Abstract
Background Acquired aplastic anemia (AA) is an immune-mediated hematopoietic disorder. It is characterized by pancytopenia and hypocellular bone marrow. The first-line treatment approach to AA includes immunosuppressive treatment (IST) using the combination of antithymocyte globulin (ATG) and cyclosporine A. Those patients who do not respond to this first-line treatment or have refractory disease succumb to bleeding or infections within five years following their diagnosis even after IST. Romiplostim, a thrombopoietin (TPO) receptor antagonist, promotes trilineage hematopoiesis in patients with AA. A retrospective study assessed the safety and effectiveness of romiplostim, as monotherapy among patients with refractory AA in Indian settings. Methods The case record forms of patients diagnosed with refractory AA and receiving treatment with weekly doses of 250 mg romiplostim and concomitant medications were reviewed at Yashoda Hematology Clinic, India. The primary outcome was to evaluate the increase in platelet count/percentage and the safety of romiplostim in these patients. The secondary outcomes were a change in total leucocyte count (TLC) and hemoglobin (Hb) levels from baseline after romiplostim therapy. Results Data from 28 patients diagnosed with AA and having received romiplostim subcutaneously in a dose of 250 mg weekly were analyzed. There was a significant improvement in platelet count which increased by 0.064 × 10(9)/L units (95% CI: 0.021-0.107) at a 100-day interval from the initial measurement. The TLCs also increased by 0.022 × 10(9)/L units (95% CI: 0.002-0.042). The mean Hb levels increased from 7.61 gm/L to 13.38 gm/L (95% CI, p < 0.001). No severe adverse events were reported. Conclusion Romiplostim demonstrated clinically significant outcomes with a favorable safety profile in patients with refractory AA.