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Management of acquired aplastic anemia in children.
MedLine Citation:
PMID:  23292240     Owner:  NLM     Status:  Publisher    
Abstract/OtherAbstract:
The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The gold standard of therapy remains hematopoietic SCT with a graft of BM cells for those children with matched sibling donors. Conversely for children without a sibling donor the high response and markedly improved overall survival rates of combined immunosuppressive therapy have proven robust, especially when horse derived anti-thymocyte globuline plus ciclosporine A are used. Incomplete response, relapse and progression to myelodysplasia/leukemia however have emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Regardless of the type of therapeutic approach, patients require centralized treatment in a center of excellence, ongoing monitoring for recurrence of disease and/or therapy-related immediate side effects and long-term effects.Bone Marrow Transplantation advance online publication, 7 January 2013; doi:10.1038/bmt.2012.235.
Authors:
E T Korthof; A N Békássy; A A Hussein
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2013-1-07
Journal Detail:
Title:  Bone marrow transplantation     Volume:  -     ISSN:  1476-5365     ISO Abbreviation:  Bone Marrow Transplant.     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2013-1-7     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  8702459     Medline TA:  Bone Marrow Transplant     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
Affiliation:
1] Department of Pediatrics/Willem-Alexander Children's Hospital, Division of Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands [2] Sanquin-LUMC Jon J van Rood Centre for Clinical Transfusion Research, Leiden University Medical Center, Leiden, The Netherlands.
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