Materiały źródłowe

• #1 Aplastic anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/aplastic-anemia/diagnosis-treatment/drc-20355020

  Treatments for aplastic anemia depend on the symptoms and your age and overall health. Treatment options might include observation, blood transfusions, medicines or bone marrow transplantation. Severe aplastic anemia, in which your blood cell counts are extremely low, is life-threatening and requires immediate hospitalization. […] Although not a cure for aplastic anemia, blood transfusions can provide blood cells your bone marrow isn’t producing. This can help control bleeding and relieve symptoms. […] A stem cell transplant to rebuild the bone marrow with stem cells from a donor might be the only successful treatment option for people with severe aplastic anemia. […] For people who can’t undergo a bone marrow transplant or for those whose aplastic anemia is due to an autoimmune disorder, treatment can involve drugs that alter or suppress the immune system, called immunosuppressants.

• #2 Aplastic Anemia: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/16747-aplastic-anemia

  Aplastic anemia is a rare but serious blood disorder. It happens when something damages your bone marrow so it cant make enough new blood cells and platelets. Left untreated, aplastic anemia is a life-threatening condition. Healthcare providers can treat aplastic anemia, but a stem cell transplantation is the only cure. […] There are treatments to manage aplastic anemia symptoms, but a stem cell transplantation is the only cure. […] Treatments vary depending on your situation. For example, some people develop aplastic anemia because theyre receiving cancer or autoimmune disease treatments. In that case, providers may be able to treat aplastic anemia by changing the treatments. […] Treatments for more serious forms of aplastic anemia include: Immunosuppressants. These medicines hold back your immune system so it stops attacking your stem cells. Antithymocyte globulin injection (Atgam) and cyclosporine (Sandimmune) are examples of immunosuppressants that treat aplastic anemia.

• #3 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). Severe and very severe aplastic anemia (SAA and VSAA, respectively; see Workup/Staging) have a mortality rate of greater than 70% with supportive care alone and are therefore a hematologic emergency. Treatment should be instituted promptly for SAA or VSAA, and clinicians must stress the need for patient compliance with therapy. […] Inpatient care for patients with aplastic anemia may be needed during periods of infection and for specific therapies, such as antithymocyte globulin (ATG) or HCT. In addition, iron chelation may be required in chronically transfused patients who develop elevated serum ferritin levels above 1000 g/L. […] The British Committee for Standards in Haematology recommends treating infection or uncontrolled bleeding before administering immunosuppressive therapy, including in patients scheduled for HCT.

• #4 Treatment for aplastic anaemia | NHS inform

  https://www.nhsinform.scot/illnesses-and-conditions/blood-and-lymph/aplastic-anaemia/treatment-for-aplastic-anaemia/

  If you have aplastic anaemia, your treatment will usually be managed by haematologists. They are specialist blood doctors. […] Treatments will be tailored to your individual needs and can vary depending on: how severe your aplastic anaemia is, your age, your general health, if you have any underlying health conditions. […] The general approach to treatment for patients with aplastic anaemia involves: Looking for any specific causes. Stopping any medications or drugs that might have contributed to the problem. Providing supportive care to treat symptoms and reduce the risk of bleeding and infections. Considering definitive therapy with immunosuppression or stem cell transplant. […] Supportive care aims to treat symptoms of aplastic anaemia. It involves different treatments depending on how severe the condition is. For example, you may need blood or platelet transfusions, and/or medicines to help prevent infections.

• #5 Narrative review of aplastic anemia—the importance of supportive treatment – Urbanowicz – Annals of Palliative Medicine

  https://apm.amegroups.org/article/view/58521/html

  Aplastic anemia (AA) is a rare, life-threatening syndrome of bone marrow failure resulted from bone marrow hypoplasia or aplasia, leading to pancytopenia (not only anemia). […] The concepts of treatment of patients with AA have significantly evolved in recent years. […] All patients diagnosed with AA require appropriate supportive treatment adapted to the current clinical situation. Supportive treatment is necessary both before, during and after invasive causal treatment, it mainly involves the transfusion of leukocyte-depleted blood components, the use of anti-infectious prophylaxis or treatment of infections. […] In many cases AA, supportive therapy is the only therapeutic option, especially in elderly patients with comorbidities. […] Appropriate supportive treatment is an integral element of therapy in patients with AA.

• #6 Treatment of Aplastic Anemia & Myelodysplastic Syndromes – NIDDK

  https://www.niddk.nih.gov/health-information/blood-diseases/aplastic-anemia-myelodysplastic-syndromes/treatment

  Health care professionals treat aplastic anemia by first treating any underlying condition that might be triggering the aplastic anemia, if an underlying condition is suspected. This may include removing exposure to a chemical, stopping a certain medicine, or treating a viral infection. […] Your care team may recommend one or more of the following treatment options: blood transfusion, bone marrow stem-cell transplants, immunosuppressive therapy, medicines. […] Treatments for aplastic anemia can ease your symptoms, improve your quality of life, and, in some cases, provide a cure for the disorder. […] Stem-cell transplant is the only possible cure for aplastic anemia. […] Talk with your health care professional about the risk and benefits of a stem-cell transplant and if the procedure is right for you.

• #7 Aplastic anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/aplastic-anemia/diagnosis-treatment/drc-20355020

  Your hematologist may prescribe medicines to stimulate your bone marrow to make new blood cells. These drugs are called growth factors or colony-stimulating factors. […] If you have aplastic anemia, see your healthcare professional at the first sign of infection, such as a fever. […] Aplastic anemia caused by radiation and chemotherapy treatments for cancer usually improves after those treatments stop. The same is true for most other drugs that can cause aplastic anemia. […] Pregnant women with aplastic anemia are treated with blood transfusions. For many women, pregnancy-related aplastic anemia improves once the pregnancy ends. If that doesn’t happen, treatment is still necessary.

• #8 Narrative review of aplastic anemia—the importance of supportive treatment – Urbanowicz – Annals of Palliative Medicine

  https://apm.amegroups.org/article/view/58521/html

  Aplastic anemia (AA) is a rare, life-threatening syndrome of bone marrow failure resulted from bone marrow hypoplasia or aplasia, leading to pancytopenia (not only anemia). […] The concepts of treatment of patients with AA have significantly evolved in recent years. […] All patients diagnosed with AA require appropriate supportive treatment adapted to the current clinical situation. Supportive treatment is necessary both before, during and after invasive causal treatment, it mainly involves the transfusion of leukocyte-depleted blood components, the use of anti-infectious prophylaxis or treatment of infections. […] In many cases AA, supportive therapy is the only therapeutic option, especially in elderly patients with comorbidities. […] Appropriate supportive treatment is an integral element of therapy in patients with AA.

• #9 Aplastic Anemia Drugs and Treatments | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/aplastic-anemia/drugs-treatments

  More drugs and treatments are available to treat bone marrow failure disease than ever before. […] Alemtuzumab is in clinical trials for treating aplastic anemia. […] Androgens are sometimes used to treat aplastic anemia and PNH. […] ATG is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. […] A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia will receive at least one blood transfusion. […] A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT). The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure.

• #10 Treatment for aplastic anaemia | NHS inform

  https://www.nhsinform.scot/illnesses-and-conditions/blood-and-lymph/aplastic-anaemia/treatment-for-aplastic-anaemia/

  Some patients may be offered immuno-suppressive treatment (IST) with therapies that are designed to suppress your immune system to allow your bone marrow to recover. This might include medicines like anti-thymocyte globulin (ATG) and ciclosporin. […] If you have severe or very severe aplastic anaemia, you will receive supportive care. This means that you may need: to stop any medications that could be contributing to a low blood count, blood or platelet transfusion to maintain your blood count, medicines to prevent infection. […] In addition, patients with severe or very severe aplastic anaemia will usually need definitive treatment to allow the bone marrow to recover. Definitive treatment involves either a stem cell transplant or immuno-suppressive treatment (IST). […] Some people with aplastic anaemia may be treated with anti-thymocyte globulin (ATG). This is usually given in hospital over a few days through an infusion or drip.

• #11 Aplastic anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/aplastic-anemia/diagnosis-treatment/drc-20355020

  Treatments for aplastic anemia depend on the symptoms and your age and overall health. Treatment options might include observation, blood transfusions, medicines or bone marrow transplantation. Severe aplastic anemia, in which your blood cell counts are extremely low, is life-threatening and requires immediate hospitalization. […] Although not a cure for aplastic anemia, blood transfusions can provide blood cells your bone marrow isn’t producing. This can help control bleeding and relieve symptoms. […] A stem cell transplant to rebuild the bone marrow with stem cells from a donor might be the only successful treatment option for people with severe aplastic anemia. […] For people who can’t undergo a bone marrow transplant or for those whose aplastic anemia is due to an autoimmune disorder, treatment can involve drugs that alter or suppress the immune system, called immunosuppressants.

• #12 Narrative review of aplastic anemia—the importance of supportive treatment – Urbanowicz – Annals of Palliative Medicine

  https://apm.amegroups.org/article/view/58521/html

  Patients after IST and allo-HSCT treatment require long-term symptomatic management including platelet cell concentrate and red blood cell concentrate transfusions and treatment of infectious complications. […] For any patient for allo-HSCT, transfusions should be used selectively to reduce the risk of immunization to donor antigens. […] The indication for the administration of red blood cell concentrate transfusions is a decrease in hemoglobin (HGB) concentration below 6 g/dL and prophylactic platelet transfusions should be performed when the patients platelet count (PLT) drops below 10109/L. […] Patients with AA who receive multiple red blood cell transfusions are at risk of iron overload and associated organ damage. […] Comprehensive therapy of a patient with AA, taking into account his causal treatment and the necessary supportive treatment, depending on the clinical condition, will guarantee the achievement of therapeutic success.

• #13 Treatment for aplastic anaemia | NHS inform

  https://www.nhsinform.scot/illnesses-and-conditions/blood-and-lymph/aplastic-anaemia/treatment-for-aplastic-anaemia/

  Some patients may be offered immuno-suppressive treatment (IST) with therapies that are designed to suppress your immune system to allow your bone marrow to recover. This might include medicines like anti-thymocyte globulin (ATG) and ciclosporin. […] If you have severe or very severe aplastic anaemia, you will receive supportive care. This means that you may need: to stop any medications that could be contributing to a low blood count, blood or platelet transfusion to maintain your blood count, medicines to prevent infection. […] In addition, patients with severe or very severe aplastic anaemia will usually need definitive treatment to allow the bone marrow to recover. Definitive treatment involves either a stem cell transplant or immuno-suppressive treatment (IST). […] Some people with aplastic anaemia may be treated with anti-thymocyte globulin (ATG). This is usually given in hospital over a few days through an infusion or drip.

• #14 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). Severe and very severe aplastic anemia (SAA and VSAA, respectively; see Workup/Staging) have a mortality rate of greater than 70% with supportive care alone and are therefore a hematologic emergency. Treatment should be instituted promptly for SAA or VSAA, and clinicians must stress the need for patient compliance with therapy. […] Inpatient care for patients with aplastic anemia may be needed during periods of infection and for specific therapies, such as antithymocyte globulin (ATG) or HCT. In addition, iron chelation may be required in chronically transfused patients who develop elevated serum ferritin levels above 1000 g/L. […] The British Committee for Standards in Haematology recommends treating infection or uncontrolled bleeding before administering immunosuppressive therapy, including in patients scheduled for HCT.

• #15 Aplastic Anaemia: Causes and Treatment | Doctor

  https://patient.info/doctor/aplastic-anaemia

  Blood transfusions should be given to improve quality of life. […] Aplastic anaemia patients who are severely neutropenic should be given prophylactic antibiotics and antifungal therapy according to local policies. […] Aplastic anaemia patients receiving immunosuppressive therapy should also receive prophylactic antiviral agents, although routine prophylaxis against Pneumocystis jirovecii is not necessary. […] The major causes of morbidity and mortality from aplastic anaemia include infection and bleeding. […] Improvements in bone marrow transplantation and immunosuppression have increased the number of long-term survivors of patients with aplastic anaemia.

• #16 Aplastic anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/aplastic-anemia/diagnosis-treatment/drc-20355020

  Your hematologist may prescribe medicines to stimulate your bone marrow to make new blood cells. These drugs are called growth factors or colony-stimulating factors. […] If you have aplastic anemia, see your healthcare professional at the first sign of infection, such as a fever. […] Aplastic anemia caused by radiation and chemotherapy treatments for cancer usually improves after those treatments stop. The same is true for most other drugs that can cause aplastic anemia. […] Pregnant women with aplastic anemia are treated with blood transfusions. For many women, pregnancy-related aplastic anemia improves once the pregnancy ends. If that doesn’t happen, treatment is still necessary.

• #17 Treatment of Aplastic Anemia & Myelodysplastic Syndromes – NIDDK

  https://www.niddk.nih.gov/health-information/blood-diseases/aplastic-anemia-myelodysplastic-syndromes/treatment

  Immunosuppressive therapy may be used for people who cannot have a stem-cell transplant or to control aplastic anemia in people who are waiting for a stem-cell transplant. […] Your health care professional may use a medicine called eltrombopag in combination with an immunosuppressant to increase the number of blood cells in your body. […] Your care team may give you medicine to kill bacteria, fungi, or viruses that cause infection.

• #18 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Immunosuppressive drug therapy lowers your body’s immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. […] For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the treatment of choice. It is also used for aplastic anemia patients who do not have a matched bone marrow donor. […] The three drugs used in immunosuppressive therapy include: Anti-Thymocyte Globulin (ATG), Cyclosporine, Eltrombopag. […] ATG is approved in the U.S. to treat acquired aplastic anemia and to reduce the chance of organ rejection after a kidney or other organ transplant. […] ATG works by killing specific cells in your immune system called T-lymphocytes — the cells that are attacking bone marrow stem cells in aplastic anemia.

• #19 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Immunosuppressive drug therapy lowers your body’s immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. […] For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the treatment of choice. It is also used for aplastic anemia patients who do not have a matched bone marrow donor. […] The three drugs used in immunosuppressive therapy include: Anti-Thymocyte Globulin (ATG), Cyclosporine, Eltrombopag. […] ATG is approved in the U.S. to treat acquired aplastic anemia and to reduce the chance of organ rejection after a kidney or other organ transplant. […] ATG works by killing specific cells in your immune system called T-lymphocytes — the cells that are attacking bone marrow stem cells in aplastic anemia.

• #20 Aplastic Anaemia: Causes and Treatment | Doctor

  https://patient.info/doctor/aplastic-anaemia

  Management of aplastic anaemia […] Treatment should be based on the degree of cytopenia, not the marrow cellularity. Patients with asymptomatic cytopenias probably need no treatment. […] Indications for haemopoietic stem cell transplant (HSCT): HLA identical sibling donor: severe aplastic anaemia in young and adult patients who have a matched sibling donor. […] Immunosuppressive therapy is recommended first-line therapy for: Non-severe aplastic anaemia requiring treatment. Severe or very severe aplastic anaemia patients who lack a matched sibling donor; and Severe or very severe aplastic anaemia patients aged over 35-50 years. […] The first-line immunosuppressive therapy is horse antithymocyte globulin (ATG) combined with ciclosporin. Other agents such as alemtuzumab are also used.

• #21 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Usually, ATG is given by IV infusion into a vein for 8-12 hours a day, for 4 days. […] When used alone, ATG improves blood counts for aplastic anemia patients about half of the time. […] When used with cyclosporine, ATG improves blood counts in about 7 out of 10 cases. […] When a platelet growth factor called eltrombopag (Promacta) is also given, more than 7 in 10 people see improved blood counts. […] If it works, ATG usually stops the need for blood transfusions within 3 months. […] Cyclosporine prevents T-lymphocytes, a type of white blood cell, from becoming active. […] Cyclosporine is typically used in combination with ATG to treat acquired aplastic anemia. […] Eltrombopag (Promacta) is used to increase the number of platelets (cells that help blood clot) and to decrease the risk of bleeding. […] Eltrombopag was approved by the U.S. Food and Drug Administration in 2014 to treat refractory aplastic anemia. […] In 2018, Eltrombopag was FDA approved as a first line therapy to treat aplastic anemia.

• #22 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Immunosuppressive therapy is also recommended in patients with nonsevere aplastic anemia who are transfusion dependent. […] Immunosuppressive therapy is associated with an overall response rate of 60-80% and a 5-year survival rate of 75% in most reports, but event-free survival rates are in the range of 35-50%. […] The treatment response in aplastic anemia, unlike in other autoimmune diseases, is slow. At least 4-12 weeks is usually needed to observe early improvement, and the patient may continue to improve slowly thereafter. Late responses may also occur. About 50% of patients respond by 3 months after ATG administration, and about 75% respond by 6 months. Full hematologic recovery is rare, but most patients improve to a functional hematologic recovery and become transfusion independent.

• #23 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Immunosuppressive drug therapy lowers your body’s immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. […] For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the treatment of choice. It is also used for aplastic anemia patients who do not have a matched bone marrow donor. […] The three drugs used in immunosuppressive therapy include: Anti-Thymocyte Globulin (ATG), Cyclosporine, Eltrombopag. […] ATG is approved in the U.S. to treat acquired aplastic anemia and to reduce the chance of organ rejection after a kidney or other organ transplant. […] ATG works by killing specific cells in your immune system called T-lymphocytes — the cells that are attacking bone marrow stem cells in aplastic anemia.

• #24 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Usually, ATG is given by IV infusion into a vein for 8-12 hours a day, for 4 days. […] When used alone, ATG improves blood counts for aplastic anemia patients about half of the time. […] When used with cyclosporine, ATG improves blood counts in about 7 out of 10 cases. […] When a platelet growth factor called eltrombopag (Promacta) is also given, more than 7 in 10 people see improved blood counts. […] If it works, ATG usually stops the need for blood transfusions within 3 months. […] Cyclosporine prevents T-lymphocytes, a type of white blood cell, from becoming active. […] Cyclosporine is typically used in combination with ATG to treat acquired aplastic anemia. […] Eltrombopag (Promacta) is used to increase the number of platelets (cells that help blood clot) and to decrease the risk of bleeding. […] Eltrombopag was approved by the U.S. Food and Drug Administration in 2014 to treat refractory aplastic anemia. […] In 2018, Eltrombopag was FDA approved as a first line therapy to treat aplastic anemia.

• #25 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Usually, ATG is given by IV infusion into a vein for 8-12 hours a day, for 4 days. […] When used alone, ATG improves blood counts for aplastic anemia patients about half of the time. […] When used with cyclosporine, ATG improves blood counts in about 7 out of 10 cases. […] When a platelet growth factor called eltrombopag (Promacta) is also given, more than 7 in 10 people see improved blood counts. […] If it works, ATG usually stops the need for blood transfusions within 3 months. […] Cyclosporine prevents T-lymphocytes, a type of white blood cell, from becoming active. […] Cyclosporine is typically used in combination with ATG to treat acquired aplastic anemia. […] Eltrombopag (Promacta) is used to increase the number of platelets (cells that help blood clot) and to decrease the risk of bleeding. […] Eltrombopag was approved by the U.S. Food and Drug Administration in 2014 to treat refractory aplastic anemia. […] In 2018, Eltrombopag was FDA approved as a first line therapy to treat aplastic anemia.

• #26 Treatment for aplastic anaemia | NHS inform

  https://www.nhsinform.scot/illnesses-and-conditions/blood-and-lymph/aplastic-anaemia/treatment-for-aplastic-anaemia/

  ATG helps to reduce T cells which helps bone marrow recover from the damage. […] ATG is usually combined with a tablet immunosuppressant called Ciclosporin. Its likely that youll need to continue taking this for about a year. […] Some patients may also be offered other treatments like Eltrombopag. This is a medication that stimulates stem cell and platelet production. […] The treatments for aplastic anaemia can be significant and there can be a long recovery time. […] Patients on immunosuppressive therapy will need to be monitored closely. Theyll also need regular blood tests to check for any problems. […] Some people with aplastic anaemia may be considered for a stem cell transplant. This depends on: the severity of your aplastic anaemia, your underlying health and fitness, the availability of a suitable donor.

• #27 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Eltrobombopag (Promacta) is an oral thrombopoietin receptor agonist that was approved in 2014 for patients with severe aplastic anemia that failed to respond adequately to at least one prior immunosuppressive therapy (IST) regimen. […] Eltrombopag has also shown benefit as first-line therapy. […] In 2018, the FDA expanded approval for eltrombopag to include use as first-line therapy, in combination with standard IST, for adult and pediatric patients 2 years and older with severe aplastic anemia. […] Romiplostim is a peptibody (Fc antibody fusion protein) with thrombopoietin receptor (also known as c-MPL) agonist activity that stimulates endogenous thrombopoietin production, promoting the proliferation and differentiation of megakaryocytes in the bone marrow.

• #28 Aplastic Anemia – Hematology and Oncology – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/hematology-and-oncology/anemias-caused-by-deficient-erythropoiesis/aplastic-anemia

  For adults with severe aplastic anemia receiving immunosuppressive therapy, expert consensus guidelines recommend ATG, cyclosporine, and eltrombopag as initial management based on donor availability and fitness for bone marrow transplant. […] Addition of eltrombopag to ATG and cyclosporine yielded faster and improved hematologic response rates compared to ATG and cyclosporine alone but with similar overall survival at 2 years. […] While many experts recommend 'triple immunosuppressive therapy’ (eg, ATG, cyclosporine, and eltrombopag) over ATG plus cyclosporine alone, others avoid addition of eltrombopag due to concerns regarding earlier malignant clonal evolution and lack of improvement in overall survival.

• #29 Immunosuppressive Therapy | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/treatment/immunosuppressive-therapy

  Usually, ATG is given by IV infusion into a vein for 8-12 hours a day, for 4 days. […] When used alone, ATG improves blood counts for aplastic anemia patients about half of the time. […] When used with cyclosporine, ATG improves blood counts in about 7 out of 10 cases. […] When a platelet growth factor called eltrombopag (Promacta) is also given, more than 7 in 10 people see improved blood counts. […] If it works, ATG usually stops the need for blood transfusions within 3 months. […] Cyclosporine prevents T-lymphocytes, a type of white blood cell, from becoming active. […] Cyclosporine is typically used in combination with ATG to treat acquired aplastic anemia. […] Eltrombopag (Promacta) is used to increase the number of platelets (cells that help blood clot) and to decrease the risk of bleeding. […] Eltrombopag was approved by the U.S. Food and Drug Administration in 2014 to treat refractory aplastic anemia. […] In 2018, Eltrombopag was FDA approved as a first line therapy to treat aplastic anemia.

• #30 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Immunosuppressive therapy is also recommended in patients with nonsevere aplastic anemia who are transfusion dependent. […] Immunosuppressive therapy is associated with an overall response rate of 60-80% and a 5-year survival rate of 75% in most reports, but event-free survival rates are in the range of 35-50%. […] The treatment response in aplastic anemia, unlike in other autoimmune diseases, is slow. At least 4-12 weeks is usually needed to observe early improvement, and the patient may continue to improve slowly thereafter. Late responses may also occur. About 50% of patients respond by 3 months after ATG administration, and about 75% respond by 6 months. Full hematologic recovery is rare, but most patients improve to a functional hematologic recovery and become transfusion independent.

• #31 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Both the British Committee for Standards in Haematology and the Pediatric Haemato-Oncology Italian Association recommend HCT from a matched sibling donor for severe aplastic anemia. If a matched donor is not available, options include immunosuppressive therapy or unrelated donor HCT. […] Approximately one third of patients with aplastic anemia do not respond to immunosuppression. The thrombopoietin receptor agonist eltrombopag is approved for use in patients with severe aplastic anemia who fail to respond adequately to immunosuppressive therapy. […] Pregnant women with aplastic anemia have a 33% risk of relapse. […] Note that monotherapy with hematopoietic growth factors (eg, recombinant human erythropoietin [rHuEPO], granulocyte colony-stimulating factor [G-CSF]) is not recommended for newly diagnosed patients.

• #32 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Immunosuppressive therapy is also recommended in patients with nonsevere aplastic anemia who are transfusion dependent. […] Immunosuppressive therapy is associated with an overall response rate of 60-80% and a 5-year survival rate of 75% in most reports, but event-free survival rates are in the range of 35-50%. […] The treatment response in aplastic anemia, unlike in other autoimmune diseases, is slow. At least 4-12 weeks is usually needed to observe early improvement, and the patient may continue to improve slowly thereafter. Late responses may also occur. About 50% of patients respond by 3 months after ATG administration, and about 75% respond by 6 months. Full hematologic recovery is rare, but most patients improve to a functional hematologic recovery and become transfusion independent.

• #33 Treatment for Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia/treatment.html

  Aplastic anemia can be mild, moderate, or severe. […] Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with blood transfusions and/or medications, immunosuppressive drugs, and growth factors. […] Severe aplastic anemia, in which blood cell counts are extremely low, is life-threatening, and requires immediate hospitalization for treatment, which is usually a bone marrow transplant. Replacing diseased bone marrow with healthy bone marrow from a donor often successfully cures aplastic anemia. […] Because one possible cause of aplastic anemia is the immune system working against itself, one or more immunosuppressive drugs are often given until a stem cell transplant can be performed. […] Growth factors are drugs that help the body make more blood cells, which may be given to reduce the number or need for blood transfusions.

• #34 Treatment of Aplastic Anemia & Myelodysplastic Syndromes – NIDDK

  https://www.niddk.nih.gov/health-information/blood-diseases/aplastic-anemia-myelodysplastic-syndromes/treatment

  Health care professionals treat aplastic anemia by first treating any underlying condition that might be triggering the aplastic anemia, if an underlying condition is suspected. This may include removing exposure to a chemical, stopping a certain medicine, or treating a viral infection. […] Your care team may recommend one or more of the following treatment options: blood transfusion, bone marrow stem-cell transplants, immunosuppressive therapy, medicines. […] Treatments for aplastic anemia can ease your symptoms, improve your quality of life, and, in some cases, provide a cure for the disorder. […] Stem-cell transplant is the only possible cure for aplastic anemia. […] Talk with your health care professional about the risk and benefits of a stem-cell transplant and if the procedure is right for you.

• #35 Clinical management of aplastic anemia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3138728/

  Acquired aplastic anemia is a potentially fatal bone marrow failure disorder that is characterized by pancytopenia and a hypocellular bone marrow. Hematopoietic stem-cell transplantation or bone marrow transplantation (BMT) is the treatment of choice for young patients who have a matched sibling donor. […] Immunosuppression with either anti-thymocyte globulin and cyclosporine or high-dose cyclophosphamide is an effective therapy for patients who are not suitable BMT candidates owing to age or lack of a suitable donor. […] Aplastic anemia is classified as non-severe (NSAA), severe (SAA) and very severe based on the degree of the peripheral blood cytopenias. […] Before initiating more definitive therapies for acquired SAA, the clinician must consider the significant supportive care required for these patients.

• #36 Aplastic Anemia: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/16747-aplastic-anemia

  Allogeneic stem cell transplantation. Providers replace damaged stem cells in your bone marrow with healthy stem cells from donated blood or bone marrow. […] Depending on your situation, a successful allogeneic stem cell transplantation may cure the condition. […] If you have aplastic anemia, your healthcare provider will outline treatment options and side effects so you know what to expect.

• #37 Clinical management of aplastic anemia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3138728/

  Patients with severe cytopenias require urgent support with blood products. […] Bone marrow transplantation is potentially curative and is the treatment of choice for children and young adults (age of 30 years) with SAA who have an HLA-matched sibling donor. […] Another highly effective therapy for SAA is ATG and CsA IST and is generally first-line therapy for SAA patients who lack matched sibling donors or are not good candidates for BMT. […] High-dose CY therapy seems to produce higher quality remissions with fewer relapses, but this has not been confirmed in randomized controlled trials.

• #38 Aplastic anemia – UF Health

  https://ufhealth.org/conditions-and-treatments/aplastic-anemia

  Mild cases of aplastic anemia that do not have symptoms may not require treatment. […] As blood cell counts become lower and symptoms develop, blood and platelets are given through transfusions. Over time, transfusions may stop working, resulting in very low blood cell counts. This is a life-threatening condition. […] Bone marrow, or stem cell transplant may be recommended for younger people. It is more likely to be recommended for those 50 years and younger, but people over 50 may receive a transplant if they are healthy enough. This treatment works best when the donor is a fully-matched brother or sister. This is called a matched sibling donor. […] Older people and those who do not have a matched sibling donor are given medicine to suppress the immune system. These medicines may allow the bone marrow to once again make healthy blood cells. But the disease may return (relapse). A bone marrow transplant with an unrelated donor may be tried if these medicines do not help or if the disease comes back after getting better.

• #39 Treatment for Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia/treatment.html

  Fred Hutch physicians pioneered bone marrow transplants for aplastic anemia treatment. […] If blood and marrow or stem cell transplantation is indicated, patients will be seen by Fred Hutch physicians who are world leaders in stem cell and bone marrow transplantation. […] When patients have the opportunity to use a matched siblings stem cells or bone marrow, there is a 90 percent success rate.

• #40 Clinical management of aplastic anemia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3138728/

  Patients with severe cytopenias require urgent support with blood products. […] Bone marrow transplantation is potentially curative and is the treatment of choice for children and young adults (age of 30 years) with SAA who have an HLA-matched sibling donor. […] Another highly effective therapy for SAA is ATG and CsA IST and is generally first-line therapy for SAA patients who lack matched sibling donors or are not good candidates for BMT. […] High-dose CY therapy seems to produce higher quality remissions with fewer relapses, but this has not been confirmed in randomized controlled trials.

• #41 Aplastic anemia – Wikipedia

  https://en.wikipedia.org/wiki/Aplastic_anemia

  First-line treatment for aplastic anemia consists of immunosuppressive drugs typically either anti-lymphocyte globulin or anti-thymocyte globulin combined with corticosteroids, chemotherapy, and ciclosporin. Hematopoietic stem cell transplantation is also used, especially for patients under 30 years of age with a related, matched marrow donor. […] Treating immune-mediated aplastic anemia involves suppression of the immune system, an effect achieved by daily medicine or, in more severe cases, a bone marrow transplant, a potential cure. The transplanted bone marrow replaces the failing bone marrow cells with new ones from a matching donor. […] In young patients with an HLA-matched sibling donor, bone marrow transplant can be considered as a first-line treatment. Patients lacking a matched sibling donor typically pursue immunosuppression as a first-line treatment, and matched, unrelated donor transplants are considered second-line therapy.

• #42 New Recommendations for Severe Aplastic Anemia Highlight Critical Gaps in Pediatric Research | Research | Advances in Pediatric Oncology | NewYork-Presbyterian

  https://www.nyp.org/advances/article/pediatric-oncology/new-recommendations-for-severe-aplastic-anemia-highlight-critical-gaps-in-pediatric-research

  A matched sibling donor stem cell transplant is the gold-standard first-line treatment for pediatric patients with severe aplastic anemia, with cure rates of 90% or greater. […] When a matched donor is not available, the recommendations call for immunosuppressive therapy though in some clinical settings, a matched unrelated donor stem cell transplant could be an option. […] For newly diagnosed patients without a matched sibling donor, the best current evidence supports immunosuppressive therapy with horse anti-thymocyte globulin (ATG) in combination with cyclosporine. […] Right now, transplant with an unrelated donor is not a universally accepted standard of care first-line option. However, as we get better at matching donors, decreasing the toxicity of conditioning regimens, improving therapies for Graft Versus Host Disease (GVHD) and decreasing the morbidity associated with the transplant, it could move up. […] More studies are needed to help define a subgroup of pediatric patients who could potentially benefit from this therapy.

• #43 Aplastic anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/aplastic-anemia/diagnosis-treatment/drc-20355020

  Your hematologist may prescribe medicines to stimulate your bone marrow to make new blood cells. These drugs are called growth factors or colony-stimulating factors. […] If you have aplastic anemia, see your healthcare professional at the first sign of infection, such as a fever. […] Aplastic anemia caused by radiation and chemotherapy treatments for cancer usually improves after those treatments stop. The same is true for most other drugs that can cause aplastic anemia. […] Pregnant women with aplastic anemia are treated with blood transfusions. For many women, pregnancy-related aplastic anemia improves once the pregnancy ends. If that doesn’t happen, treatment is still necessary.

• #44 Treatment for Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia/treatment.html

  Aplastic anemia can be mild, moderate, or severe. […] Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with blood transfusions and/or medications, immunosuppressive drugs, and growth factors. […] Severe aplastic anemia, in which blood cell counts are extremely low, is life-threatening, and requires immediate hospitalization for treatment, which is usually a bone marrow transplant. Replacing diseased bone marrow with healthy bone marrow from a donor often successfully cures aplastic anemia. […] Because one possible cause of aplastic anemia is the immune system working against itself, one or more immunosuppressive drugs are often given until a stem cell transplant can be performed. […] Growth factors are drugs that help the body make more blood cells, which may be given to reduce the number or need for blood transfusions.

• #45 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Both the British Committee for Standards in Haematology and the Pediatric Haemato-Oncology Italian Association recommend HCT from a matched sibling donor for severe aplastic anemia. If a matched donor is not available, options include immunosuppressive therapy or unrelated donor HCT. […] Approximately one third of patients with aplastic anemia do not respond to immunosuppression. The thrombopoietin receptor agonist eltrombopag is approved for use in patients with severe aplastic anemia who fail to respond adequately to immunosuppressive therapy. […] Pregnant women with aplastic anemia have a 33% risk of relapse. […] Note that monotherapy with hematopoietic growth factors (eg, recombinant human erythropoietin [rHuEPO], granulocyte colony-stimulating factor [G-CSF]) is not recommended for newly diagnosed patients.

• #46 Treatment for Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia/treatment.html

  Aplastic anemia can be mild, moderate, or severe. […] Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with blood transfusions and/or medications, immunosuppressive drugs, and growth factors. […] Severe aplastic anemia, in which blood cell counts are extremely low, is life-threatening, and requires immediate hospitalization for treatment, which is usually a bone marrow transplant. Replacing diseased bone marrow with healthy bone marrow from a donor often successfully cures aplastic anemia. […] Because one possible cause of aplastic anemia is the immune system working against itself, one or more immunosuppressive drugs are often given until a stem cell transplant can be performed. […] Growth factors are drugs that help the body make more blood cells, which may be given to reduce the number or need for blood transfusions.

• #47 Aplastic Anemia Drugs and Treatments | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/aplastic-anemia/drugs-treatments

  More drugs and treatments are available to treat bone marrow failure disease than ever before. […] Alemtuzumab is in clinical trials for treating aplastic anemia. […] Androgens are sometimes used to treat aplastic anemia and PNH. […] ATG is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. […] A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia will receive at least one blood transfusion. […] A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT). The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure.

• #48 Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study | Leukemia

  https://www.nature.com/articles/s41375-023-02047-z

  Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. […] The majority required treatment with cyclosporine (CyA) alone (N=84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. […] The different management strategies were registered and categorized as: 1) cyclosporin plus/minus steroids (CyA), 2) ATG plus CyA, 3) eltrombopag plus CyA, 4) eltrombopag single agent, and 4) other. […] Treatments included CyA (46%), CyA plus ATG (24%), CyA plus eltrombopag (11%), eltrombopag alone (5%), and other treatments (14%; N=16 androgens, 3 alemtuzumab, 2 daclizumab, 1 azathioprine, and 3 tacrolimus).

• #49 Clinical management of aplastic anemia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3138728/

  Patients with severe cytopenias require urgent support with blood products. […] Bone marrow transplantation is potentially curative and is the treatment of choice for children and young adults (age of 30 years) with SAA who have an HLA-matched sibling donor. […] Another highly effective therapy for SAA is ATG and CsA IST and is generally first-line therapy for SAA patients who lack matched sibling donors or are not good candidates for BMT. […] High-dose CY therapy seems to produce higher quality remissions with fewer relapses, but this has not been confirmed in randomized controlled trials.

• #50 Aplastic Anemia Drugs and Treatments | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/aplastic-anemia/drugs-treatments

  Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. […] Cyclophosphamide is in a class of medications called alkylating agents. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body’s immune system.

• #51 Treatment for Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia/treatment.html

  Aplastic anemia can be mild, moderate, or severe. […] Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with blood transfusions and/or medications, immunosuppressive drugs, and growth factors. […] Severe aplastic anemia, in which blood cell counts are extremely low, is life-threatening, and requires immediate hospitalization for treatment, which is usually a bone marrow transplant. Replacing diseased bone marrow with healthy bone marrow from a donor often successfully cures aplastic anemia. […] Because one possible cause of aplastic anemia is the immune system working against itself, one or more immunosuppressive drugs are often given until a stem cell transplant can be performed. […] Growth factors are drugs that help the body make more blood cells, which may be given to reduce the number or need for blood transfusions.

• #52 Aplastic Anemia: Types, Symptoms, Causes & Treatment

  https://www.webmd.com/a-to-z-guides/aplastic-anemia

  Aplastic Anemia Treatments […] If your doctor can identify the cause of your aplastic anemia and get rid of that exposure, the condition may go away. For instance, if your aplastic anemia is related to a treatment you’re getting for another condition, changing treatments may help. But doctors can rarely pinpoint the exact cause. […] If your aplastic anemia isnt severe, you may not need treatment unless or until your blood count drops below a certain level. Your doctor may suggest you to watch and wait. If your condition is more severe, you’ll have a range of treatment options. […] Drug treatments […] Some medicines may help increase your blood cell counts, including: Bone marrow stimulants. Your doctor may prescribe growth factors or medicines to help your bone marrow make more blood cells. Immunosuppressants. Your doctor may prescribe steroids or other immune-suppressing medicines if your aplastic anemia is caused by an autoimmune condition. […]

• #53 Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study | Leukemia

  https://www.nature.com/articles/s41375-023-02047-z

  Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. […] The majority required treatment with cyclosporine (CyA) alone (N=84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. […] The different management strategies were registered and categorized as: 1) cyclosporin plus/minus steroids (CyA), 2) ATG plus CyA, 3) eltrombopag plus CyA, 4) eltrombopag single agent, and 4) other. […] Treatments included CyA (46%), CyA plus ATG (24%), CyA plus eltrombopag (11%), eltrombopag alone (5%), and other treatments (14%; N=16 androgens, 3 alemtuzumab, 2 daclizumab, 1 azathioprine, and 3 tacrolimus).

• #54 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). Severe and very severe aplastic anemia (SAA and VSAA, respectively; see Workup/Staging) have a mortality rate of greater than 70% with supportive care alone and are therefore a hematologic emergency. Treatment should be instituted promptly for SAA or VSAA, and clinicians must stress the need for patient compliance with therapy. […] Inpatient care for patients with aplastic anemia may be needed during periods of infection and for specific therapies, such as antithymocyte globulin (ATG) or HCT. In addition, iron chelation may be required in chronically transfused patients who develop elevated serum ferritin levels above 1000 g/L. […] The British Committee for Standards in Haematology recommends treating infection or uncontrolled bleeding before administering immunosuppressive therapy, including in patients scheduled for HCT.

• #55 Aplastic anemia – Wikipedia

  https://en.wikipedia.org/wiki/Aplastic_anemia

  First-line treatment for aplastic anemia consists of immunosuppressive drugs typically either anti-lymphocyte globulin or anti-thymocyte globulin combined with corticosteroids, chemotherapy, and ciclosporin. Hematopoietic stem cell transplantation is also used, especially for patients under 30 years of age with a related, matched marrow donor. […] Treating immune-mediated aplastic anemia involves suppression of the immune system, an effect achieved by daily medicine or, in more severe cases, a bone marrow transplant, a potential cure. The transplanted bone marrow replaces the failing bone marrow cells with new ones from a matching donor. […] In young patients with an HLA-matched sibling donor, bone marrow transplant can be considered as a first-line treatment. Patients lacking a matched sibling donor typically pursue immunosuppression as a first-line treatment, and matched, unrelated donor transplants are considered second-line therapy.

• #56 Aplastic anaemia | Great Ormond Street Hospital

  https://www.gosh.nhs.uk/conditions-and-treatments/conditions-we-treat/aplastic-anaemia/

  Aplastic anaemia is a serious condition affecting the blood, where the bone marrow and stem cells do not produce enough blood cells. […] This page explains about aplastic anaemia, how it is treated and what to expect when a child comes to Great Ormond Street Hospital (GOSH) for treatment. […] The aim of treatment for aplastic anaemia is twofold supportive management to correct the child’s initial symptoms and treatment of the bone marrow failure. […] When a child is diagnosed with aplastic anaemia, the first treatment is to correct his or her blood count. This may involve blood transfusions of platelets and red blood cells. […] The best treatment for idiopathic aplastic anaemia remains a bone marrow transplant from a brother or sister who is a tissue match. […] For those children who do not have a matched sibling, we have in the past given them immunosuppressive treatment with a medicine called ATG and ciclosporin.

• #57 New Recommendations for Severe Aplastic Anemia Highlight Critical Gaps in Pediatric Research | Research | Advances in Pediatric Oncology | NewYork-Presbyterian

  https://www.nyp.org/advances/article/pediatric-oncology/new-recommendations-for-severe-aplastic-anemia-highlight-critical-gaps-in-pediatric-research

  A matched sibling donor stem cell transplant is the gold-standard first-line treatment for pediatric patients with severe aplastic anemia, with cure rates of 90% or greater. […] When a matched donor is not available, the recommendations call for immunosuppressive therapy though in some clinical settings, a matched unrelated donor stem cell transplant could be an option. […] For newly diagnosed patients without a matched sibling donor, the best current evidence supports immunosuppressive therapy with horse anti-thymocyte globulin (ATG) in combination with cyclosporine. […] Right now, transplant with an unrelated donor is not a universally accepted standard of care first-line option. However, as we get better at matching donors, decreasing the toxicity of conditioning regimens, improving therapies for Graft Versus Host Disease (GVHD) and decreasing the morbidity associated with the transplant, it could move up. […] More studies are needed to help define a subgroup of pediatric patients who could potentially benefit from this therapy.

• #58 Aplastic anaemia treatment – Leukaemia Foundation

  https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/aplastic-anaemia/treatment/

  A child treated with immunosuppressive treatment has around an 80 percent chance of responding to treatment and requiring no further treatment options. A child who undergoes a donor transplant from a matched sibling has around a 90 percent chance at cure. […] Your doctor or your child’s treating doctor will discuss with you treatment options in the occurrence of relapse.

• #59 Aplastic anaemia | Great Ormond Street Hospital

  https://www.gosh.nhs.uk/conditions-and-treatments/conditions-we-treat/aplastic-anaemia/

  Aplastic anaemia is a serious condition affecting the blood, where the bone marrow and stem cells do not produce enough blood cells. […] This page explains about aplastic anaemia, how it is treated and what to expect when a child comes to Great Ormond Street Hospital (GOSH) for treatment. […] The aim of treatment for aplastic anaemia is twofold supportive management to correct the child’s initial symptoms and treatment of the bone marrow failure. […] When a child is diagnosed with aplastic anaemia, the first treatment is to correct his or her blood count. This may involve blood transfusions of platelets and red blood cells. […] The best treatment for idiopathic aplastic anaemia remains a bone marrow transplant from a brother or sister who is a tissue match. […] For those children who do not have a matched sibling, we have in the past given them immunosuppressive treatment with a medicine called ATG and ciclosporin.

• #60 Aplastic Anemia Treatment & Management: Approach Considerations, Blood Transfusion, Treatment of Infections

  https://emedicine.medscape.com/article/198759-treatment

  Frequent outpatient follow-up for patients with aplastic anemia is needed to monitor blood counts and any adverse effects of various drugs. Transfusions of packed red blood cells (RBCs) and platelets are administered on an outpatient basis. […] Patients with aplastic anemia should be treated by physicians who are experts in the care of immunocompromised patients and in consultation with a hematologist and/or an HCT physician. […] Immunosuppressive therapy using antithymocyte globulin (ATG) and cyclosporine is used as first-line therapy for patients with severe or very severe aplastic anemia (SAA or VSAA, respectively) who are older than 50 years (35-50 years in presence of comorbidities) and as second-line therapy in younger patients with SAA or VSAA if a human leukocyte antigen (HLA)matched sibling donor is not available.

• #61

  https://www.theaat.org.uk/treatment-with-ciclosporin

  It is very important that your blood pressure is checked regularly. Your kidney and liver blood tests and ciclosporin blood levels also need to be monitored regularly, alongside monitoring of your blood counts. […] One side effect of ciclosporin is the swelling and thickening of gums. […] The good news is that the side effects are temporary in nature and will disappear when you no longer need to take Ciclosporin.

• #62 Aplastic Anemia – Stanford Medicine Children’s Health

  https://www.stanfordchildrens.org/en/services/stem-cell-transplantation/conditions/aplastic-anemia.html

  Before a stem cell transplant, your child will receive therapy to eliminate their immune system to permit the new donor stem cells to grow. Patients usually receive anti-thymocyte globulin (ATG) and cyclophosphamide to suppress their immune system. At Stanford, we use a tailored dose of ATG to maximize its benefit (decreasing the risks of graft rejection and graft-versus-host disease) while minimizing toxicity. […] Your child will likely be discharged from the hospital 40 to 50 days after transplantation when his or her blood and immune systems are functioning at healthy levels.

• #63 Aplastic anaemia treatment – Leukaemia Foundation

  https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/aplastic-anaemia/treatment/

  An allogeneic (donor) stem cell transplant may be recommended as a curative option for younger people. […] Supportive therapies are also important. Blood transfusions are often required to replace circulating blood cells while antibiotics may be used to treat infections. Patients undergoing supportive therapies need regular monitoring to detect any change in their condition. […] People with aplastic anaemia may show residual damage in their bone marrow following treatment despite normal blood counts. Unfortunately some patients who are successfully treated for aplastic anaemia relapse with their disease. Generally the relapse of aplastic anaemia is not the result of re-exposure to the original trigger of the disease. Relapse can occur as a result of pregnancy or when the immune system is heavily challenged.

• #64 Aplastic anaemia treatment – Leukaemia Foundation

  https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/aplastic-anaemia/treatment/

  An allogeneic (donor) stem cell transplant may be recommended as a curative option for younger people. […] Supportive therapies are also important. Blood transfusions are often required to replace circulating blood cells while antibiotics may be used to treat infections. Patients undergoing supportive therapies need regular monitoring to detect any change in their condition. […] People with aplastic anaemia may show residual damage in their bone marrow following treatment despite normal blood counts. Unfortunately some patients who are successfully treated for aplastic anaemia relapse with their disease. Generally the relapse of aplastic anaemia is not the result of re-exposure to the original trigger of the disease. Relapse can occur as a result of pregnancy or when the immune system is heavily challenged.

• #65

  https://haematologica.org/article/view/9081

  The majority of patients (n=264) received hATG plus CyA. […] Following treatment with hATG/CyA, data regarding the cyclosporine taper were available for 194 patients. […] For the cohort of 314 patients and for the subset treated with hATG/CyA, median time to initial response was six months (range: 3-48 months; IQR: 3-12 months). […] The quality of response to hATG/CyA was good, with 59.8% (95%CI: 53.7,65.8) achieving a CR. […] The estimated probability of sustained response for all subjects was 94% (95%CI: 89,96) at 24 months and 83% (95%CI: 76,88) at 60 months. […] EFS was superior for patients receiving second-line treatment with HSCT compared with IST even after adjusting for age, gender, time from initial IST to second treatment, and lymphocytopenia.

• #66 Aplastic anaemia treatment – Leukaemia Foundation

  https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/aplastic-anaemia/treatment/

  A child treated with immunosuppressive treatment has around an 80 percent chance of responding to treatment and requiring no further treatment options. A child who undergoes a donor transplant from a matched sibling has around a 90 percent chance at cure. […] Your doctor or your child’s treating doctor will discuss with you treatment options in the occurrence of relapse.

• #67

  https://haematologica.org/article/view/9081

  The majority of patients (n=264) received hATG plus CyA. […] Following treatment with hATG/CyA, data regarding the cyclosporine taper were available for 194 patients. […] For the cohort of 314 patients and for the subset treated with hATG/CyA, median time to initial response was six months (range: 3-48 months; IQR: 3-12 months). […] The quality of response to hATG/CyA was good, with 59.8% (95%CI: 53.7,65.8) achieving a CR. […] The estimated probability of sustained response for all subjects was 94% (95%CI: 89,96) at 24 months and 83% (95%CI: 76,88) at 60 months. […] EFS was superior for patients receiving second-line treatment with HSCT compared with IST even after adjusting for age, gender, time from initial IST to second treatment, and lymphocytopenia.

• #68 Caring For Aplastic Anemia Patients In NJ, CT, and MD | Regional Cancer Care Associates

  https://www.regionalcancercare.org/hematology/malignant-blood-disorders/aplastic-anemia/

  Aplastic anemia can occur at any time throughout ones life, and symptoms can range from mild to severe. […] We treat a variety of conditions, such as aplastic anemia, using state-of-the-art therapies. […] If the cause is unknown, physicians may conduct further testing to rule out other types of anemia. From there, they can work to treat symptoms and return the body to a healthy state. […] To properly diagnose this condition, physicians take a thorough history, perform a comprehensive medical examination, and then employ tests including: […] A complete blood count can determine if a patients red blood cell levels are low, which could indicate aplastic anemia and signal a need for further blood tests. […] Using the latest advances in medicine, we can treat aplastic anemia in many ways, including with: Medication, Blood transfusion, Bone marrow transplant. […] To learn more about our patient-centered approach to aplastic anemia, request an appointment with us today.

• #69 Aplastic Anemia Drugs and Treatments | Aplastic Anemia and MDS International Foundation (AAMDSIF)

  https://www.aamds.org/aplastic-anemia/drugs-treatments

  More drugs and treatments are available to treat bone marrow failure disease than ever before. […] Alemtuzumab is in clinical trials for treating aplastic anemia. […] Androgens are sometimes used to treat aplastic anemia and PNH. […] ATG is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. […] A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia will receive at least one blood transfusion. […] A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT). The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure.

• #70 Aplastic Anemia: Practice Essentials, Background, Etiology

  https://emedicine.medscape.com/article/198759-overview

  Nonpharmacologic management of aplastic anemia includes the following: Supportive care; Blood transfusions with blood products that have undergone leukocyte reduction and irradiation; Hematopoietic cell transplantation. […] The British Society for Standards in Haematology has issued guidelines on diagnosis and management of aplastic anemia in adults and pediatric patients. […] Specific therapies, including hematopoietic cell transplantation (HCT) and immunosuppressive regimens, can be curative, particularly in cases with an identical HLA family donor, where overall survival probability is as high as 90%. […] In cases of immunosuppression, relapse and late clonal disease are risks. […] Eltrombopag and improved hematopoiesis in refractory aplastic anemia. […] Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. […] Upfront eltrombopag monotherapy induces stable hematologic remission in pediatric patients with nonsevere idiopathic aplastic anemia. […] High-dose romiplostim accelerates hematologic recovery in patients with aplastic anemia refractory to eltrombopag.

• #71 Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia.html

  Fred Hutch Cancer Center offers comprehensive consultations and treatments for aplastic anemia and other blood disorders. […] This means that people who have been diagnosed with aplastic anemia will find more treatment options at here than might be found elsewhere, including one of many clinical studies being conducted at Fred Hutch and UW Medicine. […] If your condition requires a bone marrow transplant, you should know that the Fred Hutch Blood and Marrow Transplant Program at is a leader in transplants. […] Aplastic anemia can be mild, moderate, or severe. Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with […] Blood transfusions and/or medications […] Immunosuppressive drugs […] Growth factors. […] For some people, taking part in a clinical study may be the best treatment choice. […] Many patients at Fred Hutch receive promising therapies by taking part in clinical trials. […] We offer more active clinical trials than anywhere else, which means more treatment options for patients like you.

• #72 Aplastic Anemia | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/aplastic-anemia.html

  Fred Hutch Cancer Center offers comprehensive consultations and treatments for aplastic anemia and other blood disorders. […] This means that people who have been diagnosed with aplastic anemia will find more treatment options at here than might be found elsewhere, including one of many clinical studies being conducted at Fred Hutch and UW Medicine. […] If your condition requires a bone marrow transplant, you should know that the Fred Hutch Blood and Marrow Transplant Program at is a leader in transplants. […] Aplastic anemia can be mild, moderate, or severe. Mild or moderate aplastic anemia is serious but usually doesn’t require hospitalization and may be treated with […] Blood transfusions and/or medications […] Immunosuppressive drugs […] Growth factors. […] For some people, taking part in a clinical study may be the best treatment choice. […] Many patients at Fred Hutch receive promising therapies by taking part in clinical trials. […] We offer more active clinical trials than anywhere else, which means more treatment options for patients like you.

• #73

  https://link.springer.com/article/10.1007/s12185-024-03713-3

  The treatment of aplastic anemia (AA) has significantly advanced in the last 50 years, evolving from a fatal condition to one where survival rates now exceed 8085%. Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) have become the primary treatments, with the latter widely adopted due to factors like the scarcity of compatible donors, patient age, comorbidities, and limited HSCT access. […] A therapy breakthrough was the introduction of antithymocyte globulin (ATG), with its effectiveness further boosted by cyclosporine. […] A major turning point was combining immunosuppression with stem cell stimulation, surpassing the efficacy of IST alone. […] Thrombopoietin receptor agonists represented a significant advancement. […] Horse ATG is the preferred formulation combined with cyclosporine and eltrombopag. […] This progress in AA treatment offers improved outcomes for patients afflicted with this once-lethal disease.

• #74 What to Know About the Prognosis of Aplastic Anemia in Adults

  https://www.healthline.com/health/iron-deficiency-anemia/aplastic-anemia-prognosis-in-adults

  The treatment you receive for AA can depend on things like how severe your AA is as well as your age and overall health. […] Immunosuppressive therapy can help stop your immune system from attacking stem cells in your bone marrow. Younger people with AA who are in good health may be eligible for a stem cell transplant. It uses stem cells from a healthy matched donor. […] A 2018 study also notes that a stem cell transplant could also be considered in older individuals whose AA has not responded to immunosuppressive therapy and are in good overall health. […] Other potential treatments for AA include: transfusions to supply healthy RBCs or platelets, medications that stimulate the bone marrow to make more blood cells, antibiotics or antifungals to prevent or treat infections. […] The treatment for AA can include a stem cell transplant or immunosuppressive therapy. Like outlook, treatment choices depend on your age, overall health, and AA severity. In some cases, a stem cell transplant cures AA.

• #75 Acquired Aplastic Anemia Therapies: Immunosuppressive Therapy Versus Alternative Donor Hematopoietic Cell Transplantation | Wirk | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1264/818

  Immunosuppressive therapy for acquired severe aplastic anemia improves pancytopenia but has a significant risk of relapse (40%) and clonal evolution to myeloid neoplasms (15%), especially in patients older than 40. […] Current guidelines recommend a matched sibling donor (MSD) allogeneic hematopoietic cell transplantation (HCT) for severe and very severe aplastic anemia (SAA/VSAA) patients 40 years or younger. Patients older than 40 receive immunosuppressive therapy (IST) with eltrombopag (EPAG), horse anti-thymocyte globulin (hATG), and cyclosporine (CSA). Alternative donor allogeneic HCT is reserved for those who fail a cycle of IST. […] Although IST helps improve pancytopenia, the response is not durable, and the risk of clonal disease persists. […] Current treatment guidelines for SAA/VSAA recommend MSD allogeneic HCT for fit patients 40 years or younger. Patients over 40 receive IST with EPAG, hATG, and CSA regardless of matched donor availability.

• #76 Acquired Aplastic Anemia Therapies: Immunosuppressive Therapy Versus Alternative Donor Hematopoietic Cell Transplantation | Wirk | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1264/818

  Immunosuppressive therapy for acquired severe aplastic anemia improves pancytopenia but has a significant risk of relapse (40%) and clonal evolution to myeloid neoplasms (15%), especially in patients older than 40. […] Current guidelines recommend a matched sibling donor (MSD) allogeneic hematopoietic cell transplantation (HCT) for severe and very severe aplastic anemia (SAA/VSAA) patients 40 years or younger. Patients older than 40 receive immunosuppressive therapy (IST) with eltrombopag (EPAG), horse anti-thymocyte globulin (hATG), and cyclosporine (CSA). Alternative donor allogeneic HCT is reserved for those who fail a cycle of IST. […] Although IST helps improve pancytopenia, the response is not durable, and the risk of clonal disease persists. […] Current treatment guidelines for SAA/VSAA recommend MSD allogeneic HCT for fit patients 40 years or younger. Patients over 40 receive IST with EPAG, hATG, and CSA regardless of matched donor availability.

• #77 Get Aplastic Anemia Treatment | Cleveland Clinic

  https://my.clevelandclinic.org/services/aplastic-anemia-treatment

  Cleveland Clinic has a dedicated team of healthcare providers that focuses on treating bone marrow failures. We explore all treatment options to make sure you get the best possible results and a chance to live a long, healthy life. […] No two cases of aplastic anemia are alike. Thats why we make sure you get a diagnosis and treatment personalized to your needs. […] Our providers lead research studies and clinical trials for aplastic anemia and other bone marrow conditions. […] Your provider may prescribe medications to treat aplastic anemia by suppressing your immune system (immunosuppressants) and stimulating blood cell production (bone marrow stimulants) or by treating infections (antibiotics). […] During a blood transfusion, you receive donated blood to help restore your blood cells. […] A successful transplant may cure aplastic anemia in some people. […] Cleveland Clinic providers believe your emotional well-being matters as much as the physical.

Zobacz więcej