Primary Immune Deficiency Treatment Consortium (PIDTC) update.
Primary Immune Deficiency Treatment Consortium (PIDTC) update.
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DOI：
10.1016/j.jaci.2016.01.051
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发表时间：
2016-08
期刊：
影响因子：
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通讯作者：
workshop participants
中科院分区：
文献类型：
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作者：
Griffith LM;Cowan MJ;Notarangelo LD;Kohn DB;Puck JM;Shearer WT;Burroughs LM;Torgerson TR;Decaluwe H;Haddad E;workshop participants
原发性免疫缺陷治疗联盟 (PIDTC) 是由 41 个北美中心合作组成，研究罕见的原发性免疫缺陷疾病 (PID) 的治疗方法，包括严重联合免疫缺陷 (SCID)、Wiskott-Aldrich 综合征 (WAS) 和慢性肉芽肿病 (CGD)。另外 3 个欧洲中心与 PIDTC 合作研究 CGD。 PIDTC 的自然史方案通过多中心纵向回顾性、前瞻性和横断面研究分析罕见 PID 的治疗结果。自 2009 年以来，参与中心已根据 PIDTC 方案招募了 800 多名 SCID 受试者，WAS 和 CGD 研究的招募正在进行中。四个试点项目已获得资助，12 名初级研究人员获得了奖学金。该联盟的重要出版物描述了 2000-2009 年 SCID 造血细胞移植 (HCT) 的结果、SCID 的诊断标准以及纳瓦霍族 SCID 新生儿筛查 (NBS) 试点项目。 PIDTC 年度科学研讨会提供了加强与初级研究人员、患者倡导团体和国际同事合作的机会。 PIDTC 由 NIAID 和 ORDR、NCATS 资助，最近又获得了 5 年的续展。在这里，我们回顾了该小组的成就、正在进行的项目、最近研讨会的亮点以及未来的挑战。
The Primary Immune Deficiency Treatment Consortium (PIDTC) is a collaboration of 41 North American centers studying therapy for rare primary immune deficiency diseases (PID) including Severe Combined Immune Deficiency (SCID), Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). An additional 3 European centers have partnered with the PIDTC to study CGD. Natural history protocols of the PIDTC analyze outcomes of treatment for rare PID in multicenter longitudinal retrospective, prospective and cross-sectional studies. Since 2009, participating centers have enrolled over 800 subjects on PIDTC protocols for SCID, and enrollment on the studies in WAS and CGD is underway. Four pilot projects have been funded and 12 junior investigators have received fellowship awards. Important publications of the consortium describe outcomes of hematopoietic cell transplantation (HCT) for SCID during 2000–2009, diagnostic criteria for SCID, and the pilot project of newborn screening (NBS) for SCID in the Navajo Nation. The PIDTC Annual Scientific Workshops provide an opportunity to strengthen collaborations with junior investigators, patient advocacy groups and international colleagues. Funded by the NIAID and ORDR, NCATS, the PIDTC has recently received renewal for another 5 years. Here, we review accomplishments of the group, projects underway, highlights of recent workshops and challenges for the future.