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Management of acquired aplastic anemia in children

Korthof, E. T.; Békássy, Albert LU and Hussein, A. A. (2013) In Bone Marrow Transplantation 48(2). p.191-195
Abstract
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... (More)
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-ternn effects. Bone Marrow Transplantation (2013) 48, 191-195; doi:10.1038/bmt.2012.235; published online 7 January 2013 (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
acquired aplastic anemia, childhood, supportive care, management, hematopoietic SCT, immunosuppressive therapy
in
Bone Marrow Transplantation
volume
48
issue
2
pages
191 - 195
publisher
Nature Publishing Group
external identifiers
  • wos:000315239200009
  • scopus:84873443351
ISSN
1476-5365
DOI
10.1038/bmt.2012.235
language
English
LU publication?
yes
id
354a991c-88e2-4b96-ba12-7750f71c887c (old id 3674561)
date added to LUP
2013-05-02 08:59:15
date last changed
2019-01-06 04:24:54
@article{354a991c-88e2-4b96-ba12-7750f71c887c,
  abstract     = {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-ternn effects. Bone Marrow Transplantation (2013) 48, 191-195; doi:10.1038/bmt.2012.235; published online 7 January 2013},
  author       = {Korthof, E. T. and Békássy, Albert and Hussein, A. A.},
  issn         = {1476-5365},
  keyword      = {acquired aplastic anemia,childhood,supportive care,management,hematopoietic SCT,immunosuppressive therapy},
  language     = {eng},
  number       = {2},
  pages        = {191--195},
  publisher    = {Nature Publishing Group},
  series       = {Bone Marrow Transplantation},
  title        = {Management of acquired aplastic anemia in children},
  url          = {http://dx.doi.org/10.1038/bmt.2012.235},
  volume       = {48},
  year         = {2013},
}