@article{196c22ce71e14bed8ff5aa0cde469a80,
title = "Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach",
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.",
keywords = "Allogeneic hematopoietic SCT, MNGIE, consensus, mitochondrial neurogastrointestinal encephalomyopathy, recommendation",
author = "J. Halter and Sch{\"u}pbach, {W. M.M.} and C. Casali and R. Elhasid and K. Fay and S. Hammans and I. Illa and L. Kappeler and S. Kr{\"a}henb{\"u}hl and T. Lehmann and H. Mandel and R. Marti and H. Mattle and K. Orchard and D. Savage and Sue, {C. M.} and D. Valcarcel and A. Gratwohl and M. Hirano",
note = "Funding Information: RM is supported by a grant from Instituto de Salud Carlos III (PI06/0735). MH is supported by US National Institutes of Health (NIH) Grants HD32062 and RCNS070232 by grants from the Muscular Dystrophy Association, and by the Marriott Mitochondrial Disorders Clinical Research Fund (MMDCRF). The consensus conference was supported by a travel grant from Swiss Blood Stem Cells and a grant from the Swiss Foundation for Research on Muscle Diseases.",
year = "2011",
month = mar,
doi = "10.1038/bmt.2010.100",
language = "אנגלית",
volume = "46",
pages = "330--337",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Springer Nature",
number = "3",
}