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What is the cause and treatment for aplastic anaemia?

Q: My cousin is suffering from aplastic anaemia for the past 8-9 months. He took some Alg therapy in the form of an injection. Even after 5 months of the therapy, the platelet counts did not improve. He often has to take blood transfusion. Doctors suggest to take Eprex injections weekly to help increase the counts. Is Eprex useful in such case? Is there any tested treatment of this disease?

A:Aplastic anaemia is a condition in which there is bone-marrow failure that is characterised by anaemia (low haemoglobin), leukopaenia (low white cell count) and thrombocytopaenia (low platelet count). The bone marrow is hypoplastic and shows large fat cells with marked reduction in normal blood forming cells. The problems due to this disease relate to infection (due to low white cell count) and bleeding (because of thrombocytopaenia). It is postulated that the disease arises due a defect in or damage to bone marrow stem cell, the cell which gives rise to all other blood cells. The condition is usually acquired and is presumed to be an autoimmune disease but it may also be secondary to some infections or exposure to drugs and chemicals. Treatment includes transfusions, treatment of infections and immunosuppressive therapy. The definitive treatment is a bone marrow transplant (BMT) from an HLA-matched sibling but this too has a rejection rate of about 10%. Immunosuppressive therapy is planned if BMT is not possible. This includes the use of steroids, ATG and CSA. The response to treatment is slow and may take upto 3 months after initiating therapy. Following this initial response, there is slow improvement. About 50% patients respond by 3 months after ATG administration, and about 75% respond by 6 months. Most patients improve and become transfusion independent, but bone marrow changes may persist. Relapse is common and patients often need continued immune suppression. Several studies have shown that the addition of cytokines (e.g. G-CSF, GM-CSF) may hasten the neutrophil recovery. The estimated 5-year survival of a patient on immunosuppression is about 75%. Erythropoietin (EPO) is a hormone produced by the kidney, which is the primary regulator of red blood cell formation (erythropoiesis). Eprex is recombinant human erythropoietin (r-HuEPO), which stimulates erythropoiesis. It is usually given to patients of chronic renal failure who have transfusion-dependent anaemia; anaemia in cancer patients due to the effect of chemotherapy; individuals wanting autologous transfusion; and some other patients of anaemia. However, in many patients with aplastic anaemia the shortage of stem cells is so severe that there is little left to stimulate, and EPO may have very little effect, if at all.


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