Update on Management of Aplastic Anemia

Compiled for you by PharmaWrite

Presented by LeAnne Kennedy, PharmD, BCOP
Wake Forest Baptist Health, Wake Forest, NC

Key Takeaways

• Severe aplastic anemia is a curable disease that requires immunosuppressive therapy and/or stem cell transplantation depending on age and availability of matched donors for transplantation
• Bone marrow transplantation has improved 5-year overall survival compared to peripheral blood transplantation
• Pharmacists can play a key role in counseling and managing therapy for treatment related toxicities

This session focused on the role of immunosuppressive therapies and stem cell transplantation in the treatment of aplastic anemia. Aplastic anemia is a curative disease that has a bimodal age distribution with two peaks, young adults and the elderly. Information from the Bone Marrow Transplantation Clinical Trials Network (Pulsipher 2011) and the 2016 British Society for Standards in Haematology Guidelines (Killick 2016) for diagnosis and management were presented.

Standard first-line treatment consists of immunosuppressive therapy (ie, anti-thymocyte globulin), cyclosporine (including therapeutic drug monitoring), and glucocorticoids for older patients, followed by stem cell transplantation if relapse occurs. Eltrombopag is effective at increasing platelets, white blood cells, and hemoglobin without an increased risk of bone marrow fibrosis in patients treated with standard therapy having persistently low platelets. The use of stem cell transplantation is also effective in the treatment and management of aplastic anemia, particularly for younger patients, and bone marrow transplantation has improved 5-year overall survival compared to peripheral blood transplantation. Matched related donor allotransplants are associated with less treatment-related toxicities vs matched unrelated donors, which carries an increased risk for the most concerning complication of graft vs host disease.

The speaker concluded by presenting data on alternative cell sources, including cord blood transplantation (3-year overall survival of 38%) and haplo-identical transplantation (with a 1-year survival of 67.1%).

This session summary was prepared by Ginah Nightingale, PharmD, BCOP, a member of the PharmaWrite Oncology Working Group.

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