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.
authors.affiliation: Department of Hematology, University Hospital Basel, Basel, Switzerland.
authors.forename: J
authors.initials: J
authors.lastname: Halter
authors.affiliation: Centre d'Investigation Clinique, Fédération des Maladies du Système Nerveux & INSERM UMR 679, Pitié-Salpxêtrière Group, Paris, France.|Department of Neurology, University Hospital-Inselspital Bern, Bern, Switzerland.
authors.forename: Wmm
authors.initials: W
authors.lastname: Schüpbach
authors.affiliation: Neurology, La Sapienza University, University Hospital, Rome, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Casali
authors.affiliation: Pediatric-Oncology, Rambam Medical Centre, Haifa, Israel.
authors.forename: R
authors.initials: R
authors.lastname: Elhasid
authors.affiliation: Department of Hematology, St Vincent's Hospital, Darlinghurst, Sydney, Australia.
authors.forename: K
authors.initials: K
authors.lastname: Fay
authors.affiliation: Wessex Neurological Centre, Southampton University Hospital Trust, Southampton, UK.
authors.forename: S
authors.initials: S
authors.lastname: Hammans
authors.affiliation: Neurology, Hospital de la Santa Creu i Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain.
authors.forename: I
authors.initials: I
authors.lastname: Illa
authors.affiliation: Department of Neurology, University Hospital-Inselspital Bern, Bern, Switzerland.
authors.forename: L
authors.initials: L
authors.lastname: Kappeler
authors.affiliation: Clinical Pharmacology and Toxicology, University Hospital Basel, Basel, Switzerland.
authors.forename: S
authors.initials: S
authors.lastname: Krähenbühl
authors.affiliation: Department of Hematology, University Hospital Basel, Basel, Switzerland.
authors.forename: T
authors.initials: T
authors.lastname: Lehmann
authors.affiliation: Pediatrics, Rambam Medical Centre, Haifa, Israel.
authors.forename: H
authors.initials: H
authors.lastname: Mandel
authors.affiliation: Institut de Recerca, University Hospital Vall d'Hebron and CIBERER, Barcelona, Spain.
authors.forename: R
authors.initials: R
authors.lastname: Marti
authors.affiliation: Centre d'Investigation Clinique, Fédération des Maladies du Système Nerveux & INSERM UMR 679, Pitié-Salpxêtrière Group, Paris, France.|Department of Neurology, University Hospital-Inselspital Bern, Bern, Switzerland.
authors.forename: H
authors.initials: H
authors.lastname: Mattle
authors.affiliation: Department of Haematology, University of Southampton, Southampton, UK.
authors.forename: K
authors.initials: K
authors.lastname: Orchard
authors.affiliation: Department of Haematology, Columbia University Medical Centre, New York, NY, USA.
authors.forename: D
authors.initials: D
authors.lastname: Savage
authors.affiliation: 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.
authors.forename: C M
authors.initials: CM
authors.lastname: Sue
authors.affiliation: Department of Hematology, Santa Creu i San Pau Hospital, Barcelona, Spain.
authors.forename: D
authors.initials: D
authors.lastname: Valcarcel
authors.affiliation: Department of Hematology, University Hospital Basel, Basel, Switzerland.
authors.forename: A
authors.initials: A
authors.lastname: Gratwohl
authors.affiliation: Department of Neurology, Columbia University Medical Centre, New York, NY, USA.
authors.forename: M
authors.initials: M
authors.lastname: Hirano
mesh_terms: D006801:Humans; D007418:Intestinal Pseudo-Obstruction / Q000235:genetics / Q000601:surgery; D017237:Mitochondrial Encephalomyopathies / Q000235:genetics / Q000601:surgery; D039141:Muscular Dystrophy, Oculopharyngeal; D009886:Ophthalmoplegia / Q000151:congenital; D033581:Stem Cell Transplantation / Q000592:standards*; D028361:Mitochondrial Diseases