Aplastic anemia medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]; Associate Editor(s)-In-Chief: Priyamvada Singh, M.D. [3]

Overview

Treatments for aplastic anemia include blood transfusions, blood and marrow stem cell transplants, and medications.

Medical Therapy

• Treatments for aplastic anemia include blood transfusions, blood and marrow stem cell transplants, and medications.
• These treatments can prevent or limit complications, relieve symptoms, and improve quality of life.
• Blood and marrow stem cell transplants may cure the disorder in some people who are eligible for a transplant.
• Removing a known cause of aplastic anemia, such as exposure to a toxins such as benzene or chloramphenicol, may cure the condition.
• People who have mild or moderate aplastic anemia may not need treatment as long as the condition does not get worse.
• People who have severe aplastic anemia need medical treatment right away to prevent complications.
• People who have very severe aplastic anemia need emergency medical care in a hospital. Very severe aplastic anemia can be fatal if it's not treated right away.
• Blood transfusions can help keep blood cell counts at acceptable levels.
• Blood transfusions help relieve the symptoms of aplastic anemia, but they are not a permanent treatment.
• Blood and marrow stem cell transplants may cure aplastic anemia in people who can have this type of treatment.
  • The transplant works best in children and young adults with severe aplastic anemia who are in good health and who have matched donors.
  • Older people may be less able to handle the treatments needed to prepare the body for the transplant. They are also more likely to have complications after the transplant.
• Medications are used to:

1. Stimulate the bone marrow

• Erythropoietin and colony-stimulating factors can stimulate the bone marrow.
• Eltrombopag is used to stimulate platelet production and has been shown in a recent trial to improve the hematologic response rate when given in combination with anti-thymocyte globulin (ATG) plus cyclosporine, compared to ATG plus cyclosporine alone.
• These medicines have some risks. However, if this treatment works well, it can help avoid the need for blood transfusions.

2. Suppress the immune system

• Immunosuppressive therapy is recommended for patients with non-severe aplastic anaemia who are transfusion-dependent.
• Immunosuppressive therapy is recommended for patients with severe or very severe disease who are >40 years old.
• Immunosuppressive therapy is recommended for younger patients with severe or very severe disease who do not have an HLA-identical sibling donor.
• Anti-thymocyte globulin is a powerful immunosuppressive drug used in severely neutropenic patients and requires very careful monitoring, prophylaxis, and treatment of fevers.
• Anti-thymocyte globulin must only be given in the inpatient setting.
• Cyclosporine should be continued for at least 12 months after achieving maximal hematological response, followed by a very slow tapering, to reduce the risk of relapse.

3. Prevent and treat infections

• It may take a few months to notice the effects of anti-viral, anti-fungal, and anti-bacterial agents. Most often, as blood cell counts rise, symptoms lessen.
• Blood cell counts in people who respond well to these medicines usually do not reach normal levels.
• However, the blood cell counts often are high enough to allow people to do their normal activities.

Supportive care in treatment of aplastic anemia (DONOT EDIT)

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Supportive Care Prophylactic platelet transfusions should be given when the platelet count is <10 x 109/L (or <20 x 109/L in the presence of fever). Irradiated blood products should be given routinely to all patients having antithymocyte globulin (ATG) treatment. Transfusion of irradiated granulocyte transfusions may be considered in patients with life-threatening neutropenic sepsis. The routine use of recombinant human erythropoietin (rHuEPO) in aplastic anaemia is not recommended. A short course of granulocyte colony-stimulating factor (G-CSF) may be considered for severe systemic infection that is not responding to intravenous antibiotics and anti-fungal drugs, but should be discontinued after 1 week if there is no increase in the neutrophil count. Prophylactic antibiotic and antifungal drugs should be given to patients with neutrophil count <0.2 x 109/L. Systemic antifungal therapy should be introduced into the febrile neutropenia regimen early if fevers persist. Iron chelation therapy should be considered when the serum ferritin is >1000 µg/L. Patients with iron overload after successful HSCT should undergo venesection.

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Specific Treatment of Aplastic Anaemia: General Comments (DONOT EDIT)

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General Comments Infection or uncontrolled bleeding should be treated first before giving immunosuppressive therapy. This also applies to patients scheduled for bone marrow transplant (BMT), although it may sometimes be necessary to proceed straight to BMT in the presence of severe infection as a BMT may offer the best chance of early neutrophil recovery. Haemopoietic growth factors, such as rHuEPO or G-CSF, should not be used on their own in newly diagnosed patients in an attempt to 'treat' the aplastic anaemia. Prednisolone should not be used to treat patients with aplastic anaemia because it is ineffective and encourages bacterial and fungal infection.

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Specific Treatment of Aplastic Anaemia: Human Leucocyte Antigen (HLA)-identical Sibling Donor Transplantation(DONOT EDIT)

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Human Leucocyte Antigen (HLA)-identical Sibling Donor Transplantation Allogeneic BMT from an HLA-identical sibling donor is the initial treatment of choice for newly diagnosed patients if they have severe or very severe aplastic anaemia, are <40 years old, and have an HLA-compatible sibling donor. Patients with Fanconi anaemia and other types of inherited aplastic anaemia need special consideration and should not follow recommendations made in this guideline. There is no indication for using irradiation-based conditioning regimens for patients undergoing HLA-identical sibling BMT for aplastic anaemia. The recommended source of stem cells for transplantation in aplastic anaemia is bone marrow. Fertility is well preserved after high dose cyclophosphamide conditioning in BMT for aplastic anaemia, and patients should be given appropriate contraceptive advice to prevent unwanted pregnancy. Until longer term data is available in patients receiving fludarabine-based regimens, cryopreservation of sperm and oocytes should be planned.

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Specific Treatment of Aplastic Anaemia: Immunosuppressive Therapy: ATG and Ciclosporin (DONOT EDIT)

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Immunosuppressive Therapy: ATG and Ciclosporin Immunosuppressive therapy is recommended for: patients with non-severe aplastic anaemia who are transfusion dependent patients with severe or very severe disease who are >40 years old, and younger patients with severe or very severe disease who do not have an HLA-identical sibling donor. The current first line immunosuppressive treatment for aplastic anemia is horse ATG (ATG_ATGAM) combined with ciclosporin. ATG is a powerful immunosuppressive drug and its use in severely neutropenic patients requires very careful monitoring, prophylaxis and treatment of fevers, and adequate (and sometimes intensive) platelet transfusional support. ATG must only be given as an in-patient: Ciclosporin should be continued for at least 12 months after achieving maximal haematological response, followed by a very slow tapering, to reduce the risk of relapse.

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Specific Treatment of Aplastic Anaemia: Matched Unrelated Donor Bone Marrow Transplantation (MUD BMT): ATG and Ciclosporin (DONOT EDIT)

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Matched Unrelated Donor Bone Marrow Transplantation (MUD BMT) MUD BMT may be considered when a patient has a fully matched donor, is <50 years old (or 50–60 years old with good performance status), has failed at least one course of ATG and ciclosporin, and has severe aplastic anaemia. There is currently insufficient data on outcome for patients >60 years of age. The optimal conditioning regimen for MUD BMT is uncertain, but currently a fludarabine, non–irradiation-based regimen is favoured for younger patients.

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Specific Treatment of Aplastic Anaemia: Trial Therapy or Clinical Research Protocols (DONOT EDIT)

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Trial Therapy or Clinical Research Protocols The use of high dose cyclophosphamide without stem cell support is not recommended in the treatment of aplastic anaemia. Mycophenolate mofetil (MMF) does not appear to be effective in the treatment of aplastic anaemia. The routine use of long term G-CSF, or other haemopoietic growth factors, after ATG and ciclosporin is not recommended outside the setting of prospective clinical trials.

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Specific Treatment of Aplastic Anaemia: Management of Aplastic Anaemia in the Presence of an Abnormal Cytogenetic Clone (DONOT EDIT)

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Management of Aplastic Anaemia in the Presence of an Abnormal Cytogenetic Clone The presence of an abnormal cytogenetic clone in the presence of an otherwise typical aplastic anaemia does not necessarily equate with a diagnosis of myelodysplastic syndromes (MDS) or acute myeloid leukaemia (AML), as abnormal cytogenetic clones occur in up to 12% of patients with aplastic anaemia. The presence of monosomy 7 is more often sinister with a high risk of transformation to MDS or AML.

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Specific Treatment of Aplastic Anaemia: Management of Aplastic Anaemia in the Presence of an Abnormal Cytogenetic Clone (DONOT EDIT)

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Management of Aplastic Anaemia in the Presence of an Abnormal Cytogenetic Clone Management of Patients with Aplastic Anaemia Who Have a Significant PNH Clone, Resulting in Clinical and/or Laboratory Evidence of Haemolysis Small PNH clones, in the absence of evidence of haemolysis, occur in up to 50% of patients with aplastic anaemia. ATG is not recommended if there is a history of PNH-associated thrombosis. All patients who are not transplanted should be screened for PNH by flow cytometry every 12 months.

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Specific Treatment of Aplastic Anaemia: Management of Aplastic Anaemia in Pregnancy(DONOT EDIT)

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Management of Aplastic Anaemia in Pregnancy There is a high risk (33%) of relapse of aplastic anaemia in pregnancy. Supportive care is the mainstay of treatment in pregnancy. the platelet count should be maintained >20 x 109/L, if possible. It is safe to use ciclosporin in pregnancy.

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