotoxicity. […] Avatrombopag, an oral TPO receptor agonist, was approved by the FDA in 2019 for adults with chronic ITP who have had an insufficient response to a previous treatment. […] For pediatric patients requiring treatment, ASH suggests a short course of corticosteroids for first-line treatment rather than IVIG or anti-D immunoglobulin. […] For children or adolescents with ITP who have significant ongoing bleeding despite treatment with IVIG, RhIG, or conventional doses of corticosteroids, ASH suggests a TPO-RA rather than rituximab or splenectomy as second-line treatment.
- #82 Immune Thrombocytopenia | Treatment & Management | Point of Carehttps://www.statpearls.com/point-of-care/23385
Second-line treatments for adults include TPO-RAs (ie, eltrombopag, romiplostim, or avatrombopag), rituximab, and splenectomy. […] Splenectomy is typically reserved for children with severe thrombocytopenia and significant hemorrhagic symptoms that require multiple pharmacological interventions. […] In 2018, the FDA approved the spleen tyrosine kinase inhibitor fostamatinib as an option for ITP refractory to second-line treatments. […] The ASH guideline panel recommends either prednisone 0.5-2.0 mg/kg per day or dexamethasone 40 mg daily for 4 days as initial corticosteroid therapy for adults newly diagnosed with ITP.
- #83 Conventional Treatmentshttps://pdsa.org/treatments/conventional
This is a surgical procedure to remove the spleen. […] More of a strategy than a treatment, Watchful Waiting means choosing to live with you or your child’s current platelet counts while carefully monitoring the disease and treatment options. […] Treatment information does not represent an endorsement by PDSA or its medical advisors. For advice on your unique medical condition, please consult your healthcare provider.
- #84 Immune Thrombocytopenia (ITP): Symptoms, Diagnosis & Treatmenthttps://my.clevelandclinic.org/health/diseases/5726-immune-thrombocytopenia
Whats the treatment for immune thrombocytopenia? Often, children with immune thrombocytopenia have mild symptoms and dont need treatment, but most adults will. If you do need treatment, your healthcare provider may prescribe medications to boost your platelet count or keep your immune system from attacking your platelets. Medications may include: […] Your provider may recommend other steps like: […] Treatment side effects […] The outlook varies depending on your or your childs unique situation. If your child has ITP, theyll probably get better within a few weeks or months without needing treatment. If you have persistent or chronic ITP, you may need medications and other treatments to manage your symptoms depending on how low your platelet count is, but youll always have the condition. Healthcare providers cant cure immune thrombocytopenia, but many people with chronic ITP live for decades with the condition.
- #85 Thrombocytopenia and Platelet Function Disorders | Doctorhttps://patient.info/doctor/thrombocytopenia-and-platelet-function-disorders
Managing thrombocytopenia […] Patients must be aware that they should be seen straightaway if new symptoms such as bruising or bleeding occur. […] Indications for urgent referral include severe thrombocytopenia (20109/L), severe bleeding, and red cell fragments or blasts on the blood film. […] Further management depends on the underlying cause. […] Treatment of patients with suspected platelet dysfunction is generally specific to the underlying cause but may include desmopressin and platelet transfusion. […] Antifibrinolytic therapy (epsilon aminocaproic acid or tranexamic acid) may be useful, especially for mucosal bleeding, but should not be used in patients with haematuria or DIC. […] rFVIIa has been used to treat bleeding in patients with acquired and inherited platelet disorders, but is associated with an increased risk of thrombosis.
- #86 Self-Care for ITPhttps://www.everydayhealth.com/immune-thrombocytopenia/itp-self-care-tips/
For most people who have ITP, treatment includes the use of drugs that suppress your immune systems attack on your platelets, according to the Mayo Clinic at least initially, until your platelet levels are high enough. […] While some people with ITP are at high risk for bleeding and will need to follow stricter precautions for injuries, such as topical or oral medications, most people who have ITP can treat minor cuts in the usual manner, says Tellez. […] Medications like aspirin or ibuprofen, and some supplements like fish oil, alter the way platelets work, says Tellez. And so theyre going to potentially put someone at increased risk for bleeding. […] Connecting with other people online or in a local support group can help you learn what to expect with ITP, but its important to make sure that any online forums you visit are moderated and supply accurate information. […] To get a good grasp of how to manage ITP, you should read up on the disorder. It doesnt have to be pages of heavy-duty literature, says Tellez. Its just some basic concepts, some bullet points about things to do. Just those few things can be very helpful.
- #87 Thrombocytopenia (Low Platelet Count): Definition, Causes, Symptoms, and Treatmenthttps://www.webmd.com/a-to-z-guides/thrombocytopenia-causes-treatment
If your platelet count isn’t too low, you might not need treatment. […] For severe thrombocytopenia, your doctor might give you: steroid medicines to keep your body from destroying platelets if the problem is related to your immune system, intravenous immune globulin (IVIG) if you can’t take steroids or if you need a higher platelet count quickly, blood or platelets from a healthy person, called a transfusion, surgery to remove your spleen. […] If your condition continues despite other treatments, your doctor may prescribe medicines such as eltrombopag (Promacta, Revolade), fostamatinib (Tavalisse), and romiplostim (Nplate). […] To prevent bleeding when your platelet count is low: don’t take medicines that can affect how well your platelets work, like aspirin and ibuprofen, limit how much alcohol you drink, because it can make bleeding worse, don’t take part in contact sports, like football or boxing, in which you might get injured, use a soft toothbrush to protect your gums, wear a seatbelt when you’re in a car, and put on gloves and goggles when you work with power tools to prevent injury.
- #88 The Immune Thrombocytopenia Treatment Landscape: A Focus on Patient Perspectives and Unmet Clinical Needs – European Medical Journalhttps://www.emjreviews.com/hematology/symposium/the-immune-thrombocytopenia-treatment-landscape-a-focus-on-patient-perspectives-and-unmet-clinical-needs-s060424/
There is therefore still a need for therapies with both improved efficacy and safety profiles, Ghanima concluded. […] Physicians therefore need to move beyond platelet count and consider the therapy-related disease impact, such as AEs and direct/indirect costs, as well as the patient experience in terms of bleeding severity, health-related QoL (HRQoL), fatigue, and cognition, alongside the priorities of patients and their families. […] There is also a need to expand the incorporation of HRQoL measures into clinical studies. […] Shared decision-making itself represents a process by which clinicians and patients come together in a deliberate conversation to align their respective priorities, which can be facilitated by decision-aids. […] With a plethora of new treatment approaches on the horizon for ITP, Kuter gave a pathophysiological overview of the disorder and outlined the mode of action (MOA) of novel therapies in clinical development.
- #89 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
A key aspect in caring for ITP patients is to have a mutual understanding of the terms response, remission and cure. […] My overall treatment recommendations in ITP are as follows: Many ITP patients do not need treatment; Initial treatment is corticosteroids, preferably prednisone, and if severe bleeding, IVIG; Splenectomy works but should be delayed at least 12 to 24 months. […] TPO-RA are highly effective; low rate of adverse effects; improve Health-related Quality of Life; may not need to be forever; over half come off therapy within 2 years. […] A large number of very promising new ITP treatments (Bruton kinase inhibitors, neonatal Fc receptor inhibitors, anti-CD 38 antibodies) are being developed and patients should be offered access to these trials.
- #90 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
A key aspect in caring for ITP patients is to have a mutual understanding of the terms response, remission and cure. […] My overall treatment recommendations in ITP are as follows: Many ITP patients do not need treatment; Initial treatment is corticosteroids, preferably prednisone, and if severe bleeding, IVIG; Splenectomy works but should be delayed at least 12 to 24 months. […] TPO-RA are highly effective; low rate of adverse effects; improve Health-related Quality of Life; may not need to be forever; over half come off therapy within 2 years. […] A large number of very promising new ITP treatments (Bruton kinase inhibitors, neonatal Fc receptor inhibitors, anti-CD 38 antibodies) are being developed and patients should be offered access to these trials.
- #91 The treatment of immune thrombocytopenia (ITP)âfocus on thrombopoietin receptor agonists – Kuter – Annals of Bloodhttps://aob.amegroups.org/article/view/6335/html
A key aspect in caring for ITP patients is to have a mutual understanding of the terms response, remission and cure. […] My overall treatment recommendations in ITP are as follows: Many ITP patients do not need treatment; Initial treatment is corticosteroids, preferably prednisone, and if severe bleeding, IVIG; Splenectomy works but should be delayed at least 12 to 24 months. […] TPO-RA are highly effective; low rate of adverse effects; improve Health-related Quality of Life; may not need to be forever; over half come off therapy within 2 years. […] A large number of very promising new ITP treatments (Bruton kinase inhibitors, neonatal Fc receptor inhibitors, anti-CD 38 antibodies) are being developed and patients should be offered access to these trials.
- #92 Recent advances in treatments of adult immune thrombocytopeniahttps://pmc.ncbi.nlm.nih.gov/articles/PMC9057657/
Immune thrombocytopenia (ITP) is isolated thrombocytopenia characterized by autoimmune-mediated disruption of platelet without other etiologies. Treatments for chronic ITP consist of corticosteroids, intravenous immunoglobulins, anti-D immunoglobulin, rituximab, thrombopoietin receptor agonists, immunosuppressants and splenectomy. Although current therapies are effective in over two-thirds of patients, some patients are refractory to therapies or fail to achieve long-term responses. Recently, great advance has been made in identifying various mechanisms involved in ITP pathogenesis, and new treatments targeting these pathways are being developed. Novel agents such as splenic tyrosine kinase inhibitor, Bruton kinase inhibitor, plasma cell targeting therapies, neonatal Fc receptor inhibitor, platelet desialylation inhibitor, and inhibition of the classical complement pathway are expected to be effective for ITP treatment. This review summarizes current strategies and emerging therapies of ITP.
- #93 Recent advances in treatments of adult immune thrombocytopeniahttps://pmc.ncbi.nlm.nih.gov/articles/PMC9057657/
Immune thrombocytopenia (ITP) is isolated thrombocytopenia characterized by autoimmune-mediated disruption of platelet without other etiologies. Treatments for chronic ITP consist of corticosteroids, intravenous immunoglobulins, anti-D immunoglobulin, rituximab, thrombopoietin receptor agonists, immunosuppressants and splenectomy. Although current therapies are effective in over two-thirds of patients, some patients are refractory to therapies or fail to achieve long-term responses. Recently, great advance has been made in identifying various mechanisms involved in ITP pathogenesis, and new treatments targeting these pathways are being developed. Novel agents such as splenic tyrosine kinase inhibitor, Bruton kinase inhibitor, plasma cell targeting therapies, neonatal Fc receptor inhibitor, platelet desialylation inhibitor, and inhibition of the classical complement pathway are expected to be effective for ITP treatment. This review summarizes current strategies and emerging therapies of ITP.
- #94 Thrombocytopenia – Wikipediahttps://en.wikipedia.org/wiki/Thrombocytopenia
Human-induced pluripotent stem cell-derived platelets is a technology currently being researched by the private sector, in association with the Biomedical Advanced Research and Development Authority and the U.S. Department of Health and Human Services, that would create platelets outside the human body.
- #95 Treatment of Immune Thrombocytopenia: Contextualization from a Historical Perspectivehttps://www.mdpi.com/2038-8330/16/3/39
Splenectomy was the first scientifically based treatment for ITP. […] The main complications associated with splenectomy are postoperative hemorrhage, infection by encapsulated bacteria, sepsis, and venous or arterial thrombosis. […] In the absence of specific clinical guidelines, treatment must be individualized.
- #96 Thrombocytopenia – symptoms, causes, diagnosis and treatment | healthdirecthttps://www.healthdirect.gov.au/thrombocytopenia
Thrombocytopenia is when you have low levels of platelets in your blood. […] Treatment depends on the cause of your thrombocytopenia. […] Treatment will depend on the cause of your thrombocytopenia.
- #97 Chronic Immune Thrombocytopenia (ITP)https://www.webmd.com/a-to-z-guides/chronic-immune-thrombocytopenia
ITP may not need treatment. Your doctor might just check your platelet count and watch your symptoms on a regular schedule. Your treatment will depend on your platelet count, and the risk and seriousness of a potential bleed. […] If you do need treatment, the one your doctor recommends will depend on your: Symptoms, Platelet count, Age, Other medical conditions, Preferences. […] Corticosteroids are the first treatment for most people with newly diagnosed ITP and a low platelet count. These medicines may stop your immune system from destroying platelets. They can also lower your chances of bleeding for no reason (spontaneous bleeding). […] You may need a platelet transfusion if you have: Serious bleeding (is constant or causes you to fall), Critical bleeding (bleeding in organs like your brain, stomach, or spine), Platelets lower than 20,000 per microliter.
- #98 Second-Line Treatment of Persistent Immune Thrombocytopenia – Hematology Advisorhttps://www.hematologyadvisor.com/cch/second-line-treatment-persistent-immune-thrombocytopenia/
Immune thrombocytopenia (ITP) is an acquired autoimmune disease associated with an increased risk of bleeding in adults and children. Treatment of ITP starts with a correct diagnosis. For patients newly diagnosed with ITP, the American Society of Hematology (ASH) guidelines recommends initiating treatment when their platelet count falls below 30 109/L regardless of the presence of bleeding. The goal of treatment is to increase platelet counts to within a hemostatic range to decrease bleeding risk. The first-line treatments for the management of ITP include corticosteroids (prednisone or dexamethasone), intravenous immunoglobulin, and anti-D immunoglobulin. The primary treatment goals are to halt active bleeding events, limit future hemorrhage, and achieve a sustained increase in platelet count with minimal adverse effects. To prevent the misuse of corticosteroids and improve long-term clinical outcomes, patients should switch from treatment with corticosteroids to alternative options earlier in the course of treatment. Consequently, second-line treatments are needed to achieve more durable responses. The current treatment options used in the second-line setting include thrombopoietin receptor agonists (TPO-RAs), splenectomy, rituximab. The choice of therapy is determined by likelihood of remission, patient preference, adverse effects, route of administration, and cost. TPO-RAs are currently the only therapy specifically developed to treat ITP. These agents stimulate platelet production by binding to the transmembrane domain of the thrombopoietin receptor and inducing proliferation and differentiation of megakaryocytes. TPO-RAs have been shown to be effective and safe in randomized clinical trials, reduce bleeding events and the need for emergency treatments, and improve HRQOL in patients with chronic ITP. The ASH guideline panel conditionally recommends the following treatment options in adults with ITP lasting 3 months who are corticosteroid dependent or have no response to corticosteroids: Either splenectomy or a TPO-RA; TPO-RAs are recommended over rituximab; Rituximab is recommended over a splenectomy. The choice of treatment should be based on disease duration, frequency of bleeding episodes, the patients age, comorbidities, treatment preference regarding adverse effects, route of administration, cost, and effects on HRQOL.
- #99 Second-Line Treatment of Persistent Immune Thrombocytopenia – Hematology Advisorhttps://www.hematologyadvisor.com/cch/second-line-treatment-persistent-immune-thrombocytopenia/
Immune thrombocytopenia (ITP) is an acquired autoimmune disease associated with an increased risk of bleeding in adults and children. Treatment of ITP starts with a correct diagnosis. For patients newly diagnosed with ITP, the American Society of Hematology (ASH) guidelines recommends initiating treatment when their platelet count falls below 30 109/L regardless of the presence of bleeding. The goal of treatment is to increase platelet counts to within a hemostatic range to decrease bleeding risk. The first-line treatments for the management of ITP include corticosteroids (prednisone or dexamethasone), intravenous immunoglobulin, and anti-D immunoglobulin. The primary treatment goals are to halt active bleeding events, limit future hemorrhage, and achieve a sustained increase in platelet count with minimal adverse effects. To prevent the misuse of corticosteroids and improve long-term clinical outcomes, patients should switch from treatment with corticosteroids to alternative options earlier in the course of treatment. Consequently, second-line treatments are needed to achieve more durable responses. The current treatment options used in the second-line setting include thrombopoietin receptor agonists (TPO-RAs), splenectomy, rituximab. The choice of therapy is determined by likelihood of remission, patient preference, adverse effects, route of administration, and cost. TPO-RAs are currently the only therapy specifically developed to treat ITP. These agents stimulate platelet production by binding to the transmembrane domain of the thrombopoietin receptor and inducing proliferation and differentiation of megakaryocytes. TPO-RAs have been shown to be effective and safe in randomized clinical trials, reduce bleeding events and the need for emergency treatments, and improve HRQOL in patients with chronic ITP. The ASH guideline panel conditionally recommends the following treatment options in adults with ITP lasting 3 months who are corticosteroid dependent or have no response to corticosteroids: Either splenectomy or a TPO-RA; TPO-RAs are recommended over rituximab; Rituximab is recommended over a splenectomy. The choice of treatment should be based on disease duration, frequency of bleeding episodes, the patients age, comorbidities, treatment preference regarding adverse effects, route of administration, cost, and effects on HRQOL.
- #100 Chronic Immune Thrombocytopenia (ITP)https://www.webmd.com/a-to-z-guides/chronic-immune-thrombocytopenia
ITP may not need treatment. Your doctor might just check your platelet count and watch your symptoms on a regular schedule. Your treatment will depend on your platelet count, and the risk and seriousness of a potential bleed. […] If you do need treatment, the one your doctor recommends will depend on your: Symptoms, Platelet count, Age, Other medical conditions, Preferences. […] Corticosteroids are the first treatment for most people with newly diagnosed ITP and a low platelet count. These medicines may stop your immune system from destroying platelets. They can also lower your chances of bleeding for no reason (spontaneous bleeding). […] You may need a platelet transfusion if you have: Serious bleeding (is constant or causes you to fall), Critical bleeding (bleeding in organs like your brain, stomach, or spine), Platelets lower than 20,000 per microliter.
- #101 The Immune Thrombocytopenia Treatment Landscape: A Focus on Patient Perspectives and Unmet Clinical Needs – European Medical Journalhttps://www.emjreviews.com/hematology/symposium/the-immune-thrombocytopenia-treatment-landscape-a-focus-on-patient-perspectives-and-unmet-clinical-needs-s060424/
Accumulated data on TPO-RAs indicate that only around 25-32% of patients are able to maintain stable platelet counts 6-12 months after therapy discontinuation. […] Side effects constitute another key consideration when selecting ITP therapy, most notably infections and the risk of thromboembolism. […] Thromboembolism represents another important risk with currently available ITP therapies. […] Ultimately, Ghanima stressed that patient satisfaction with treatment remains the most important metric. […] In summary, the main efficacy limitations of current therapies for ITP lie in the limited rates of durable responses and remissions. […] Key safety concerns include the increased risk of infection associated with immunosuppressants and the increased risk of thromboembolism associated with TPO-RAs.
- #102 Treatment of Immune Thrombocytopenia: Contextualization from a Historical Perspectivehttps://www.mdpi.com/2038-8330/16/3/39
Immune thrombocytopenia (ITP) is an autoimmune disease characterized by an isolated decrease in platelet count and an increased risk of bleeding. […] In this article, we review the treatment of ITP from a historical perspective, discussing first line and second line treatments, and management of refractory disease. […] The first-line treatments for ITP continue to be corticosteroids and nonspecific intravenous immunoglobulins (IVIGs). Second-line therapy should be individualized, taking into account both comorbidities and the patientâs opinion. Thrombopoietin receptor agonists (TPO-RAs) have drastically changed the management of the disease, practically displacing splenectomy. […] The treatment of ITP has the following two main objectives: on the one hand, to maintain a platelet count that prevents severe bleeding, which is usually achieved with platelet counts greater than 30â50 Ã 10^9/L, and, on the other hand, to avoid unnecessary treatments in asymptomatic patients, since the side effects may be more important than the complications derived from the disease itself.