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

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

95 Scopus citations

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.

Original languageEnglish
Pages (from-to)330-337
Number of pages8
JournalBone Marrow Transplantation
Volume46
Issue number3
DOIs
StatePublished - Mar 2011
Externally publishedYes

Funding

FundersFunder number
Marriott Mitochondrial Disorders Clinical Research Fund
Swiss Blood Stem Cells
National Institutes of HealthRCNS070232
National Institute of Child Health and Human DevelopmentP01HD032062
Muscular Dystrophy Association
Stiftung für die Erforschung der Muskelkrankheiten
Instituto de Salud Carlos IIIPI06/0735

    Keywords

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

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