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Aplastic anemia is a blood disorder caused by failure of the bone marrow to make enough new blood cells. Bone marrow is a sponge-like tissue inside the bones that makes stem cells that develop into red blood cells, white blood cells, and platelets. Symptoms may include fatigue, weakness, dizziness, shortness of breath, frequent infections, and bleeding. Aplastic anemia can lead to other health concerns such as an irregular heartbeat, an enlarged heart, and heart failure. It can be caused by injury to blood stem cells due to exposure to certain drugs, chemotherapy, congenital disorders, drug therapy to suppress the immune system, pregnancy, radiation therapy, or toxins such as benzene or arsenic. When the cause is unknown, it is referred to as idiopathic aplastic anemia. In about half of all cases, no cause can be found. The blood disorder can be acute or chronic. Treatment may consist of supportive care only, blood transfusions, medicines to suppress the immune system, or hematopoietic cell transplantation.
The British Committee for Standards in Haematology recommends treating infection or uncontrolled bleeding before administering immunosuppressive therapy, including in patients scheduled for HCT. In the presence of severe infection, however, it may be necessary to proceed directly to HCT to provide the patient with the best chance for early neutrophil recovery. The Pediatric Haemato-Oncology Italian Association recommends HCT from a matched sibling donor for severe aplastic anemia, and 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. Independent of response or degree of response, risks include relapse and late-onset clonal disease, such as paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome (MDS), or leukemia. [14, 38, 39, 40, 41]
Pregnant women with aplastic anemia have a 33% risk of relapse. Provide supportive care in these patients, maintain the platelet count above 20 × 109/L, if possible, and consider administering cyclosporine.
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.
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.
Source: GARD Last updated on 05-01-20
The symptoms of aplastic anemia vary depending on how severe it is and how low blood counts are. Signs and symptoms may include:
Last updated on 05-01-20
A small number of people with aplastic anemia may spontaneously recover with supportive care; however, for most individuals, the condition worsens without identification and treatment of the underlying cause and/or treatment of the disease. Bone marrow transplant may cure the disease in children and young patients and has a 10 year survival rate of approximately 73%. For many, bone marrow transplant is not an option due to the risks and potential long-term side effects.
Last updated on 05-01-20
Treatment for aplastic anemia varies depending on the severity. While some individuals with mild to moderate aplastic anemia may not require treatment, for others, treatment may include:
Blood and marrow stem cell transplants may cure aplastic anemia in some instances. This treatment option works best in children and young adults with severe aplastic anemia who are otherwise in good health.
For patients with severe aplastic anemia who are under the age of 20 years, and those ages 20 to 50 years who are otherwise in good health the first option is the transplant when a sibling donor is available. For those who do not have an available sibling donor, the medication eltrombopag or eltrombopag plus immunesuppression therapy can be used.
For patients over 50 years of age, the decision is based on the patient’s overall health, and preferences, and treatment may include eltrombopag or eltrombopag plus immunesuppression therapy (horse anti-thymocyte globulin (ATG), cyclosporin A (CSA), and glucocorticoids). People older than 50 years old have more risks of having rejection with the transplant and have greater risks of treatment toxicity and early mortality.
Last updated on 05-01-20
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