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

Publikation

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

Wissenschaftlicher Artikel/Review - 03.05.2010

Halter J, Schüpbach Wmm, Casali C, Elhasid R, Fay K, Hammans S, Illa I, Kappeler L, Krähenbühl S, Lehmann Thomas, Mandel H, Martinez Raquel, Mattle Heinrich P, Orchard K, Savage David B, Sue C M, Valcarcel D, Gratwohl A, Hirano M

Zitation

Halter J, Schüpbach W, Casali C, Elhasid R, Fay K, Hammans S, Illa I, Kappeler L, Krähenbühl S, Lehmann T, Mandel H, Martinez R, Mattle H, Orchard K, Savage D, Sue C, 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. Bone Marrow Transplant 2010; 46:330-337.

Art

Wissenschaftlicher Artikel/Review (Englisch)

Zeitschrift

Bone Marrow Transplant 2010; 46

Veröffentlichungsdatum

03.05.2010

eISSN (Online)

1476-5365

Seiten

330-337

Kurzbeschreibung/Zielsetzung

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.