Abstract
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. © 2011 Macmillan Publishers Limited All rights reserved.
Original language | English |
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Pages (from-to) | 330-337 |
Journal | Bone Marrow Transplantation |
Volume | 46 |
Issue number | 3 |
DOIs | |
Publication status | Published - 1 Mar 2011 |
Keywords
- Allogeneic hematopoietic SCT
- consensus
- mitochondrial neurogastrointestinal encephalomyopathy
- MNGIE
- recommendation