期刊
BONE MARROW TRANSPLANTATION
卷 46, 期 3, 页码 330-337出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/bmt.2010.100
关键词
allogeneic hematopoietic SCT; mitochondrial neurogastrointestinal encephalomyopathy; MNGIE; consensus; recommendation
资金
- Instituto de Salud Carlos III [PI06/0735]
- US National Institutes of Health (NIH) [HD32062, RCNS070232]
- Muscular Dystrophy Association
- Marriott Mitochondrial Disorders Clinical Research Fund (MMDCRF)
- Swiss Blood Stem Cells
- Swiss Foundation for Research on Muscle Diseases
Allogeneic hematopoietic SCT (HSCT) has been proposed as a treatment for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). HSCT has been performed in nine patients using different protocols with varying success. Based on this preliminary experience, participants of the first consensus conference propose a common approach to allogeneic HSCT in MNGIE. Standardization of the transplant protocol and the clinical and biochemical assessments will allow evaluation of the safety and efficacy of HSCT as well as optimization of therapy for patients with MNGIE. Bone Marrow Transplantation (2011) 46, 330-337; doi:10.1038/bmt.2010.100; published online 3 May 2010
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