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. Bone Marrow Transplantation (2011) 46, 330-337; doi:10.1038/bmt.2010.100; published online 3 May 2010
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., Schupbach; Casali, Carlo; R., Elhasid; K., Fay; S., Hammans; I., Illa; L., Kappeler; S., Krahenbuhl; T., Lehmann; H., Mandel; R., Marti; H., Mattle; K., Orchard; D., Savage; C. M., Sue; D., Valcarcel; A., Gratwohl; M., Hirano. - In: BONE MARROW TRANSPLANTATION. - ISSN 0268-3369. - 46:3(2011), pp. 330-337. [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
CASALI, Carlo;
2011
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. Bone Marrow Transplantation (2011) 46, 330-337; doi:10.1038/bmt.2010.100; published online 3 May 2010I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
