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

异基因造血 SCT 作为线粒体神经胃肠脑肌病 （ MNGIE ） 患者的治疗选择 ： 标准化方法的共识会议建议。

• 影响指数: 5.174
• 发表时间: Mar 2011
• 来源期刊: Bone Marrow Transplant
• DOI: 10.1038/bmt.2010.100
• PMID: 20436523
• 文章类型: Consensus Development Conference
• 中科院分区: Q1
• 方向: 医学
• 作者列表: Halter J,Schüpbach W,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

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

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.

摘要

已提出异基因造血SCT（HSCT）作为线粒体神经胃肠脑肌病（MNGIE）患者的治疗方法。已使用不同的方案在9名患者中进行了HSCT，并取得了不同的成功。根据这些初步经验,第一次共识会议的参与者提出了MNGIE中同种异体HSCT的通用方法。移植方案的标准化以及临床和生化评估将允许评估HSCT的安全性和有效性，以及优化MNGIE患者的治疗。