Dissemin is shutting down on January 1st, 2025

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Springer Nature [academic journals on nature.com], Bone Marrow Transplantation, 3(46), p. 330-337, 2010

DOI: 10.1038/bmt.2010.100

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Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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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. Bone Marrow Transplantation (2011) 46, 330-337; doi:10.1038/bmt.2010.100; published online 3 May 2010