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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

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

0268-3369
330-337
Halter, J.
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Schüpbach, W.M.M.
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Casali, C.
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Elhasid, R.
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Fay, K.
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Hammans, S.
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Illa, I.
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Kappeler, L.
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Krähenbühl, S.
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Lehmann, T.
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Mandel, H.
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Marti, R.
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Mattle, H.
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Orchard, K.
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Savage, D.
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Sue, C. M.
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Valcarcel, D.
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Gratwohl, A.
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Hirano, M.
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Halter, J.
8ebc0922-3eea-4b34-ad59-ca064967be73
Schüpbach, W.M.M.
a96c251b-5654-4b11-9d75-e8241d28d9cc
Casali, C.
12f83f10-329f-465f-b509-e0e4134870b5
Elhasid, R.
5d911651-8b5a-4166-8715-6b6fe3dea206
Fay, K.
5dc7b983-9ad5-47b4-9590-09796dd9a50e
Hammans, S.
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Illa, I.
9b66a9ab-4f1a-475d-b9b4-6103f0acf2ed
Kappeler, L.
7d8ee772-2edb-4544-9ccb-b12c376b97f6
Krähenbühl, S.
bab622d0-ad8a-48b5-bf3f-b594c4758252
Lehmann, T.
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Mandel, H.
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Marti, R.
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Mattle, H.
ec99128e-01bb-4613-b411-5d2e087aadac
Orchard, K.
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Savage, D.
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Sue, C. M.
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Valcarcel, D.
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Gratwohl, A.
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Hirano, M.
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Halter, J., Schüpbach, W.M.M., Casali, C., Elhasid, R., Fay, K., Hammans, S., Illa, I., Kappeler, L., Krähenbühl, S., Lehmann, T., Mandel, H., Marti, R., Mattle, H., Orchard, K., Savage, D., Sue, C. M., Valcarcel, D., Gratwohl, A. and Hirano, M. (2011) Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach. Bone Marrow Transplantation, 46 (3), 330-337. (doi:10.1038/bmt.2010.100).

Record type: Article

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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Published date: May 2011
Related URLs:
Organisations: Cancer Sciences

Identifiers

Local EPrints ID: 341438
URI: http://eprints.soton.ac.uk/id/eprint/341438
DOI: doi:10.1038/bmt.2010.100
ISSN: 0268-3369
PURE UUID: ca71396e-4cf0-4a43-b746-ac5b98a3a46f
ORCID for K. Orchard: ORCID iD orcid.org/0000-0003-2276-3925

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Date deposited: 25 Jul 2012 10:48
Last modified: 15 Mar 2024 03:13

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Contributors

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

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