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

J Halter; Wmm 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

Bone Marrow Transplant. 2011 Mar;46(3):330-337. doi: 10.1038/bmt.2010.100. Epub 2010 May 3.

Authors

J Halter^#¹, Wmm Schüpbach^#^{2

3}, C Casali⁴, R Elhasid⁵, K Fay⁶, S Hammans⁷, I Illa⁸, L Kappeler³, S Krähenbühl⁹, T Lehmann¹, H Mandel¹⁰, R Marti¹¹, H Mattle^{2

3}, K Orchard¹², D Savage¹³, C M Sue¹⁴, D Valcarcel¹⁵, A Gratwohl^#¹, M Hirano^#¹⁶

Affiliations

¹ Department of Hematology, University Hospital Basel, Basel, Switzerland.
² Centre d'Investigation Clinique, Fédération des Maladies du Système Nerveux & INSERM UMR 679, Pitié-Salpxêtrière Group, Paris, France.
³ Department of Neurology, University Hospital-Inselspital Bern, Bern, Switzerland.
⁴ Neurology, La Sapienza University, University Hospital, Rome, Italy.
⁵ Pediatric-Oncology, Rambam Medical Centre, Haifa, Israel.
⁶ Department of Hematology, St Vincent's Hospital, Darlinghurst, Sydney, Australia.
⁷ Wessex Neurological Centre, Southampton University Hospital Trust, Southampton, UK.
⁸ Neurology, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
⁹ Clinical Pharmacology and Toxicology, University Hospital Basel, Basel, Switzerland.
¹⁰ Pediatrics, Rambam Medical Centre, Haifa, Israel.
¹¹ Institut de Recerca, University Hospital Vall d'Hebron and CIBERER, Barcelona, Spain.
¹² Department of Haematology, University of Southampton, Southampton, UK.
¹³ Department of Haematology, Columbia University Medical Centre, New York, NY, USA.
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.
¹⁵ Department of Hematology, Santa Creu i San Pau Hospital, Barcelona, Spain.
¹⁶ Department of Neurology, Columbia University Medical Centre, New York, NY, USA.

^# Contributed equally.

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.

Publication types

Consensus Development Conference
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Humans
Intestinal Pseudo-Obstruction / genetics
Intestinal Pseudo-Obstruction / surgery
Mitochondrial Encephalomyopathies / genetics
Mitochondrial Encephalomyopathies / surgery
Muscular Dystrophy, Oculopharyngeal
Ophthalmoplegia / congenital
Stem Cell Transplantation / standards*

Supplementary concepts

Visceral myopathy familial external ophthalmoplegia

Abstract

Publication types

MeSH terms

Supplementary concepts

Grants and funding