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Aplastic Anemia

Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched familial donor

Bone Marrow Transplantation volume 52, pages 47–52 (2017)Cite this article

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Abstract

We compared the outcomes of immunosuppressive treatment (IST) with those of alternative donor hematopoietic stem cell transplantation (HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 42 patients with SAA who received frontline IST (N=19) or frontline HSCT with an alternative donor (N=23) between 1998 and 2012 were analyzed retrospectively. Six patients responded in the frontline IST group, whereas 11 underwent salvage HSCT after IST failure. Twenty-one of 23 patients who underwent frontline HSCT survived without treatment failure. The estimated failure-free survival rate of the frontline HSCT group was higher than that of the frontline IST group (91.3% vs 30.7% respectively, P<0.001). Six of 11 patients who underwent salvage HSCT experienced event-free survival (EFS). The estimated EFS of the frontline HSCT group was higher than that of the salvage HSCT group (91.3% vs 50.9% respectively, P=0.015). The outcome of alternative donor HSCT was better than commonly reported rates, especially in patients who underwent frontline HSCT. These results suggest that frontline alternative donor HSCT may be a better treatment option than IST for children and adolescents with SAA who lack a human leukocyte Ag-matched familial donor.

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Acknowledgements

We thank the residents and nurses who cared for the patients, and without whom this study would not have been possible.

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Authors and Affiliations

  1. Department of Pediatrics, Chung-Ang University Hospital, Seoul, Korea

    Y B Choi

  2. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea

    E S Yi, J W Lee, K W Sung, H H Koo & K H Yoo

  3. Department of Medical Device Management and Research, SAIHST, Sungkyunkwan University, Seoul, Korea

    K H Yoo

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  1. Y B Choi
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Correspondence to K H Yoo.

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Choi, Y., Yi, E., Lee, J. et al. Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched familial donor. Bone Marrow Transplant 52, 47–52 (2017). https://doi.org/10.1038/bmt.2016.223

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