Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis

Bone marrow transplantation, antithymocyte globulin/cyclosporine and eltrombopag are recommended as first-line therapy of severe aplastic anemia (SAA). However, androgens could be considered as front-line treatment among any patients ineligible for better methods although unsatisfactory efficacy is presented.

This retrospective study aimed to evaluate response and survival rate of practical-based treatment with oxymetholone.

This constituted an analysis of patients receiving a diagnosis of acquired aplastic anemia (AA) at the age of 15 or over and receiving oxymetholone between January 2004 and December 2018. Propensity Score Analysis (PSA) 1:1 matching was performed, according to sex, age and interval from first symptom to treatment. The primary outcome was one-year overall response (OR).

Seventy-four patients were successfully matched by PSA. The 1-year OR of oxymetholone in the nonsevere AA (nSAA) and SAA/very severe AA (vSAA) groups was 54.1 and 13.5%, respectively (P <0.001). With median follow-up 2.7 years, the overall survival was 59.5% in nSAA and 37.8% in SAA/vSAA (P = 0.051). Median survival in nSAA and SAA/vSAA were 7.0 years and 1.8 years, respectively (P = 0.045). However, the responders of SAA/vSAA had longer survival than nonresponders of the nSAA group.

These results revealed longer survival among the responders of patients with AA, even in the SAA/vSAA group. However, close monitoring of therapeutic responses is still performed. Switching therapy is necessary when remission is undetected after 6 months of oxymetholone treatment.