期刊
KLINISCHE PADIATRIE
卷 227, 期 3, 页码 157-165出版社
GEORG THIEME VERLAG KG
DOI: 10.1055/s-0035-1548841
关键词
fanconi anemia; stem cell transplantation; consensus; recommendations
类别
资金
- Deutsche Kinderkrebs-stiftung
- Deutsche Fanconi Anamie Hilfe e.V.
- Aktionskreis Fanconi-Anamie e.V.
- Fanconi-Anamie-Stiftung
Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative therapy for the severe hematopoietic complications associa-ted with Fanconi anemia (FA). In Germany, it is estimated that 10-15 transplants are performed annually for FA. However, because FA is a DNA repair disorder, standard conditioning regimens confer a high risk of excessive regimen-related toxicities and mortality, and reduced intensity regimens are linked with graft failure in some FA patients. Moreover, development of graft-versus-host disease is a major contributing factor for secondary solid tumors. The relative rarity of the disorder limits HSCT experience at any single center. Consensus meetings were convened to develop a national approach for HSCT in FA. This manuscript outlines current experience and knowledge about HSCT in FA and, based on this analysis, general recommendations reached at these meetings.
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