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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.
AuthorsCheng-Juan Luo, Yi-Jin Gao, Jing-Yan Tang, Xiao-Hua Zhu, Hui-Liang Xue, Feng-Juan Lu, Ci Pan, Hua Jiang, Chang-Ying Luo, Qi-Dong Ye, Min Zhou, Jing Chen
JournalJournal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 36 Issue 5 Pg. 374-8 (Jul 2014) ISSN: 1536-3678 [Electronic] United States
PMID24807005 (Publication Type: Journal Article)
Chemical References
  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclosporine
Topics
  • Adolescent
  • Anemia, Aplastic (drug therapy, mortality)
  • Antilymphocyte Serum (therapeutic use)
  • Child
  • Child, Preschool
  • Cyclosporine (therapeutic use)
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Humans
  • Immunosuppressive Agents (therapeutic use)
  • Infant
  • Male
  • Prognosis
  • Retrospective Studies
  • Secondary Prevention
  • Survival Rate

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