A promising immunosuppressive strategy of cyclosporine alternately combined with levamisole is highly effective for moderate aplastic anemia.

The appropriate management of patients with moderate aplastic anemia (mAA) remains to be unclear and controversial. A cohort of 118 patients with mAA received a novel immunosuppressive strategy of cyclosporine alternately combined with levamisole (CSA and LMS regimen), which included 42 newly diagnosed and 76 chronic (disease duration >6 months) cases. CSA and LMS regimen was orally administrated with the initial dose of CSA 3 mg/kg per day in adults or 5 mg/kg per day in children, and LMS 150 mg per day in adults or 2.5 mg/kg per day in children, continued for 12 more months after achieving maximal hematologic response, followed by a slow tapering. The overall response rates were of 100 and 86.8 % for newly diagnosed and chronic group, respectively. The 24-month progression-free survival were 95.2 % (95 % confidence intervals [CI], 85.9-100 %) and 93.6 % (95 % CI, 86.9-100 %) for newly diagnosed and chronic group, respectively (P = 0.50). The 2-year event-free survival for the patients in newly diagnosed group (86.6 %; 95 % CI, 70.4-100 %) was superior to that in chronic group (57.0 %; 95 % CI, 43.5-70.4 %, P = 0.001). To date, 11 patients relapsed and no patients evolved to clonal disorders. Thus, CSA and LMS regimen represents a promising immunosuppressive strategy for mAA.