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.
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Authors | Cheng-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 |
Journal | Journal of pediatric hematology/oncology
(J Pediatr Hematol Oncol)
Vol. 36
Issue 5
Pg. 374-8
(Jul 2014)
ISSN: 1536-3678 [Electronic] United States |
PMID | 24807005
(Publication Type: Journal Article)
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Chemical References |
- Antilymphocyte Serum
- Immunosuppressive Agents
- Cyclosporine
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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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