Comparison of Haploidentical Hematopoietic Stem Cell Transplantation and Immunosuppressive Therapy for the Treatment of Acquired Severe Aplastic Anemia in Pediatric Patients.

Abstract	This study aimed to compare the efficacy and safety between haploidentical hematopoietic stem cell transplantation (HHCT) and immunosuppressive therapy (IST) for the treatment of pediatric acquired severe aplastic anemia (SAA). The clinical data of 28 children with SAA treated from June 2010 to October 2014 at our hospital were retrospectively reviewed. Of these patients, 18 were treated with HHCT and 10 with IST. The median follow-up time was 23.5 months (range, 3-52 months). There was no significant difference in overall survival rate between the HHCT group and the IST group (66.7% vs. 70%, P > 0.05). Graft-versus-host disease occurred in 83.3% (15/18) of the HHCT group, including 5 cases with grade III or higher. In comparison with IST, HHCT has similar efficacy and safety profiles in the treatment of pediatric SAA.
Authors	Yuan Zhang, Zhi Guo, Xiao-Dong Liu, Xue-Peng He, Kai Yang, Peng Chen, Hui-Ren Chen
Journal	American journal of therapeutics (Am J Ther) 2017 Mar/Apr Vol. 24 Issue 2 Pg. e196-e201 ISSN: 1536-3686 [Electronic] United States
PMID	26872138 (Publication Type: Comparative Study, Journal Article)
Chemical References	Antilymphocyte Serum Immunosuppressive Agents Myeloablative Agonists Cyclosporine Cyclophosphamide Vidarabine Mycophenolic Acid fludarabine Methylprednisolone Methotrexate
Topics	Adolescent Anemia, Aplastic (mortality, therapy) Antilymphocyte Serum (therapeutic use) Child Child, Preschool Cyclophosphamide (therapeutic use) Cyclosporine (therapeutic use) Female Graft vs Host Disease (prevention & control) Haplotypes Hematopoietic Stem Cell Transplantation (methods) Humans Immunosuppressive Agents (therapeutic use) Infections (epidemiology, mortality) Kaplan-Meier Estimate Male Methotrexate (therapeutic use) Methylprednisolone (therapeutic use) Mycophenolic Acid (therapeutic use) Myeloablative Agonists (therapeutic use) Severity of Illness Index Survival Rate Transplantation Conditioning Treatment Outcome Vidarabine (analogs & derivatives, therapeutic use) Whole-Body Irradiation

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