title: ATG-Fresenius S combined with cyclosporine a: an effective immunosuppressive therapy for children with aplastic anemia.
abstract: For the first time, we conducted a 2-center retrospective study to show the efficacy of antithymocyte globulin (ATG)-Fresenius S plus cyclosporine treatment of children with severe aplastic anemia. From March 1997 to May 2011, a total of 124 patients (median age, 7.5 y; range, 1.5 to 16 y) from 2 centers with acquired AA treated with an immunosuppressive therapy (IST) regimen, consisting of ATG-Fresenius S (5 mg/kg per day for 5 d) and cyclosporine, were enrolled. The response rate was 55.6%. The median time between IST and response was 6 (0.5 to 18) months. After a median follow-up time of 29 (6 to 153) months, the rates of relapse and clonal evolution were 3.2% and 0.8%, respectively. Overall, 17 patients (13.7%) died in this study: 14 resulted from sepsis, 1 resulted from intracranial hemorrhage, 1 occurred after hematopoietic stem cell transplantation, and 1 resulted from clonal disease progression. The 5-year overall survival rate for the entire cohort was 74.7%. IST responders had a better survival rate (100%) than nonresponders (70.7%). The use of ATG-Fresenius S plus cyclosporine as a first-line immunosuppressive treatment appeared to be effective for children with severe aplastic anemia in our study. ATG-Fresenius S could be another option in the treatment arsenal, especially in countries where the other ATG products are harder to acquire.
authors.affiliation: *Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University †Children's Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
authors.forename: Cheng-Juan
authors.initials: CJ
authors.lastname: Luo
authors.forename: Yi-Jin
authors.initials: YJ
authors.lastname: Gao
authors.forename: Jing-Yan
authors.initials: JY
authors.lastname: Tang
authors.forename: Xiao-Hua
authors.initials: XH
authors.lastname: Zhu
authors.forename: Hui-Liang
authors.initials: HL
authors.lastname: Xue
authors.forename: Feng-Juan
authors.initials: FJ
authors.lastname: Lu
authors.forename: Ci
authors.initials: C
authors.lastname: Pan
authors.forename: Hua
authors.initials: H
authors.lastname: Jiang
authors.forename: Chang-Ying
authors.initials: CY
authors.lastname: Luo
authors.forename: Qi-Dong
authors.initials: QD
authors.lastname: Ye
authors.forename: Min
authors.initials: M
authors.lastname: Zhou
authors.forename: Jing
authors.initials: J
authors.lastname: Chen
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000401:mortality; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D011379:Prognosis; D012189:Retrospective Studies; D055502:Secondary Prevention; D015996:Survival Rate
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine