hematology and oncology
Aplastic Anemia
Treatment of Aplastic Anemia
Hematopoietic stem cell transplantation If transplantation is not an option, immunosuppression with equineantithymocyte globulinandcyclosporineand sometimeseltrombopag In aplastic anemia, Hematopoietic Stem Cell Transplantation can be curative and is the treatment of choice, particularly in younger patients with a matched donor. At diagnosis, siblings are evaluated for HLA (human leukocyte antigen) compatibility. In those patients unfit for transplant or lacking a donor, immunosuppressive treatment with equineantithymocyte globulin(ATG) combined withcyclosporineproduces overall response rates of approximately 60 to 80%. Allergic reactions and serum sickness may occur. Long-term relapse or clonal evolution to myeloid malignancy occurs in up to half of patients. , a thrombopoietin receptor agonist, has shown potential efficacy in clinical trials in the upfront and refractory settings with immunosuppression, and haploidentical bone marrow transplants are promising to expand access to Hematopoietic Stem Cell Transplantation ( 1.Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al.EltrombopagAdded to Immunosuppression in Severe Aplastic Anemia.N Engl J Med2022;386(1):11-23. doi:10.1056/NEJMoa2109965 2.DeZern A, Zahurak ML, Symons HJ, et al. Alternative donor BMT with post-transplantcyclophosphamideas initial therapy for acquired severe aplastic anemia [published online ahead of print, 2023 Apr 21].Blood2023;blood.2023020435. doi:10.1182/blood.2023020435 3.Winkler T, Fan X, Cooper J, et al: Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated witheltrombopag.Blood133(24):2575–2585, 2019. doi: 10.1182/blood.2019000478 