Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: Current status and critical needs
Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: Current status and critical needs
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DOI：
10.1016/j.jaci.2008.09.045
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发表时间：
2008-12-01
影响因子：
14.2
通讯作者：
Shearer, William T.
中科院分区：
文献类型：
--
作者：
Griffith, Linda M.;Cowan, Morton J.;Shearer, William T.
异基因造血细胞移植（HCT）已被用于改善或治愈原发性免疫缺陷（PID）疾病（包括严重联合免疫缺陷（SCID）和非SCID PID）40年。有一个关键的需要进行评估的不同HCT方法对这些疾病的北美经验，以确定最佳实践和规划未来的调查性临床试验。我们在北美实践场所对PID发病率和患病率的调查表明，此类研究是可行的。HCT治疗PID的专家会议建议：（1）对HCT存活的SCID患者进行全面的横断面和回顾性分析;（2）对接受HCT的SCID患者进行前瞻性研究，在参与中心之间进行可比的基线和随访检测;（3）对新生儿进行SCID筛查的试点研究，以在感染危害之前识别受影响的婴儿;和（4）对Wiskott-Aldrich综合征和慢性肉芽肿病等非SCID疾病接受或未接受RCT的患者进行疾病自然史研究。为了实现这些目标，提议由北美的一个机构联合体进行合作。这些研究的成功将需要对PID感兴趣的免疫学家和HCT医生以及实验室方法、临床结局评估、数据库和分析方面的专家的参与。（J Allergy Clin Immunol 2008; 122：1087-96.）
Allogeneic hematopoietic cell transplantation (HCT) has been used for 40 years to ameliorate or cure primary immune deficiency (PID) diseases, including severe combined immunodeficiency (SCID) and non-SCID PID. There is a critical need for evaluation of the North American experience of different HCT approaches for these diseases to identify best practices and plan future investigative clinical trials. Our survey of incidence and prevalence of PID in North American practice sites indicates that such studies are feasible. A conference of experts in HCT treatment of PID has recommended (1) a comprehensive cross-sectional and retrospective analysis of HCT survivors with SCID; (2) a prospective study of patients with SCID receiving HCT, with comparable baseline and follow-up testing across participating centers; (3) a pilot study of newborn screening for SCID to identify affected infants before compromise by infection; and (4) studies of the natural history of disease in patients who do or do not receive RCT for the non-SCID diseases of Wiskott-Aldrich syndrome and chronic granulomatous disease. To accomplish these goals, collaboration by a consortium of institutions in North America is proposed. Participation of immunologists and HCT physicians having interest in PID and experts in laboratory methods, clinical outcomes assessment, databases, and analysis will be required for the success of these studies. (J Allergy Clin Immunol 2008; 122:1087-96.)