Recommendations for Screening and Management of Late Effects in Patients with Severe Combined Immunodeficiency after Allogenic Hematopoietic Cell Transplantation: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.

异基因造血细胞移植后严重联合免疫缺陷患者晚期效应的筛查和管理建议 ： 第二届儿科血液和骨髓移植联盟国际会议关于儿科 HCT 后晚期效应的共识声明。

• 影响指数: 5.609
• 发表时间: Aug 2017
• 来源期刊: Biol Blood Marrow Transplant
• DOI: 10.1016/j.bbmt.2017.04.026
• PMID: 28479164
• 文章类型: Journal Article
• 中科院分区: Q2
• 方向: 医学
• 作者列表: Heimall J,Buckley RH,Puck J,Fleisher TA,Gennery AR,Haddad E,Neven B,Slatter M,Roderick S,Baker KS,Dietz AC,Duncan C,Griffith LM,Notarangelo L,Pulsipher MA,Cowan MJ

关键词: Bone marrow transplantation; Long-term follow-up guidelines; Severe combined immune deficiency

Mesh: Adolescent; Adult; Allografts; Child; Child, Preschool; Consensus; Female; Hematopoietic Stem Cell Transplantation; Humans; Infant; Infant, Newborn; Male; Practice Guidelines as Topic; Severe Combined Immunodeficiency / immunology therapy

Abstract

Severe combined immunodeficiency (SCID) is effectively treated with hematopoietic cell transplantation (HCT), with overall survival approaching 90% in contemporary reports. However, survivors are at risk for developing late complications because of the variable durability of high-quality immune function, underlying genotype of SCID, comorbidities due to infections in the pretransplantation and post-transplantation periods, and use of conditioning before transplantation. An international group of transplantation experts was convened in 2016 to review the current knowledge of late effects seen in SCID patients after HCT and to develop recommendations for screening and monitoring for late effects. This report provides recommendations for screening and management of pediatric and adult SCID patients treated with HCT.

摘要

严重的联合免疫缺陷（SCID）可以通过造血细胞移植（HCT）有效治疗。在当代报告中，总生存率接近90%。然而,由于高质量免疫功能的持久性，幸存者有发生晚期并发症的风险，潜在的SCID基因型，由于移植前和移植后感染引起的合并症，和移植前的调理运用。2016年召集了一个国际移植专家小组，以审查HCT后SCID患者对晚期效应的最新知识，并提出筛查和监测晚期效应的建议。本报告为筛查和管理接受HCT治疗的儿童和成人SCID患者提供了建议。