Stanozolol in Aplastic Anemia: Pharmacological mechanisms, clinical applications, and future perspectives

Aplastic anemia (AA) is a heterogeneous bone marrow failure syndrome with hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) as standard treatments. For patients failed to response to IST or unable to meet the HSCT, hematopoiesis stimulation regimes are recommended as sole or combination therapy, including recombinant human erythropoietin (rhEPO), thrombopoietin receptor agonists (TPO-RAs) as well as androgen (such as danazol, stanozolol, etc.). Based on clinical practice, danazol works well for patients with short telomeres, while the overall response was more profound in stanozolol management. Herein, this review summarized the pharmacology of stanozolol, and compared with other androgens, the mechanisms and clinical evidence were also reviewed. Stanozolol deserves more attention as an oral option in selected non-severe/chronic AA, while better comparative studies and biologic stratification are needed.