Zespół poems – Leczenie

Zespół poems
Leczenie

Zespół POEMS jest rzadkim klonalnym zaburzeniem komórek plazmatycznych o wieloukładowym przebiegu, wymagającym wczesnej diagnozy i natychmiastowego leczenia ukierunkowanego na klon plazmatyczny. Leczenie dzieli się na terapię celowaną oraz objawową, a wybór metody zależy od zaawansowania choroby. W przypadku ograniczonej choroby (1-3 ogniska kostne bez zajęcia szpiku) preferowana jest radioterapia w dawce około 40 Gy, często wspomagana kortykosteroidami (np. deksametazon 40 mg/dzień lub prednizon 1 mg/kg/dzień). Przy zajęciu szpiku lub liczbie zmian kostnych >3 wskazana jest chemioterapia wysokodawkowa z melfalanem (140-200 mg/m²) i autologicznym przeszczepem komórek macierzystych (ASCT), który zapewnia trwałe odpowiedzi u ~90% pacjentów, z przeżyciem wolnym od progresji 98% po roku i 75% po 5 latach. U pacjentów niekwalifikujących się do ASCT stosuje się schematy oparte na lenalidomidzie z deksametazonem, melfalanie z deksametazonem lub inhibitorach proteasomu, z uwzględnieniem neurotoksyczności bortezomibu. Terapia objawowa obejmuje leczenie polineuropatii, przewodnienia, endokrynopatii oraz trombocytozy, a monitorowanie choroby opiera się m.in. na poziomach VEGF, IL-12 i ocenie obrzęku tarczy nerwu wzrokowego.

Leczenie zespołu POEMS

Strategie leczenia w zależności od zaawansowania choroby
Autologiczne przeszczepienie komórek macierzystych
Leki immunomodulujące i inhibitory proteasomu
Radioterapia
Leczenie objawowe
Monitorowanie i ocena odpowiedzi na leczenie

Leczenie zespołu POEMS w kontekście chorób współistniejących

Perspektywy i nowe kierunki leczenia

Rokowanie i wyniki leczenia

Podsumowanie strategie terapeutyczne
Kolejne rozdziały

Leczenie zespołu POEMS

Zespół POEMS (polineuropatia, organomegalia, endokrynopatia, białko monoklonalne i zmiany skórne) jest rzadkim zaburzeniem klonalnym komórek plazmatycznych charakteryzującym się objawami wieloukładowymi. Ze względu na rzadkość występowania tego zespołu, jednoznaczne wytyczne dotyczące leczenia nadal nie zostały w pełni opracowane. Natychmiastowe leczenie ukierunkowane na proliferację komórek plazmatycznych prowadzi do dramatycznej poprawy u większości pacjentów. Kluczową kwestią jest wczesna diagnoza i natychmiastowa terapia przeciwko klonalnym komórkom plazmatycznym, co zapewnia najlepsze wyniki kliniczne¹².

Leczenie zespołu POEMS można podzielić na dwie główne kategorie: terapię ukierunkowaną na klon komórek plazmatycznych oraz leczenie objawowe. Dla osiągnięcia najlepszych wyników, podejście terapeutyczne powinno obejmować obie strategie³⁴.

Strategie leczenia w zależności od zaawansowania choroby

Wybór metody leczenia zależy głównie od rozległości choroby oraz od stanu ogólnego pacjenta. Leczenie można podzielić na następujące kategorie⁵⁶:

Choroba ograniczona

Jeśli choroba jest ograniczona do 1-3 ognisk kostnych bez zajęcia szpiku kostnego, preferowaną metodą leczenia jest radioterapia. Stosuje się dawki promieniowania rzędu 40 Gy, ponieważ celem jest potencjalne wyleczenie, a nie tylko łagodzenie objawów⁷⁸. W przypadku gdy pacjent doświadcza szybkiego pogorszenia stanu zdrowia, równoczesne stosowanie kortykosteroidów (np. deksametazonu 40 mg/dzień przez 1-4 dni co 2 tygodnie lub prednizonu 1 mg/kg dziennie przez dwa tygodnie) stanowi racjonalną terapię wspomagającą radiację⁹.

Choroba rozsiana

W przypadku zajęcia szpiku kostnego lub obecności więcej niż trzech zmian kostnych, zalecane jest leczenie systemowe. Wysokodawkowa chemioterapia oparta na melfalanie (140-200 mg/m²) z następczym autologicznym przeszczepieniem komórek macierzystych krwi obwodowej (ASCT) jest uważana przez wielu ekspertów za leczenie pierwszego wyboru u pacjentów kwalifikujących się do tej procedury¹⁰¹¹¹².

Dla pacjentów niekwalifikujących się do ASCT, dostępne są inne opcje terapeutyczne, takie jak schematy oparte na lekach immunomodulujących (IMiD) czy inhibitorach proteasomu¹³.

Autologiczne przeszczepienie komórek macierzystych

Przeszczepienie autologicznych komórek macierzystych krwi obwodowej (ASCT) jest preferowaną metodą leczenia dla pacjentów z rozsianą chorobą, którzy kwalifikują się do tej procedury. ASCT zapewnia trwałe odpowiedzi u około 90% kwalifikujących się pacjentów¹⁴¹⁵.

Przeżycie wolne od progresji choroby wynosi 98% i 75% odpowiednio po 1 i 5 latach. Pacjenci z zespołem POEMS mają jednak wyższe niż oczekiwane zapotrzebowanie na transfuzje, przy medianie liczby transfuzji płytek krwi i krwinek czerwonych wynoszącej odpowiednio 5 jednostek aferezy i 6 jednostek, oraz opóźnionym wszczepieniem neutrofilów¹⁶.

Przed przeszczepieniem komórek macierzystych może być stosowana terapia indukcyjna, która poprawia stan ogólny pacjenta, zmniejsza ryzyko powikłań okołoprzeszczepowych i przyspiesza czas odpowiedzi na leczenie¹⁷.

Leki immunomodulujące i inhibitory proteasomu

Dla pacjentów niekwalifikujących się do ASCT, dostępne są inne opcje terapeutyczne¹⁸¹⁹:

Lenalidomid z deksametazonem (LDex): Zalecany schemat to lenalidomid (15-25 mg doustnie) przyjmowany w dniach 1-21 co 28 dni wraz z tygodniowym deksametazonem 20-40 mg. Schemat ten wykazuje obiecujące wyniki w redukcji VEGF do normalnych poziomów i poprawie funkcji neurologicznych u wielu pacjentów, przy minimalnych działaniach niepożądanych²⁰²¹²².
Melfalan z deksametazonem (MDex): Terapia MDex obejmuje melfalan 10 mg/m² + deksametazon 40 mg w dniach 1-4, jeden cykl co 28 dni przez łącznie 9 cykli²³.
Bortezomib: Inhibitor proteasomu, który hamuje wydzielanie VEGF i IL-6, zatrzymuje angiogenezę i indukuje apoptozę komórek B w sposób zależny od dawki. Jednak ze względu na neurotoksyczność, jego stosowanie może być ograniczone u pacjentów z już istniejącą neuropatią²⁴²⁵.
Daratumumab: W przypadkach zespołu POEMS odpowiedź na daratumumab w połączeniu z lenalidomidem była godna uwagi, co sugeruje, że ta kombinacja jest obiecującą, mniej toksyczną alternatywą dla ASCT²⁶²⁷.

Radioterapia

Radioterapia jest leczeniem pierwszego rzutu dla pacjentów z izolowanym plazmocytomem bez zajęcia szpiku kostnego. Terapia promieniowaniem w dawkach 0,4-0,5 Gy na ograniczone obszary może poprawić stan osteosklerotycznych zmian u ponad 50% pacjentów²⁸.

Niektórzy autorzy zalecają terapię systemową dla pacjentów, u których proces chorobowy nie stabilizuje się 3-6 miesięcy po zakończeniu radioterapii²⁹.

Leczenie objawowe

Oprócz leczenia ukierunkowanego na klon komórek plazmatycznych, istotne jest również leczenie objawowe³⁰:

Polineuropatia: Dwa najlepsze sposoby leczenia polineuropatii obwodowej to ukierunkowanie terapii na klon komórek plazmatycznych oraz intensywna fizjoterapia i terapia zajęciowa. Bolesną neuropatię obwodową można leczyć takimi lekami jak gabapentyna, pregabalina, amitryptylina, nortryptylina, duloksetyna, plastry z lidokainą oraz preparaty z ketaminą, lidokainą i amitryptyliną³¹³²³³.
Przewodnienie: Objawy przewodnienia występują u większości pacjentów w postaci obrzęków obwodowych; wodobrzusze, wysięki opłucnowe i osierdziowe mogą być obecne nawet u 50% pacjentów. Stosowanie seryjnych paracentez i diurezy wymuszonej albuminami może przynieść korzyści³⁴.
Endokrynopatia: Zaburzenia endokrynologiczne mogą ulec poprawie po chemioterapii, w tym możliwe jest stopniowe odstawienie hormonów tarczycy, androgenów i kortykosteroidów u co najmniej jednej trzeciej pacjentów³⁵.
Obrzęk tarczy nerwu wzrokowego: W większości przypadków obrzęk tarczy nerwu wzrokowego jest bezobjawowy, ale gdy tak nie jest i gdy ciśnienie jest wysokie, leczenie acetazolamidem i kortykosteroidami może kontrolować objawy³⁶.
Trombocytoza: Około 50% pacjentów z POEMS ma trombocytozę. Można rozważyć stosowanie hydroksymocznika u pacjentów ze znaczną trombocytozą, którzy przebyli incydent mózgowy³⁷.

Monitorowanie i ocena odpowiedzi na leczenie

Regularne monitorowanie pacjentów z zespołem POEMS jest kluczowe dla oceny odpowiedzi na leczenie. Zaleca się wizyty kontrolne co najmniej co 3 miesiące przez kilka lat³⁸. Ocena choroby powinna być przeprowadzana po sześciu miesiącach lub wcześniej, jeśli istnieją obawy dotyczące niewystarczającej odpowiedzi³⁹.

Parametry takie jak poziom VEGF, IL-12 oraz obrzęk tarczy nerwu wzrokowego wykazują pozytywną korelację z aktywnością choroby i mogą być użyteczne w monitorowaniu przebiegu choroby⁴⁰.

Leczenie zespołu POEMS w kontekście chorób współistniejących

Leczenie zespołu POEMS może być skomplikowane przez współistniejące choroby lub specyficzne manifestacje kliniczne⁴¹:

Zespół POEMS z chorobą Castlemana: Pacjenci z współistniejącą chorobą Castlemana (POEMS-CD) mogą lepiej reagować na talidomid plus deksametazon⁴². Jednak według niektórych doniesień, pacjenci ze współistniejącą chorobą Castlemana mogą mieć gorsze całkowite przeżycie w porównaniu do pacjentów bez niej⁴³.
Zespół POEMS z nadciśnieniem płucnym: U pacjentów z zespołem POEMS i nadciśnieniem płucnym leczenie wysokimi dawkami steroidów wydaje się przynosić poprawę⁴⁴.
Zakrzepica: Do 40% pacjentów doświadcza zakrzepicy tętniczej i żylnej w momencie prezentacji lub podczas aktywnego leczenia, co prawdopodobnie wynika z choroby podstawowej, unieruchomienia pacjenta z powodu upośledzenia neurologicznego, a także leczenia, w tym leków immunomodulujących i steroidów⁴⁵.

Perspektywy i nowe kierunki leczenia

Prowadzone są badania nad nowymi metodami leczenia zespołu POEMS⁴⁶:

Terapie celowane w czynniki patogenetyczne: Nowe podejścia terapeutyczne obejmują celowanie w kluczowe czynniki w patogenezie POEMS, w tym VEGF, IL-6, IL-12 i TNF⁴⁷.
Bevacizumab: Mimo że teoretycznie strategia anty-VEGF jest atrakcyjna, wyniki stosowania bevacizumabu były mieszane. Ze względu na brak skuteczności i potencjał zwiększania śmiertelności w niektórych doniesieniach, bevacizumab nie powinien być stosowany w praktyce klinicznej do leczenia zespołu POEMS⁴⁸⁴⁹.
Terapia CAR-T anty-BCMA: Istnieją doniesienia o skutecznym leczeniu zespołu POEMS za pomocą komórek CAR-T skierowanych przeciwko antygenowi dojrzewania komórek B (BCMA)⁵⁰.

Rokowanie i wyniki leczenia

Rokowanie dla pacjentów z zespołem POEMS znacznie się poprawiło w ciągu ostatnich dziesięcioleci. Mediana przeżycia wynosi obecnie około 14 lat⁵¹⁵².

Większość pacjentów pozytywnie reaguje na leczenie, jednak poprawa neurologiczna może nie rozpocząć się przez 2 lata po leczeniu i może trwać przez lata⁵³. Wczesna diagnoza i odpowiednia terapia mogą zapobiec progresji i nieodwracalnemu wtórnemu zwyrodnieniu aksonalnemu⁵⁴.

Badanie porównujące trzy schematy leczenia pierwszej linii (ASCT, MDex i LDex) wykazało, że ASCT miało najwyższe wskaźniki odpowiedzi i przeżycia wolnego od progresji spośród trzech schematów, szczególnie u pacjentów o średnim i wysokim ryzyku. Jednak całkowite przeżycie nie było znacząco lepsze u pacjentów poddanych ASCT niż w innych grupach, prawdopodobnie z powodu śmiertelności związanej z leczeniem (1,2%) i długoterminowych powikłań, takich jak wtórne nowotwory (0,6%)⁵⁵.

Multidyscyplinarne podejście, w tym opieka wspomagająca i wsparcie emocjonalne, jest niezbędne ze względu na systemowy charakter choroby i częste znaczące ograniczenia fizyczne, jakie powoduje⁵⁶⁵⁷.

Podsumowanie strategie terapeutyczne

Główne podejścia terapeutyczne w zespole POEMS obejmują⁵⁸⁵⁹:

W przypadku choroby ograniczonej (bez zajęcia szpiku kostnego i z nie więcej niż trzema zmianami osteosklerotycznymi), radioterapia jest preferowaną metodą leczenia.
W przypadku zajęcia szpiku kostnego, jeśli pacjent kwalifikuje się do ASCT, należy rozważyć ASCT jako leczenie pierwszego rzutu.
Jeśli pacjent nie kwalifikuje się do ASCT z powodu kruchości lub dysfunkcji wielu narządów, lenalidomid z deksametazonem stanowi preferowany i dobrze tolerowany schemat leczenia.
Leczenie objawowe powinno być dostosowane do indywidualnych potrzeb pacjenta i może obejmować fizjoterapię, leczenie bólu, diuretyki i terapię hormonalną.

Ze względu na rzadkość występowania zespołu POEMS i brak randomizowanych badań klinicznych, zalecenia dotyczące leczenia opierają się głównie na seriach przypadków i doświadczeniu klinicznym. Konieczne są dalsze badania, aby ustalić optymalną strategię leczenia, szczególnie w przypadku nawrotów choroby⁶⁰⁶¹.

Kolejne rozdziały

Zapraszamy do dalszego czytania naszego leksykonu.

Wybierz kolejny rozdział z menu poniżej, aby otworzyć nową podstronę kompedium wiedzy i uzyskać szczegółowe informację o leku, substancji lub chorobie.

Materiały źródłowe

#1 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
POEMS is a rare clonal plasma cell disorder characterized by multi-systemic features that include demyelinating peripheral neuropathy, organomegaly, endocrinopathy, presence of monoclonal proteins (M-protein), and skin changes. […] Due to the rarity of the syndrome, clear guidelines for treatment are still lacking. Immediate treatment targeting the underlying plasma cell proliferation results in a dramatic response in most patients. The key is early diagnosis and immediate anti-plasma cell directed therapy for the best clinical outcomes. For patients with disseminated disease as defined by bone marrow involvement or more than three osteosclerotic bone lesions, high-dose chemotherapy with autologous hematopoietic stem cell transplant (ASCT) yields durable responses and is the preferred treatment in eligible patients. For patients with localized bony disease, radiotherapy has proven to be very effective. Lenalidomide and dexamethasone is a proven therapy in patients ineligible for ASCT.
#2 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
POEMS syndrome is a paraneoplastic syndrome whose acronym stands for less than half of the defining features of the disease, that is, polyradiculoneuropathy, organomegaly, potentially including coexisting Castleman disease, endocrinopathy, monoclonal plasma cell neoplasm, and skin changes. […] Prompt treatment directed at the underlying plasma cell clone produces dramatic responses in the majority of patients. […] Although there are no randomized clinical trial data to direct best therapy, for patients with disseminated disease, high-dose chemotherapy with peripheral blood transplantation has yielded durable benefit, whereas radiation therapy is typically effective for patients with a more localized presentation. […] Treatment of the POEMS syndrome can be broken down into 2 major categories: targeting the underlying clone and targeting the rest of the syndrome.
#3 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
The treatment of POEMS syndrome can be divided into two major categories: targeting the clone by specific anti-plasma cell directed therapy and targeting the symptoms with symptomatic and supportive care. To have a better overall outcome, physicians aim to incorporate both strategies in their treatment plan. […] If the patient is rapidly deteriorating, simultaneous use of corticosteroids, e.g., dexamethasone (40 mg/day 14 times a week for 2 weeks) or prednisone (1 mg/kg daily for two weeks) is a reasonable adjuvant therapy to radiotherapy. […] In cases of bone marrow involvement or more than three bone lesions, melphalan based conditioning followed by ASCT is considered the first line treatment by many; given the patient is able to tolerate the treatment based on functional status and adequate organs function.
#4 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
Due to the rarity of this disease, there is a lack of clear evidence-based guidelines for treatment. Clinicians use therapeutic strategies mostly based on retrospective case series of POEMS syndrome with proven efficacy in MM and systemic AL amyloidosis. […] The treatment of POEMS syndrome can be divided into two major categories: targeting the clone by specific anti-plasma cell directed therapy and targeting the symptoms with symptomatic and supportive care. To have a better overall outcome, physicians aim to incorporate both strategies in their treatment plan. […] The most efficacious strategy is taking the baseline parameters assessing them every visit or every other visit. […] If diagnostic imaging tests (CT scan, MRI, or PET scan) reveal 1â3 bone lesions with no bone marrow involvement then radiation therapy is the first line of therapy with excellent long-term outcomes.
#5 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
If there is no involvement of the bone marrow on iliac crest biopsy as documented with immunohistochemical stains, but only 1 to 3 bone lesions, radiation is the preferred strategy. […] For this first group, radiation doses of 40 Gy are most standard because the goal is potential cure rather than mere palliation. […] If a patient is rapidly deteriorating, simultaneous use of corticosteroids (eg, dexamethasone 40 mg days 1-4 every 2 weeks or daily prednisone at 1 mg/kg) is reasonable as adjuvant therapy. […] The sooner this plasma cell neoplasm is addressed with systemic chemotherapy, the better will be the recovery of the patient’s peripheral neuropathy. […] Corticosteroids may provide symptomatic improvement, but response duration is limited. […] The most experience has been with alkylator-based therapy, either high dose with peripheral blood stem cell transplant or low dose with corticosteroids.
#6 A Review of POEMS Syndrome
https://www.cancernetwork.com/view/review-poems-syndrome
POEMS syndrome is a rare paraneoplastic syndrome that is caused by an underlying plasma cell disorder. […] The most commonly used therapies for POEMS include alkylators and steroids, high-dose chemotherapy with peripheral blood stem cell transplantation, lenalidomide, and bortezomib. […] In general, patients have an excellent prognosis if the diagnosis is made early and appropriate therapy is applied. […] The treatment algorithm is based on the extent of the plasma cell infiltration. […] The course of POEMS syndrome is usually chronic, with a median patient survival time of nearly 14 years. […] In our experience and in a recent report by Li and colleagues, patients with co-existing Castleman disease may have an inferior overall survival compared to patients without it. […] In the case of patients with an isolated bone lesion without clonal plasma cells found on iliac crest biopsy, use of curative doses of radiation is the recommended therapy.
#7 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
If there is no involvement of the bone marrow on iliac crest biopsy as documented with immunohistochemical stains, but only 1 to 3 bone lesions, radiation is the preferred strategy. […] For this first group, radiation doses of 40 Gy are most standard because the goal is potential cure rather than mere palliation. […] If a patient is rapidly deteriorating, simultaneous use of corticosteroids (eg, dexamethasone 40 mg days 1-4 every 2 weeks or daily prednisone at 1 mg/kg) is reasonable as adjuvant therapy. […] The sooner this plasma cell neoplasm is addressed with systemic chemotherapy, the better will be the recovery of the patient’s peripheral neuropathy. […] Corticosteroids may provide symptomatic improvement, but response duration is limited. […] The most experience has been with alkylator-based therapy, either high dose with peripheral blood stem cell transplant or low dose with corticosteroids.
#8 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
For patients with POEMS and pulmonary hypertension, high-dose steroid treatment appears to provide improvement, as confirmed by sequential hemodynamic studies. […] Surgical excision of isolated plasmacytomas may result in complete resolution of the syndrome. […] Radiation treatment of solitary osteosclerotic lesions is first-line treatment for patients with an isolated plasmacytoma without bone marrow involvement. […] Radiation therapy in doses of 0.4-0.5 Gy to limited areas can improve osteosclerotic lesions in more than 50% of patients. […] Some authors have recommended systemic therapy for patients whose disease process fails to stabilize 3-6 months after the completion of radiation therapy.
#9 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
The treatment of POEMS syndrome can be divided into two major categories: targeting the clone by specific anti-plasma cell directed therapy and targeting the symptoms with symptomatic and supportive care. To have a better overall outcome, physicians aim to incorporate both strategies in their treatment plan. […] If the patient is rapidly deteriorating, simultaneous use of corticosteroids, e.g., dexamethasone (40 mg/day 14 times a week for 2 weeks) or prednisone (1 mg/kg daily for two weeks) is a reasonable adjuvant therapy to radiotherapy. […] In cases of bone marrow involvement or more than three bone lesions, melphalan based conditioning followed by ASCT is considered the first line treatment by many; given the patient is able to tolerate the treatment based on functional status and adequate organs function.
#10 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
The treatment of POEMS syndrome can be divided into two major categories: targeting the clone by specific anti-plasma cell directed therapy and targeting the symptoms with symptomatic and supportive care. To have a better overall outcome, physicians aim to incorporate both strategies in their treatment plan. […] If the patient is rapidly deteriorating, simultaneous use of corticosteroids, e.g., dexamethasone (40 mg/day 14 times a week for 2 weeks) or prednisone (1 mg/kg daily for two weeks) is a reasonable adjuvant therapy to radiotherapy. […] In cases of bone marrow involvement or more than three bone lesions, melphalan based conditioning followed by ASCT is considered the first line treatment by many; given the patient is able to tolerate the treatment based on functional status and adequate organs function.
#11 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
In cases of bone marrow involvement or more than three bone lesions, melphalan based conditioning followed by ASCT is considered the first line treatment by many; given the patient is able to tolerate the treatment based on functional status and adequate organs function. […] For patients undergoing ASCT, high dose melphalan (140â200 mg/m2) is used as conditioning regimen. […] The recommended treatment is lenalidomide (15â25 mg orally) taken days 1â21 every 28 days with weekly dexamethasone 20â40 mg. […] The mainstay of treatment is anti-plasma cell therapy such as IMiD or ASCT which will relieve polyneuropathy in most if not all of patients over times. […] The combination of treatment using cyclophosphamide, bortezomib, and dexamethasone (CyborD or VCD) alleviated the symptoms dramatically where the patient was back to normal weight, independent of diuretics.
#12 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
For those patients well enough to tolerate high-dose melphalan (140 mg/m2 to 200 mg/m2) as conditioning for autologous peripheral blood stem cell transplantation (ASCT), ASCT is my first choice based on our own experience and reports from others. […] Other promising treatments include lenalidomide, thalidomide, and bortezomib, drugs all of which can have a direct antiplasma cell effect as well as anti-VEGF and anti-TNF effects. […] With ASCT, responses are durable, but relapses have been reported. […] The progression-free survival was 98% and 75% at 1 and 5 years, respectively. […] Patients with POEMS typically have a higher than expected transfusion need, with median numbers of platelet and erythrocyte transfusions being 5 apheresis units and 6 units, respectively, and delayed neutrophil engraftment.
#13 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
A case report of a patient with POEMS syndrome and elevated vascular endothelial growth factor (VEGF) levels described successful treatment with bevacizumab, an antiangiogenesis monoclonal antibody directed against VEGF. […] Reported responses to bevacizumab have varied. […] In a case report, one patient improved after receiving nine cycles of lenalidomide given daily for 21 days of a 28-day cycle with once-weekly dexamethasone. […] A 2015 prospective phase II trial of lenalidomide combined with dexamethasone (LEN-DEX) reported a promising reduction of VEGF to normal levels and recovery of neurologic function in many patients, with minimal adverse effects. […] Several reports have described successful therapy with bortezomib, used either alone or (as in more recent studies) in conjunction with dexamethasone.
#14 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
The use of bevacizumab was appealing in many cases. […] However, due to lack of efficacy and potential for increasing mortality in some reports, bevacizumab should not be used in clinical practice for treatment of POEMS syndrome. […] A positive correlation was found between splenomegaly/lymphadenopathy and risk of ES. […] Overall, ASCT is a safe and effective choice in many patients and can provide durable response in about 90% of eligible patients. […] The follow up of patients should be completed at least every 3 months for at least several years.
#15 Treatment and outcomes of POEMS syndrome: changes in the past 20 years | Blood Cancer Journal
https://www.nature.com/articles/s41408-021-00540-1
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome is a rare plasma cell dyscrasia with multiorgan involvement. […] The melphalan plus dexamethasone regimen has been shown to have promising neurologic and organ responses, while high-dose melphalan followed by autologous stem cell transplantation (ASCT) makes it possible to achieve a deeper hematological response and longer survival. […] In recent years, new agents, including immunomodulatory drugs (IMiDs) and protease inhibitors (PIs), have also been increasingly applied in patients with POEMS syndrome, achieving similar treatment responses compared with ASCT with reduced side effects. […] A total of 220 patients (35.4%) received high-dose melphalan followed by ASCT as first-line therapy, and the other 401 received non-ASCT regimens.
#16 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
For those patients well enough to tolerate high-dose melphalan (140 mg/m2 to 200 mg/m2) as conditioning for autologous peripheral blood stem cell transplantation (ASCT), ASCT is my first choice based on our own experience and reports from others. […] Other promising treatments include lenalidomide, thalidomide, and bortezomib, drugs all of which can have a direct antiplasma cell effect as well as anti-VEGF and anti-TNF effects. […] With ASCT, responses are durable, but relapses have been reported. […] The progression-free survival was 98% and 75% at 1 and 5 years, respectively. […] Patients with POEMS typically have a higher than expected transfusion need, with median numbers of platelet and erythrocyte transfusions being 5 apheresis units and 6 units, respectively, and delayed neutrophil engraftment.
#17
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
We perform disease evaluation at six months or sooner if there are concerns about an inadequate response. […] A suggested treatment algorithm is outlined in Figure 1. […] POEMS may be driven by an isolated osteosclerotic bone lesion and in those cases without bone marrow involvement, involved-field radiotherapy (RT) can provide definitive disease control. […] Systemic disease (involvement of the bone marrow by a monoclonal plasma cell disorder) requires chemotherapy. […] Pre-ASCT induction has been shown to improve performance status, reduce risk of peri-ASCT complications, and hasten time to disease response. […] Bortezomib is an effective treatment in POEMS but concerns about neurotoxicity have limited its use historically. […] Up to 40% of patients have arterial and venous thrombosis at presentation or during active treatment which is likely due to underlying disease, patient immobility from neurological impairment as well as treatment including immunomodulatory drugs (IMiDs) and steroids. […] There is even fewer data to guide the optimal treatment strategy in the relapse setting. […] Future clinical trials are welcomed in this setting where evidence is limited.
#18 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
Given the anti-angiogenic effect of IMiD, such as thalidomide or lenalidomide, and their effectiveness in the treatment of MM, they were an option to consider in POEMS syndrome. […] The recommended treatment is lenalidomide (15-25 mg orally) taken days 1-21 every 28 days with weekly dexamethasone 20-40 mg. […] Melphalan combined with dexamethasone (MDex) is also used in different plasma cell disorders. […] Bortezomib, a proteasome inhibitor, inhibits the secretion of VEGF and IL-6, stops angiogenesis, and induces B cell apoptosis in a dose-dependent manner. […] In patients with POEMS syndrome, the response to daratumumab and lenalidomide was remarkable as well based on case reports. This suggests that this combination is a promising less toxic alternative to ASCT that warrants further exploration in both newly diagnosed and relapsed/refractory patients.
#19 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
The mainstay of treatment is radiotherapy when there is no bone marrow involvement, and when the disease is limited to fewer than three osteoclerotic bone lesions. When bone marrow involvement is observed, and if the patient is eligible for ASCT, upfront ASCT should be considered. If ASCT is not desired by the patient or they are not eligible due to frailty or multiple organ dysfunction, lenalidomide and dexamethasone is a preferred and well-tolerated regimen.
#20 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
Given the anti-angiogenic effect of IMiD, such as thalidomide or lenalidomide, and their effectiveness in the treatment of MM, they were an option to consider in POEMS syndrome. […] The recommended treatment is lenalidomide (15-25 mg orally) taken days 1-21 every 28 days with weekly dexamethasone 20-40 mg. […] Melphalan combined with dexamethasone (MDex) is also used in different plasma cell disorders. […] Bortezomib, a proteasome inhibitor, inhibits the secretion of VEGF and IL-6, stops angiogenesis, and induces B cell apoptosis in a dose-dependent manner. […] In patients with POEMS syndrome, the response to daratumumab and lenalidomide was remarkable as well based on case reports. This suggests that this combination is a promising less toxic alternative to ASCT that warrants further exploration in both newly diagnosed and relapsed/refractory patients.
#21 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
A case report of a patient with POEMS syndrome and elevated vascular endothelial growth factor (VEGF) levels described successful treatment with bevacizumab, an antiangiogenesis monoclonal antibody directed against VEGF. […] Reported responses to bevacizumab have varied. […] In a case report, one patient improved after receiving nine cycles of lenalidomide given daily for 21 days of a 28-day cycle with once-weekly dexamethasone. […] A 2015 prospective phase II trial of lenalidomide combined with dexamethasone (LEN-DEX) reported a promising reduction of VEGF to normal levels and recovery of neurologic function in many patients, with minimal adverse effects. […] Several reports have described successful therapy with bortezomib, used either alone or (as in more recent studies) in conjunction with dexamethasone.
#22 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
In cases of bone marrow involvement or more than three bone lesions, melphalan based conditioning followed by ASCT is considered the first line treatment by many; given the patient is able to tolerate the treatment based on functional status and adequate organs function. […] For patients undergoing ASCT, high dose melphalan (140â200 mg/m2) is used as conditioning regimen. […] The recommended treatment is lenalidomide (15â25 mg orally) taken days 1â21 every 28 days with weekly dexamethasone 20â40 mg. […] The mainstay of treatment is anti-plasma cell therapy such as IMiD or ASCT which will relieve polyneuropathy in most if not all of patients over times. […] The combination of treatment using cyclophosphamide, bortezomib, and dexamethasone (CyborD or VCD) alleviated the symptoms dramatically where the patient was back to normal weight, independent of diuretics.
#23 What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone? | Leukemia
https://www.nature.com/articles/s41375-019-0391-2
POEMS syndrome is a rare plasma cell dyscrasia. This study compared the responses to and survival of 347 POEMS syndrome patients given three first-line treatment regimens: autologous stem cell transplantation (ASCT, N=165) and melphalan+dexamethasone (MDex, N=79), or lenalidomide+dexamethasone (LDex, N=103). […] High-dose chemotherapy with ASCT can achieve excellent clinical responses, with a significant improvement of neuropathy, complete hematologic response (CRH) in 60% of POEMS syndrome patients, and complete VEGF response (CRV) in 50%. […] However, treatment-related complications are frequent, with peri-engraftment syndrome reported in 12-50% of patients. […] MDex therapy included melphalan 10mg/m2+dexamethasone 40mg on days 1-4, one cycle every 28 days for a total of 9 cycles. […] Lenalidomide plus dexamethasone (LDex) achieved an RN of over 95%, a CRH of 46.3%, a CRV of 42.5%, a 3-year PFS of 75%, and an OS of 90% in a phase II single-arm prospective study including 41 patients.
#24 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
Given the anti-angiogenic effect of IMiD, such as thalidomide or lenalidomide, and their effectiveness in the treatment of MM, they were an option to consider in POEMS syndrome. […] The recommended treatment is lenalidomide (15-25 mg orally) taken days 1-21 every 28 days with weekly dexamethasone 20-40 mg. […] Melphalan combined with dexamethasone (MDex) is also used in different plasma cell disorders. […] Bortezomib, a proteasome inhibitor, inhibits the secretion of VEGF and IL-6, stops angiogenesis, and induces B cell apoptosis in a dose-dependent manner. […] In patients with POEMS syndrome, the response to daratumumab and lenalidomide was remarkable as well based on case reports. This suggests that this combination is a promising less toxic alternative to ASCT that warrants further exploration in both newly diagnosed and relapsed/refractory patients.
#25
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
We perform disease evaluation at six months or sooner if there are concerns about an inadequate response. […] A suggested treatment algorithm is outlined in Figure 1. […] POEMS may be driven by an isolated osteosclerotic bone lesion and in those cases without bone marrow involvement, involved-field radiotherapy (RT) can provide definitive disease control. […] Systemic disease (involvement of the bone marrow by a monoclonal plasma cell disorder) requires chemotherapy. […] Pre-ASCT induction has been shown to improve performance status, reduce risk of peri-ASCT complications, and hasten time to disease response. […] Bortezomib is an effective treatment in POEMS but concerns about neurotoxicity have limited its use historically. […] Up to 40% of patients have arterial and venous thrombosis at presentation or during active treatment which is likely due to underlying disease, patient immobility from neurological impairment as well as treatment including immunomodulatory drugs (IMiDs) and steroids. […] There is even fewer data to guide the optimal treatment strategy in the relapse setting. […] Future clinical trials are welcomed in this setting where evidence is limited.
#26 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
Given the anti-angiogenic effect of IMiD, such as thalidomide or lenalidomide, and their effectiveness in the treatment of MM, they were an option to consider in POEMS syndrome. […] The recommended treatment is lenalidomide (15-25 mg orally) taken days 1-21 every 28 days with weekly dexamethasone 20-40 mg. […] Melphalan combined with dexamethasone (MDex) is also used in different plasma cell disorders. […] Bortezomib, a proteasome inhibitor, inhibits the secretion of VEGF and IL-6, stops angiogenesis, and induces B cell apoptosis in a dose-dependent manner. […] In patients with POEMS syndrome, the response to daratumumab and lenalidomide was remarkable as well based on case reports. This suggests that this combination is a promising less toxic alternative to ASCT that warrants further exploration in both newly diagnosed and relapsed/refractory patients.
#27 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
The use of bevacizumab was appealing in many cases. […] However, due to lack of efficacy and potential for increasing mortality in some reports, bevacizumab should not be used in clinical practice for treatment of POEMS syndrome. […] A positive correlation was found between splenomegaly/lymphadenopathy and risk of ES. […] Overall, ASCT is a safe and effective choice in many patients and can provide durable response in about 90% of eligible patients. […] The follow up of patients should be completed at least every 3 months for at least several years.
#28 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
For patients with POEMS and pulmonary hypertension, high-dose steroid treatment appears to provide improvement, as confirmed by sequential hemodynamic studies. […] Surgical excision of isolated plasmacytomas may result in complete resolution of the syndrome. […] Radiation treatment of solitary osteosclerotic lesions is first-line treatment for patients with an isolated plasmacytoma without bone marrow involvement. […] Radiation therapy in doses of 0.4-0.5 Gy to limited areas can improve osteosclerotic lesions in more than 50% of patients. […] Some authors have recommended systemic therapy for patients whose disease process fails to stabilize 3-6 months after the completion of radiation therapy.
#29 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
For patients with POEMS and pulmonary hypertension, high-dose steroid treatment appears to provide improvement, as confirmed by sequential hemodynamic studies. […] Surgical excision of isolated plasmacytomas may result in complete resolution of the syndrome. […] Radiation treatment of solitary osteosclerotic lesions is first-line treatment for patients with an isolated plasmacytoma without bone marrow involvement. […] Radiation therapy in doses of 0.4-0.5 Gy to limited areas can improve osteosclerotic lesions in more than 50% of patients. […] Some authors have recommended systemic therapy for patients whose disease process fails to stabilize 3-6 months after the completion of radiation therapy.
#30 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
Although an anti-VEGF strategy is theoretically appealing, the results with bevacizumab have been mixed. […] A recent report, however, describes reduction in serum VEGF and clinical improvement with single-agent IVIG. […] The 2 best ways to approach the peripheral neuropathy are to target the clone and to direct the patient to work intensively with physical therapy and occupational therapy. […] I treat the painful peripheral neuropathy if present with drugs, such as gabapentin, pregabalin, amitriptyline, nortriptyline, duloxetine, topical lidocaine patches, and topical ketamine, lidocaine, and amitriptyline compounds. […] Signs of volume overload are present in the majority of patients in the form of peripheral edema; however, ascites, pleural effusions, and pericardial effusions may be present in as many as 50% of patients depending on the series.
#31 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
Although an anti-VEGF strategy is theoretically appealing, the results with bevacizumab have been mixed. […] A recent report, however, describes reduction in serum VEGF and clinical improvement with single-agent IVIG. […] The 2 best ways to approach the peripheral neuropathy are to target the clone and to direct the patient to work intensively with physical therapy and occupational therapy. […] I treat the painful peripheral neuropathy if present with drugs, such as gabapentin, pregabalin, amitriptyline, nortriptyline, duloxetine, topical lidocaine patches, and topical ketamine, lidocaine, and amitriptyline compounds. […] Signs of volume overload are present in the majority of patients in the form of peripheral edema; however, ascites, pleural effusions, and pericardial effusions may be present in as many as 50% of patients depending on the series.
#32 POEMS Syndrome: What It Is, Symptoms & Treatment
https://my.clevelandclinic.org/health/diseases/17985-poems-syndrome
Physical therapy: This treatment may help you with mobility issues you may be experiencing because of polyneuropathy. […] Your healthcare provider may also prescribe steroids and diuretics (to relieve swelling) to ease symptoms. […] Effective treatment can stop or slow nerve damage and also help ease other symptoms. Nerve damage may slowly improve over time.
#33
https://link.springer.com/article/10.1007/s00415-018-9068-4
Neurorehabilitation is essential in patients with sensory and motor impairment. […] Pain may be a dominant feature in patients with POEMS and of course should be treated pharmacologically with drugs such as gabapentin and tricyclic antidepressants. […] Early diagnosis and therapy can prevent progression and irreversible secondary axonal degeneration.
#34 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
Although an anti-VEGF strategy is theoretically appealing, the results with bevacizumab have been mixed. […] A recent report, however, describes reduction in serum VEGF and clinical improvement with single-agent IVIG. […] The 2 best ways to approach the peripheral neuropathy are to target the clone and to direct the patient to work intensively with physical therapy and occupational therapy. […] I treat the painful peripheral neuropathy if present with drugs, such as gabapentin, pregabalin, amitriptyline, nortriptyline, duloxetine, topical lidocaine patches, and topical ketamine, lidocaine, and amitriptyline compounds. […] Signs of volume overload are present in the majority of patients in the form of peripheral edema; however, ascites, pleural effusions, and pericardial effusions may be present in as many as 50% of patients depending on the series.
#35 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
I have found that the use of serial paracentesis and albumin-forced diuresis may provide benefit, but results can also be disappointing. […] The mechanism of this feature of the syndrome is not well understood, but it has been speculated that in part VEGF contributes to the capillary leak. […] The hepatosplenomegaly and lymphadenopathy do not require specific therapy. […] Endocrinopathy is a central but poorly understood feature of POEMS. […] In my experience, endocrine abnormalities can improve after chemotherapy, including successful tapering off of thyroid replacement, androgen replacement, and corticosteroid replacement in at least one-third of the patients. […] Papilledema (optic disc edema) is present in at least one-third of patients and may be associated with increased intracranial pressure.
#36 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
In most cases, the optic disc edema is asymptomatic, but when it is not and when pressures are high, treatment with acetazolamide and corticosteroids may control symptoms until definitive chemotherapy or radiation therapy directed at the underlying clone can control the disease. […] Osteosclerotic lesions occur in approximately 95% of patients and can be confused with benign bone islands, aneurysmal bone cysts, nonossifying fibromas, and fibrous dysplasia. […] With the exception of calciphylaxis, none of the skin changes requires any specific therapy, and they all gradually improve after definitive therapy. […] Approximately 50% of patients with POEMS have thrombocytosis. […] I will use hydroxyurea in those patients with significant thrombocytosis who presented with a cerebral event until I can start therapy directed at the underlying clone. […] The best choice of therapy has not been derived through clinical trials, but rather through case series, and ASCT has become a favored therapy.
#37 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
In most cases, the optic disc edema is asymptomatic, but when it is not and when pressures are high, treatment with acetazolamide and corticosteroids may control symptoms until definitive chemotherapy or radiation therapy directed at the underlying clone can control the disease. […] Osteosclerotic lesions occur in approximately 95% of patients and can be confused with benign bone islands, aneurysmal bone cysts, nonossifying fibromas, and fibrous dysplasia. […] With the exception of calciphylaxis, none of the skin changes requires any specific therapy, and they all gradually improve after definitive therapy. […] Approximately 50% of patients with POEMS have thrombocytosis. […] I will use hydroxyurea in those patients with significant thrombocytosis who presented with a cerebral event until I can start therapy directed at the underlying clone. […] The best choice of therapy has not been derived through clinical trials, but rather through case series, and ASCT has become a favored therapy.
#38 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
The use of bevacizumab was appealing in many cases. […] However, due to lack of efficacy and potential for increasing mortality in some reports, bevacizumab should not be used in clinical practice for treatment of POEMS syndrome. […] A positive correlation was found between splenomegaly/lymphadenopathy and risk of ES. […] Overall, ASCT is a safe and effective choice in many patients and can provide durable response in about 90% of eligible patients. […] The follow up of patients should be completed at least every 3 months for at least several years.
#39
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
We perform disease evaluation at six months or sooner if there are concerns about an inadequate response. […] A suggested treatment algorithm is outlined in Figure 1. […] POEMS may be driven by an isolated osteosclerotic bone lesion and in those cases without bone marrow involvement, involved-field radiotherapy (RT) can provide definitive disease control. […] Systemic disease (involvement of the bone marrow by a monoclonal plasma cell disorder) requires chemotherapy. […] Pre-ASCT induction has been shown to improve performance status, reduce risk of peri-ASCT complications, and hasten time to disease response. […] Bortezomib is an effective treatment in POEMS but concerns about neurotoxicity have limited its use historically. […] Up to 40% of patients have arterial and venous thrombosis at presentation or during active treatment which is likely due to underlying disease, patient immobility from neurological impairment as well as treatment including immunomodulatory drugs (IMiDs) and steroids. […] There is even fewer data to guide the optimal treatment strategy in the relapse setting. […] Future clinical trials are welcomed in this setting where evidence is limited.
#40 Ocular Manifestations of Polyneuropathy, Organomegaly, Endocrinopathy, Myeloma Protein, and Skin Changes (POEMS) Syndrome – EyeWiki
https://eyewiki.org/Ocular_Manifestations_of_Polyneuropathy,_Organomegaly,_Endocrinopathy,_Myeloma_Protein,_and_Skin_Changes_(POEMS)_Syndrome
The treatment for POEMS syndrome is collaborative with hematology and oncology. Most strategies are based on retrospective case studies and lack clear evidence based guidelines. Treatments should target both the symptomatic manifestations and the clonal plasma cell origin with anti-plasma cell directed therapy. Differentiating patients with POEMS and concomitant Castleman Disease (POEMS-CD) is useful as POEMS-CD patients respond better to thalidomide plus dexamethasone. […] Generally, the first step in therapy is to determine the level of systemic involvement using systemic markers, including symptoms and bone health. Studies have shown that optic disc edema, VEGF, and IL-12 levels positively relate to disease activity and may be useful in monitoring the disease. For non-metastatic disease, radiotherapy is first-line with or without corticosteroids for systemic control. For those determined to have more systemic disease, a combination of chemotherapy and autologous stem cell transplantation has been considered. Most drug agents are either alkylating agents, such as melphalan or cyclophosphamide, or anti-cytokine agents, such as thalidomide and bevacizumab. Melphalan plus dexamethasone combination has improved neurological symptoms, however with the challenge related to treating young patients due to stem cell toxicity and no clear long-term results. Autologous hematopoietic stem cell transplantation (ASCT) showed promising hematologic response and long-term survival. […] In the treatment of the patients papilledema, the first line therapy is to address the underlying cause in POEMS. Standard medical and surgical treatments of papilledema may be necessary however.
#41
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
The aim of treatment is to eradicate the underlying plasma cell dyscrasia, but there are limited trial data to guide treatment decisions. […] Effective management requires targeting the underlying plasma cell clone in parallel with proactive management of comorbid risks and POEMS-induced physiological impairments. […] The aim of this framework is to provide clinicians a blueprint for managing POEMS from the moment of diagnostic suspicion through the work-up, selection of therapy, follow-up, and treatment of relapse. […] Most patients with POEMS syndrome respond favorably to treatment, but neurological improvement may not commence for 2 years after treatment and may continue for years. […] The goal of treatment in POEMS syndrome is to suppress the underlying plasma cell clone, but although in some cases it is possible, complete disease suppression (CRH) is not always achievable particularly in frail patients.
#42 Ocular Manifestations of Polyneuropathy, Organomegaly, Endocrinopathy, Myeloma Protein, and Skin Changes (POEMS) Syndrome – EyeWiki
https://eyewiki.org/Ocular_Manifestations_of_Polyneuropathy,_Organomegaly,_Endocrinopathy,_Myeloma_Protein,_and_Skin_Changes_(POEMS)_Syndrome
The treatment for POEMS syndrome is collaborative with hematology and oncology. Most strategies are based on retrospective case studies and lack clear evidence based guidelines. Treatments should target both the symptomatic manifestations and the clonal plasma cell origin with anti-plasma cell directed therapy. Differentiating patients with POEMS and concomitant Castleman Disease (POEMS-CD) is useful as POEMS-CD patients respond better to thalidomide plus dexamethasone. […] Generally, the first step in therapy is to determine the level of systemic involvement using systemic markers, including symptoms and bone health. Studies have shown that optic disc edema, VEGF, and IL-12 levels positively relate to disease activity and may be useful in monitoring the disease. For non-metastatic disease, radiotherapy is first-line with or without corticosteroids for systemic control. For those determined to have more systemic disease, a combination of chemotherapy and autologous stem cell transplantation has been considered. Most drug agents are either alkylating agents, such as melphalan or cyclophosphamide, or anti-cytokine agents, such as thalidomide and bevacizumab. Melphalan plus dexamethasone combination has improved neurological symptoms, however with the challenge related to treating young patients due to stem cell toxicity and no clear long-term results. Autologous hematopoietic stem cell transplantation (ASCT) showed promising hematologic response and long-term survival. […] In the treatment of the patients papilledema, the first line therapy is to address the underlying cause in POEMS. Standard medical and surgical treatments of papilledema may be necessary however.
#43 A Review of POEMS Syndrome
https://www.cancernetwork.com/view/review-poems-syndrome
POEMS syndrome is a rare paraneoplastic syndrome that is caused by an underlying plasma cell disorder. […] The most commonly used therapies for POEMS include alkylators and steroids, high-dose chemotherapy with peripheral blood stem cell transplantation, lenalidomide, and bortezomib. […] In general, patients have an excellent prognosis if the diagnosis is made early and appropriate therapy is applied. […] The treatment algorithm is based on the extent of the plasma cell infiltration. […] The course of POEMS syndrome is usually chronic, with a median patient survival time of nearly 14 years. […] In our experience and in a recent report by Li and colleagues, patients with co-existing Castleman disease may have an inferior overall survival compared to patients without it. […] In the case of patients with an isolated bone lesion without clonal plasma cells found on iliac crest biopsy, use of curative doses of radiation is the recommended therapy.
#44 POEMS Syndrome Treatment & Management: Medical Care, Surgical Care, Consultations
https://emedicine.medscape.com/article/1097031-treatment
For patients with POEMS and pulmonary hypertension, high-dose steroid treatment appears to provide improvement, as confirmed by sequential hemodynamic studies. […] Surgical excision of isolated plasmacytomas may result in complete resolution of the syndrome. […] Radiation treatment of solitary osteosclerotic lesions is first-line treatment for patients with an isolated plasmacytoma without bone marrow involvement. […] Radiation therapy in doses of 0.4-0.5 Gy to limited areas can improve osteosclerotic lesions in more than 50% of patients. […] Some authors have recommended systemic therapy for patients whose disease process fails to stabilize 3-6 months after the completion of radiation therapy.
#45
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
We perform disease evaluation at six months or sooner if there are concerns about an inadequate response. […] A suggested treatment algorithm is outlined in Figure 1. […] POEMS may be driven by an isolated osteosclerotic bone lesion and in those cases without bone marrow involvement, involved-field radiotherapy (RT) can provide definitive disease control. […] Systemic disease (involvement of the bone marrow by a monoclonal plasma cell disorder) requires chemotherapy. […] Pre-ASCT induction has been shown to improve performance status, reduce risk of peri-ASCT complications, and hasten time to disease response. […] Bortezomib is an effective treatment in POEMS but concerns about neurotoxicity have limited its use historically. […] Up to 40% of patients have arterial and venous thrombosis at presentation or during active treatment which is likely due to underlying disease, patient immobility from neurological impairment as well as treatment including immunomodulatory drugs (IMiDs) and steroids. […] There is even fewer data to guide the optimal treatment strategy in the relapse setting. […] Future clinical trials are welcomed in this setting where evidence is limited.
#46
https://link.springer.com/article/10.1007/s00415-018-9068-4
POEMS syndrome is a rare, chronic, disabling paraneoplastic disorder characterized by peripheral neuropathy, organomegaly, endocrinopathy, monoclonal plasma cells disorder and skin changes. […] Treatment may be either local or systemic and is aimed at the monoclonal plasma cell disorder. […] Lenalidomide, a promising therapy for POEMS syndrome, appears to be effective particularly on oedema and peripheral neuropathy, probably due to its anti-VEGF effect. […] High-dose chemotherapy-conditioned autologous stem cell transplantation is the current gold standard treatment for POEMS syndrome, showing good hematological control, neurological response, with improvement of the neuropathy, and good survival. […] Potential new therapeutic approaches involve targeting key factors in the pathogenesis of POEMS, including VEGF, IL-6, IL-12 and TNF.
#47
https://link.springer.com/article/10.1007/s00415-018-9068-4
POEMS syndrome is a rare, chronic, disabling paraneoplastic disorder characterized by peripheral neuropathy, organomegaly, endocrinopathy, monoclonal plasma cells disorder and skin changes. […] Treatment may be either local or systemic and is aimed at the monoclonal plasma cell disorder. […] Lenalidomide, a promising therapy for POEMS syndrome, appears to be effective particularly on oedema and peripheral neuropathy, probably due to its anti-VEGF effect. […] High-dose chemotherapy-conditioned autologous stem cell transplantation is the current gold standard treatment for POEMS syndrome, showing good hematological control, neurological response, with improvement of the neuropathy, and good survival. […] Potential new therapeutic approaches involve targeting key factors in the pathogenesis of POEMS, including VEGF, IL-6, IL-12 and TNF.
#48 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
Although an anti-VEGF strategy is theoretically appealing, the results with bevacizumab have been mixed. […] A recent report, however, describes reduction in serum VEGF and clinical improvement with single-agent IVIG. […] The 2 best ways to approach the peripheral neuropathy are to target the clone and to direct the patient to work intensively with physical therapy and occupational therapy. […] I treat the painful peripheral neuropathy if present with drugs, such as gabapentin, pregabalin, amitriptyline, nortriptyline, duloxetine, topical lidocaine patches, and topical ketamine, lidocaine, and amitriptyline compounds. […] Signs of volume overload are present in the majority of patients in the form of peripheral edema; however, ascites, pleural effusions, and pericardial effusions may be present in as many as 50% of patients depending on the series.
#49 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://www.mdpi.com/2077-0383/11/23/7011
The use of bevacizumab was appealing in many cases. […] However, due to lack of efficacy and potential for increasing mortality in some reports, bevacizumab should not be used in clinical practice for treatment of POEMS syndrome. […] A positive correlation was found between splenomegaly/lymphadenopathy and risk of ES. […] Overall, ASCT is a safe and effective choice in many patients and can provide durable response in about 90% of eligible patients. […] The follow up of patients should be completed at least every 3 months for at least several years.
#50 Anti-BCMA CAR-T cells for treatment of plasma cell dyscrasia: case report on POEMS syndrome and multiple myeloma | Journal of Hematology & Oncology | Full Text
https://jhoonline.biomedcentral.com/articles/10.1186/s13045-018-0672-7
POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome still has no standard treatment. […] On the basis that both POEMS syndrome and myeloma have an underlying plasma cell dyscrasia, anti-myeloma therapy can be expected to be useful for POEMS syndrome. […] This is the first report of treatment by anti-BCMA CAR-T cells in POEMS syndrome. Our findings demonstrate the anti-BCMA CAR-T cell treatment may be a feasible therapeutic option for patients with POEMS syndrome and RRMM who do not respond well to traditional therapies. […] Our work demonstrated that anti-BCMA CAR-T cell treatment has a significant effect in patients with plasma cell dyscrasia, i.e., POEMS syndrome and RRMM. […] The patient had a dramatic improvement in neuropathy and functional status. […] The anti-BCMA CAR-T cell treatment may be a feasible therapeutic option for patients with POEMS syndrome and RRMM who do not respond well to traditional therapies.
#51 A Review of POEMS Syndrome
https://www.cancernetwork.com/view/review-poems-syndrome
POEMS syndrome is a rare paraneoplastic syndrome that is caused by an underlying plasma cell disorder. […] The most commonly used therapies for POEMS include alkylators and steroids, high-dose chemotherapy with peripheral blood stem cell transplantation, lenalidomide, and bortezomib. […] In general, patients have an excellent prognosis if the diagnosis is made early and appropriate therapy is applied. […] The treatment algorithm is based on the extent of the plasma cell infiltration. […] The course of POEMS syndrome is usually chronic, with a median patient survival time of nearly 14 years. […] In our experience and in a recent report by Li and colleagues, patients with co-existing Castleman disease may have an inferior overall survival compared to patients without it. […] In the case of patients with an isolated bone lesion without clonal plasma cells found on iliac crest biopsy, use of curative doses of radiation is the recommended therapy.
#52 POEMS Syndrome and Multiple Myeloma
https://www.webmd.com/cancer/multiple-myeloma/what-is-poems-syndrome
What Are the Treatments for POEMS Syndrome? Treatments for POEMS syndrome keep symptoms at bay, but they wont cure the condition. Some treatments doctors might recommend include: Chemotherapy: It uses drugs to kill bad plasma cells. Immunotherapy: This treatment uses your bodys immune system to target abnormal plasma cells. Radiation therapy: High doses of radiation destroy the problem plasma cells. Other medicines: Steroids or diuretics may be given to relieve swelling or other symptoms. Physical therapy (PT): PT is sometimes used to help with mobility problems or weakness issues that are caused by neuropathy. Stem cell transplant: With this procedure, high doses of chemo are given followed by an infusion of healthy blood stem cells from your own body. […] The outlook for POEMS syndrome varies from person to person. Yours might depend on how severe your disease is and what body parts are affected. Treatments to kill faulty plasma cells can help you live longer and improve your quality of life. The median survival for people with POEMS syndrome is more than 14 years. The outlook has improved a lot over the past 10 years and is likely to get even better with future medical advances.
#53
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
The aim of treatment is to eradicate the underlying plasma cell dyscrasia, but there are limited trial data to guide treatment decisions. […] Effective management requires targeting the underlying plasma cell clone in parallel with proactive management of comorbid risks and POEMS-induced physiological impairments. […] The aim of this framework is to provide clinicians a blueprint for managing POEMS from the moment of diagnostic suspicion through the work-up, selection of therapy, follow-up, and treatment of relapse. […] Most patients with POEMS syndrome respond favorably to treatment, but neurological improvement may not commence for 2 years after treatment and may continue for years. […] The goal of treatment in POEMS syndrome is to suppress the underlying plasma cell clone, but although in some cases it is possible, complete disease suppression (CRH) is not always achievable particularly in frail patients.
#54
https://link.springer.com/article/10.1007/s00415-018-9068-4
Neurorehabilitation is essential in patients with sensory and motor impairment. […] Pain may be a dominant feature in patients with POEMS and of course should be treated pharmacologically with drugs such as gabapentin and tricyclic antidepressants. […] Early diagnosis and therapy can prevent progression and irreversible secondary axonal degeneration.
#55 What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone? | Leukemia
https://www.nature.com/articles/s41375-019-0391-2
The efficacy of these treatments has not previously been compared in large patient cohorts, but the rarity of the disease makes it difficult to conduct randomized controlled trials. […] In this patient series, ASCT had the highest response and PFS rates of the three regimens, especially for patients at medium-to-high risk. […] However, OS was not significantly better in ASCT patients than in the other groups, possibly because of treatment-related mortality (1.2%), and long-term complications, such as secondary malignancy (0.6%). […] MDex and LDex regimens have both been shown effective in POEMS syndrome patients not eligible for ASCT, but it is not clear that they are less toxic alternatives to ASCT as first-line POEMS therapy. […] Lenalidomide was highly active in these newly diagnosed POEMS syndrome patients, with response rates and OS similar to those with ASCT. […] In conclusion, all three treatments had reasonable responses and survivals in newly diagnosed POEMS syndrome patients. However, ASCT might have better responses and survivals compared with the other two groups, especially for higher risk patients.
#56 POEMS syndrome | MedLink Neurology
https://www.medlink.com/articles/poems-syndrome
Systemic therapy targeting the underlying plasma cell clone is mandatory if the patient has bone marrow involvement by clonal plasma cells or more than three bone lesions. […] The first therapeutic approach to show dramatic responses in this disease was high-dose melphalan with autologous stem cell transplant (ASCT). […] An update of this series with 59 patients who received an ASCT at the Mayo Clinic and a median follow-up of 45 months, showed a hematologic response rate of 78% (including 57% complete responses) and clinical improvement in 92% of patients. […] For those patients who are not candidates for radiation therapy nor for high-dose chemotherapy and ASCT, there is no consensus on the best treatment option. […] The anti-CD38 monoclonal antibody daratumumab has been used in POEMS syndrome both alone and in combination, either with bortezomib-dexamethasone, lenalidomide-dexamethasone, or carfilzomib and pomalidomide. […] A multidisciplinary approach including supportive care and emotional support is essential given the systemic nature of the disease and the common significant physical limitations it entails.
#57 POEMS Syndrome | Condition | UAMS Health
https://uamshealth.com/condition/poems-syndrome/
Extravascular volume overload Diuretics may be used to decrease the amount of excess fluid and swelling […] Endocrine abnormalities Hormone replacement may be appropriate for the treatment of hypothyroidism (underactive thyroid) and other hormone-related symptoms. […] The effectiveness of treatment is monitored on a regular basis. […] POEMS syndrome can be effectively treated and significant improvement in overall functioning and quality of life can be expected. Autologous Stem Cell Transplantation (high-dose chemotherapy with stem cell rescue), the treatment of choice for transplant eligible patients, often results in dramatic improvement. […] A multi-disciplinary approach to the treatment of POEMS syndrome is essential. The physician team should include myeloma specialists, radiologists, neurologists, nephrologists, cardiologists and other specialists as needed.
#58 Recent Advances in the Treatment and Supportive Care of POEMS Syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC9741379/
The mainstay of treatment is radiotherapy when there is no bone marrow involvement, and when the disease is limited to fewer than three osteoclerotic bone lesions. When bone marrow involvement is observed, and if the patient is eligible for ASCT, upfront ASCT should be considered. If ASCT is not desired by the patient or they are not eligible due to frailty or multiple organ dysfunction, lenalidomide and dexamethasone is a preferred and well-tolerated regimen.
#59 How I treat POEMS syndrome
https://pmc.ncbi.nlm.nih.gov/articles/PMC3425020/
POEMS syndrome is a paraneoplastic syndrome whose acronym stands for less than half of the defining features of the disease, that is, polyradiculoneuropathy, organomegaly, potentially including coexisting Castleman disease, endocrinopathy, monoclonal plasma cell neoplasm, and skin changes. […] Prompt treatment directed at the underlying plasma cell clone produces dramatic responses in the majority of patients. […] Although there are no randomized clinical trial data to direct best therapy, for patients with disseminated disease, high-dose chemotherapy with peripheral blood transplantation has yielded durable benefit, whereas radiation therapy is typically effective for patients with a more localized presentation. […] Treatment of the POEMS syndrome can be broken down into 2 major categories: targeting the underlying clone and targeting the rest of the syndrome.
#60
https://journals.lww.com/hemasphere/fulltext/2022/11000/comprehensive_diagnosis_and_management_of_poems.12.aspx
We perform disease evaluation at six months or sooner if there are concerns about an inadequate response. […] A suggested treatment algorithm is outlined in Figure 1. […] POEMS may be driven by an isolated osteosclerotic bone lesion and in those cases without bone marrow involvement, involved-field radiotherapy (RT) can provide definitive disease control. […] Systemic disease (involvement of the bone marrow by a monoclonal plasma cell disorder) requires chemotherapy. […] Pre-ASCT induction has been shown to improve performance status, reduce risk of peri-ASCT complications, and hasten time to disease response. […] Bortezomib is an effective treatment in POEMS but concerns about neurotoxicity have limited its use historically. […] Up to 40% of patients have arterial and venous thrombosis at presentation or during active treatment which is likely due to underlying disease, patient immobility from neurological impairment as well as treatment including immunomodulatory drugs (IMiDs) and steroids. […] There is even fewer data to guide the optimal treatment strategy in the relapse setting. […] Future clinical trials are welcomed in this setting where evidence is limited.
#61 Treatment for POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) | Cochrane
https://www.cochrane.org/CD006828/NEUROMUSC_treatment-for-poems-syndrome-polyneuropathy-organomegaly-endocrinopathy-m-protein-and-skin-changes
There are no randomized or quasi-randomized controlled clinical trials of treatment for POEMS syndrome on which to base practice. […] There is a need for efficacious therapy to improve prognosis. […] We found no randomized or non-randomized prospective controlled trials of treatment for POEMS syndrome.