Management of Aplastic Anemia

Acquired aplastic anemia (AA) is a rare but potentially fatal condition characterized by pancytopenia and a hypocellular marrow. Its prevalence is significantly higher in Asia, including India. The major challenge in treating AA in developing countries is the long interval between disease onset and diagnosis and from diagnosis to treatment. So early recognition and referral to a specialist hematology center is critical because timely intervention with stem cell transplant (SCT) or immunosuppressive therapy (IST) significantly improves survival. In the last few decades, the survival of the AA has been improved substantially due to the availability of better supportive care and definitive management options in both children and adults. The definitive management of AA includes allogenic hematopoietic stem cell transplant (HSCT) and immunosuppressive therapy (IST), including anti-thymocyte globulin with Cyclosporine-A. The integration of Eltrombopag, an oral TPO-RA, into first-line IST is a recent development in the management of AA. Patients who recover from AA require regular follow-up, as around 10–15% of acquired AA patients may develop clonal evolution to develop conditions like myelodysplastic syndrome (MDS), acute leukaemia. With the addition of eltrombopag to IST, growing experience with haplo-HSCT, and refined diagnostics enabling accurate classification, aplastic anemia has become a largely curable disease, if diagnosed and treated early.