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

J. Halter, W. M.M. Schüpbach, C. Casali, R. Elhasid, K. Fay, S. Hammans, I. Illa, L. Kappeler, S. Krähenbühl, T. Lehmann, H. Mandel, R. Marti, H. Mattle, K. Orchard, D. Savage, C. M. Sue, D. Valcarcel, A. Gratwohl, M. Hirano

Research output: Contribution to journalArticleResearchpeer-review

73 Citations (Scopus)

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 languageEnglish
Pages (from-to)330-337
JournalBone Marrow Transplantation
Volume46
Issue number3
DOIs
Publication statusPublished - 1 Mar 2011

Keywords

  • Allogeneic hematopoietic SCT
  • consensus
  • mitochondrial neurogastrointestinal encephalomyopathy
  • MNGIE
  • recommendation

Fingerprint Dive into the research topics of 'Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach'. Together they form a unique fingerprint.

Cite this