Therapeutic roles of hematopoietic growth factors: treatment with a thrombopoietin mimetic (Eltrombopag) and cyclosporin A in aplastic anemia

Background Eltrombopag (EPAG) was recently approved for aplastic anemia (AA). The recommended regimen is triple agents, EPAG, anti-thymocyte globulin, and cyclosporin A (CsA). Anti-thymocyte globulin is not available in many centers and has serious adverse effects. Aim This study assessed, for the first time, two-agent therapies comprising EPAG+CsA in patients with AA. Patients and methods Group A included 33 patients with AA who were treated with EPAG+CsA and prospectively recruited. Their health-related quality of life (HRQoL) was assessed. All parameters were evaluated at enrollment and after 2−3 months. Bone marrow aspirate was done after 6 months. Another 33 patients who received CsA only (group B) were retrospectively recruited for comparison. Results Both groups were matched regarding demographic and baseline hematologic profile. Significant increment and reduction, after treatment, of absolute neutrophil count and platelets, and transfusion requirements, were respectively seen in group A compared with group B, whereas mean platelet volume was significantly increased in group A only (0.025). The overall response rate and complete remission rate were 72.7 vs. 45.5% and 15.1 vs. 3% in groups A and B, respectively. Significant improvement of the total RAND scoring was found in HRQoL items of group A; however, the social functioning score did not improve significantly. Therapeutic response to EPAG+CsA was an independent effector of HRQoL. After 6 months, one patient had acute leukemia and another one developed fibrosis. Conclusions A ramp-up, short-duration, therapy with EPAG+CsA in AA is as effective as fixed-dose triple agents, with lower risk of disease progression or fibrosis.