Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology

The treatment of choice for aplastic anemia (AA) in children has been HLA-matched family donor (MFD) hematopoietic stem cell transplantation (HSCT). For those lacking MFD, immunosuppressive therapy (IST) consisting of horse antithymocyte globulin (ATG) and cyclosporine has been successful. The choic...

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Bibliographic Details
Published in:International Journal of Hematology 2016-04, Vol.103 (4), p.380-386
Main Authors: Kook, Hoon, Chung, Nack-Gyun, Kang, Hyoung Jin, Im, Ho Joon
Format: Article
Language:English
Subjects:
Anemia, Aplastic - epidemiology
Anemia, Aplastic - therapy
Animals
Child
Hematology
Hematopoietic Stem Cell Transplantation - methods
Humans
Immunosuppression - methods
Immunosuppressive Agents - therapeutic use
Medicine
Medicine & Public Health
Oncology
Progress in Hematology
Republic of Korea - epidemiology
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Summary:The treatment of choice for aplastic anemia (AA) in children has been HLA-matched family donor (MFD) hematopoietic stem cell transplantation (HSCT). For those lacking MFD, immunosuppressive therapy (IST) consisting of horse antithymocyte globulin (ATG) and cyclosporine has been successful. The choices of second and third line treatments are more complex and debatable, especially in the situation of unavailability of horse ATG. IST with rabbit ATG seems to be less effective. Recently, improved survival of non-MFD HSCTs has been documented. The outcome of matched or mismatched unrelated donor, umbilical cord blood, or haploidentical family donor transplantations will be discussed in AA children after IST failure. Experimental approaches of upfront HSCT using non-MFDs will be briefly touched. In this review, a treatment guideline for children with AA from the Korean Society of Pediatric Hematology Oncology will be presented along with a brief review of literature on current clinical practices in Korea.
ISSN:0925-5710
1865-3774
DOI:10.1007/s12185-016-1956-8