Role of Arsenic Trioxide in the Management of Aplastic Anemia

Aplastic anemia is a common problem in the developing world with therapeutic challenges as most of the patients are non-affording to standard care owing to resource constraints. We present the results of an open label single arm, non-randomized, single center, prospective phase II trial of the compassionate use of arsenic trioxide in patients refractory to ATG or unable to afford the standard of care therapy. The study was prematurely terminated at eighth week due to non-response in 100% of patients and death in two patients. The results are contradictory to what is already published and needs validation in a multicentric setting.