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

Bone Marrow Transplant. 2011 Mar;46(3):330-337. doi: 10.1038/bmt.2010.100. Epub 2010 May 3.


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.

Publication types

  • Consensus Development Conference
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Intestinal Pseudo-Obstruction / genetics
  • Intestinal Pseudo-Obstruction / surgery
  • Mitochondrial Encephalomyopathies / genetics
  • Mitochondrial Encephalomyopathies / surgery
  • Muscular Dystrophy, Oculopharyngeal
  • Ophthalmoplegia / congenital
  • Stem Cell Transplantation / standards*

Supplementary concepts

  • Visceral myopathy familial external ophthalmoplegia