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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

Bone Marrow Transplantation volume 46, pages 330–337 (2011)Cite this article

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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.

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Acknowledgements

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.

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Author notes

  1. J Halter and W M M Schüpbach: These authors contributed equally to this work.

  2. A Gratwohl and M Hirano: These authors contributed equally to this work.

Authors and Affiliations

  1. Department of Hematology, University Hospital Basel, Basel, Switzerland

    J Halter, T Lehmann & A Gratwohl

  2. Centre d’Investigation Clinique, Fédération des Maladies du Système Nerveux & INSERM UMR 679, Pitié-Salpxêtrière Group, Paris, France

    W M M Schüpbach & H Mattle

  3. Department of Neurology, University Hospital-Inselspital Bern, Bern, Switzerland

    W M M Schüpbach, L Kappeler & H Mattle

  4. Neurology, La Sapienza University, University Hospital, Rome, Italy

    C Casali

  5. Pediatric-Oncology, Rambam Medical Centre, Haifa, Israel

    R Elhasid

  6. Department of Hematology, St Vincent's Hospital, Darlinghurst, Sydney, Australia,

    K Fay

  7. Wessex Neurological Centre, Southampton University Hospital Trust, Southampton, UK

    S Hammans

  8. Neurology, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain

    I Illa

  9. Clinical Pharmacology and Toxicology, University Hospital Basel, Basel, Switzerland

    S Krähenbühl

  10. Pediatrics, Rambam Medical Centre, Haifa, Israel

    H Mandel

  11. Institut de Recerca, University Hospital Vall d’Hebron and CIBERER, Barcelona, Spain

    R Marti

  12. Department of Haematology, University of Southampton, Southampton, UK

    K Orchard

  13. Department of Haematology, Columbia University Medical Centre, New York, NY, USA

    D Savage

  14. Department of Neurogenetics, University of Sydney Kolling Institute for Medical Research, Kolling Institute for Medical Research, Royal North Shore Hospital and University of Sydney, Sydney, Australia

    C M Sue

  15. Department of Hematology, Santa Creu i San Pau Hospital, Barcelona, Spain

    D Valcarcel

  16. Department of Neurology, Columbia University Medical Centre, New York, NY, USA

    M Hirano

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  1. J Halter
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Correspondence to J Halter.

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Halter, J., Schüpbach, W., Casali, C. et al. Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach. Bone Marrow Transplant 46, 330–337 (2011). https://doi.org/10.1038/bmt.2010.100

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  • DOI: https://doi.org/10.1038/bmt.2010.100

Keywords

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

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