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Aplastisk anemi hos barn botas med immunsuppression.

Pronk, Cornelis JH and Békássy, Albert LU (2004) In Läkartidningen 101(42). p.5-9
Abstract
Acquired aplastic anemia (AA) is considered to be an autoimmune mediated disease whereas in inherited bone marrow failure syndromes the genetic abnormalities account for the bone marrow dysfunction. The only curative treatment for inherited AA is hematopoietic stem cell transplantation (HSCT). Successful treatment for acquired AA has traditionally been considered with HSCT, whereas single agent therapy with different immunosuppressive (IS) drugs has been disappointing as alternative treatment modality when sibling donors were lacking. Over the last decades however, treatment with combined IS (e.g. Cyclosporin-A, CsA, + Anti Thymocyte Globuline, ATG, has made great progress, resulting in hematological reconstitution comparable with results... (More)
Acquired aplastic anemia (AA) is considered to be an autoimmune mediated disease whereas in inherited bone marrow failure syndromes the genetic abnormalities account for the bone marrow dysfunction. The only curative treatment for inherited AA is hematopoietic stem cell transplantation (HSCT). Successful treatment for acquired AA has traditionally been considered with HSCT, whereas single agent therapy with different immunosuppressive (IS) drugs has been disappointing as alternative treatment modality when sibling donors were lacking. Over the last decades however, treatment with combined IS (e.g. Cyclosporin-A, CsA, + Anti Thymocyte Globuline, ATG, has made great progress, resulting in hematological reconstitution comparable with results achieved with HSCT. We present a single centre retrospective analysis of 24 children with primary marrow failure at onset and treated subsequently during the years 1981-2002. 16 children were diagnosed with acquired severe aplastic anemia (SAA), 6 Fanconi anemia (FA), 1 Seckel syndrome and 1 with congenital amegakaryocytic thrombocytopenia. International randomized co-operative studies are required in order to gain knowledge on best treatment options of the disease. Long-term follow-up is of vital importance in order to elucidate the risk of secondary clonal diseases (PNH, MDS and leukemia) and other late effects. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Läkartidningen
volume
101
issue
42
pages
5 - 9
publisher
Swedish Medical Association
ISSN
0023-7205
language
Swedish
LU publication?
yes
id
8a0063dd-8fc7-4de5-8a05-94a0abd61e43 (old id 1131010)
alternative location
http://ltarkiv.lakartidningen.se/artNo29283
date added to LUP
2008-06-13 10:43:14
date last changed
2016-04-16 05:21:06
@article{8a0063dd-8fc7-4de5-8a05-94a0abd61e43,
  abstract     = {Acquired aplastic anemia (AA) is considered to be an autoimmune mediated disease whereas in inherited bone marrow failure syndromes the genetic abnormalities account for the bone marrow dysfunction. The only curative treatment for inherited AA is hematopoietic stem cell transplantation (HSCT). Successful treatment for acquired AA has traditionally been considered with HSCT, whereas single agent therapy with different immunosuppressive (IS) drugs has been disappointing as alternative treatment modality when sibling donors were lacking. Over the last decades however, treatment with combined IS (e.g. Cyclosporin-A, CsA, + Anti Thymocyte Globuline, ATG, has made great progress, resulting in hematological reconstitution comparable with results achieved with HSCT. We present a single centre retrospective analysis of 24 children with primary marrow failure at onset and treated subsequently during the years 1981-2002. 16 children were diagnosed with acquired severe aplastic anemia (SAA), 6 Fanconi anemia (FA), 1 Seckel syndrome and 1 with congenital amegakaryocytic thrombocytopenia. International randomized co-operative studies are required in order to gain knowledge on best treatment options of the disease. Long-term follow-up is of vital importance in order to elucidate the risk of secondary clonal diseases (PNH, MDS and leukemia) and other late effects.},
  author       = {Pronk, Cornelis JH and Békássy, Albert},
  issn         = {0023-7205},
  language     = {swe},
  number       = {42},
  pages        = {5--9},
  publisher    = {Swedish Medical Association},
  series       = {Läkartidningen},
  title        = {Aplastisk anemi hos barn botas med immunsuppression.},
  volume       = {101},
  year         = {2004},
}