title: Outcome of a novel immunosuppressive strategy of cyclosporine, levamisole and danazol for severe aplastic anemia.
abstract: 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.
authors.affiliation: Severe Aplastic Anemia Studying Program, State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science and Peking Union Medical College, 288 Nanjing Road, Tianjin, 300020, People's Republic of China, wang1987min1987@126.com.
authors.forename: Min
authors.initials: M
authors.lastname: Wang
authors.forename: Xingxin
authors.initials: X
authors.lastname: Li
authors.forename: Jun
authors.initials: J
authors.lastname: Shi
authors.forename: Yingqi
authors.initials: Y
authors.lastname: Shao
authors.forename: Meili
authors.initials: M
authors.lastname: Ge
authors.forename: Jinbo
authors.initials: J
authors.lastname: Huang
authors.forename: Zhendong
authors.initials: Z
authors.lastname: Huang
authors.forename: Jing
authors.initials: J
authors.lastname: Zhang
authors.forename: Neng
authors.initials: N
authors.lastname: Nie
authors.forename: Yizhou
authors.initials: Y
authors.lastname: Zheng
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D060965:Clonal Evolution; D016572:Cyclosporine / Q000008:administration & dosage; D003613:Danazol / Q000008:administration & dosage; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D007978:Levamisole / Q000008:administration & dosage; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012008:Recurrence; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D007978:Levamisole; D016572:Cyclosporine; D003613:Danazol