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

doi:10.1038/bmt.2010.100

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

Department of Medicine

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80 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 language	English
Pages (from-to)	330-337
Journal	Bone Marrow Transplantation
Volume	46
Issue number	3
DOIs	https://doi.org/10.1038/bmt.2010.100
Publication status	Published - 1 Mar 2011

Keywords

Allogeneic hematopoietic SCT
consensus
mitochondrial neurogastrointestinal encephalomyopathy
MNGIE
recommendation

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10.1038/bmt.2010.100

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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., & 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. https://doi.org/10.1038/bmt.2010.100

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. ; Hirano, M. / Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach. In: Bone Marrow Transplantation. 2011 ; Vol. 46, No. 3. pp. 330-337.

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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. {\textcopyright} 2011 Macmillan Publishers Limited All rights reserved.",

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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",

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Halter, J, Schüpbach, WMM, 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, CM, Valcarcel, D, Gratwohl, A & 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, vol. 46, no. 3, pp. 330-337. https://doi.org/10.1038/bmt.2010.100

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach. / 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.; Hirano, M.

In: Bone Marrow Transplantation, Vol. 46, No. 3, 01.03.2011, p. 330-337.

Research output: Contribution to journal › Article › Research › peer-review

TY - JOUR

T1 - Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach

AU - Halter, J.

AU - Schüpbach, W. M.M.

AU - Casali, C.

AU - Elhasid, R.

AU - Fay, K.

AU - Hammans, S.

AU - Illa, I.

AU - Kappeler, L.

AU - Krähenbühl, S.

AU - Lehmann, T.

AU - Mandel, H.

AU - Marti, R.

AU - Mattle, H.

AU - Orchard, K.

AU - Savage, D.

AU - Sue, C. M.

AU - Valcarcel, D.

AU - Gratwohl, A.

AU - Hirano, M.

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AB - 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.

KW - Allogeneic hematopoietic SCT

KW - consensus

KW - mitochondrial neurogastrointestinal encephalomyopathy

KW - MNGIE

KW - recommendation

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DO - 10.1038/bmt.2010.100

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EP - 337

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

SN - 0268-3369

IS - 3

ER -

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

Abstract

Keywords

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