Abstract
Objective The therapeutic approach for transfusion-independent non-severe aplastic anemia (NSAA) is undetermined. This study aimed to investigate the efficacy of immunosuppressive therapy (IST) for NSAA. Methods We retrospectively reviewed 42 consecutive patients with transfusion-independent NSAA. NSAA was further divided into two stages according to the degree of cytopenia. Progression was defined as transition to a transfusion-dependent state. Results Twelve (29%) patients received IST with cyclosporine A (CsA). Eleven (26%) patients became transfusion-dependent. In all patients, a univariate analysis revealed that a low hemoglobin level (p=0.006) and low reticulocyte count (p=0.005) were associated with a high probability of progression. The estimated transfusion-free survival (TFS) was significantly prolonged by IST among patients with advanced-stage NSAA (p=0.002), while IST did not reduce the incidence of progression in the overall cohort (p=0.349). In the non-IST group, an advanced clinical stage was significantly associated with progression (p=0.003). In contrast, the clinical stage was not related to progression in the IST group (p=0.318). None of the patients had to discontinue treatment with CsA due to renal failure. Conclusion IST is expected to be effective in patients with advanced-stage NSAA.