Outcome of a novel immunosuppressive strategy of cyclosporine, levamisole and danazol for severe aplastic anemia.

Treatment options for patients with severe aplastic anemia (SAA) in developing countries are limited. A cohort of 261 patients with SAA received a novel immunosuppressive strategy of cyclosporine alternately combined with levamisole plus danazol (CSA&LMS-based regimen), which included 70 VSAA and 191 moderate SAA [initial absolute neutrophil count (ANC) >200/μL] cases. The CSA&LMS-based regimen was administrated orally with an initial dose of CSA 3 mg/kg in adults and 5 mg/kg in children every other day, LMS 150 mg in adults and 2.5 mg/kg in children every other day, and danazol (5.0-10.0) mg/kg daily, continued for 12 more months, followed by slow tapering. The 6-month response rates were 24.3 and 52.9 % for VSAA and moderate SAA (P < 0.001), respectively. Univariate and multivariate analyses demonstrated that younger age, higher pretreatment absolute reticulocyte count and ANC were favorable factors for achieving response at 6 months. The estimated 5-year overall survival rates were 33.8 % (95 % CI 20.6-47 %) and 80.5 % (95 % CI 69.7-91.3 %) for VSAA and moderate SAA, respectively (P < 0.001). To date, nine patients relapsed, and six patients evolved to clonal disorders. Thus, CSA&LMS-based regimen may represent a promising immunosuppressive strategy for moderate SAA.