RDSafe: A Multi-Institutional Study of HSC Donor Safety and Quality Life

描述(申请人提供)：2006年，北美超过8000名成人和儿童捐献了骨髓(BM)或外周血干细胞(PBSC)用于造血干细胞(HSC)移植。这些捐赠大部分来自受者的亲属(每年5000人)，由照顾受者的移植中心提供便利。其余的捐赠来自匿名的无关成年志愿者，由国家骨髓捐赠者计划(NMDP)协调和仔细管理。在过去的二十年里，通过NMDP进行的高质量研究产生了大量关于与无关的HSC捐赠相关的医疗和心理毒性的数据。相反，关于亲属捐赠者的医疗和心理安全的预期的、可量化的和容易比较的数据要么过时了，要么缺乏，特别是在儿科和老年捐赠者的潜在脆弱人群中(&gt；60年)。我们建议的研究(称为RDSafe，相关捐赠者安全研究)通过使用NMDP开发的复杂和全面的数据工具比较相关HSC捐赠者和非相关捐赠者(UDS)的医疗和心理毒性来填补这一关键数据空白。急性毒性将使用常见事件(修改后的CTC标准)和不常见事件的标准量表进行报告。将使用经过充分验证的标准化健康相关生活质量(HRQL)工具(成人使用SF-36，儿童使用PedsQL“4.0)来评估捐赠者，并将与年龄匹配的正常人以及相关捐赠者和非血缘捐赠者进行比较。这项研究是由国际血液和骨髓移植研究中心(CIBMTR)捐赠者健康和安全工作委员会的成员开发的：该提案的PI是该委员会的现任联席主席。NMDP的捐献者安全经验和数据工具将与CIBMTR的血液和骨髓移植临床研究资源(RCI-BMT)提供的统计专业知识相结合。这项研究的目的是提高儿科和老年RDS的应计利润，以便收集足够的数据来提供这些群体特有的毒性风险概况。来自儿童肿瘤组的40个CIBMTR移植中心和80多个儿科移植中心已承诺加入RDS。这一队列将与70多个NMDP捐赠者中心收集的同时登记的UDS队列进行比较。在其他重大贡献中，RDSafe研究收集的信息将1)确定RD和UDS的医疗和心理结果的差异，并将其与年龄匹配的正常人进行比较；2)将RD人群分为低风险和高风险类别，识别脆弱的捐赠者；3)描述所有年龄的RD中骨髓移植和PBSC捐赠的风险差异；4)确定RD知情同意的标准；以及5)确定处于医疗或心理风险的捐赠者因捐赠而受到严重伤害。

DESCRIPTION (provided by applicant): In 2006 more than 8,000 adults and children in North America donated either bone marrow (BM) or peripheral blood stem cells (PBSC) for hematopoietic stem cell (HSC) transplantation. Most of these donations came from relatives of the recipient (5000/yr), facilitated by transplant centers caring for that recipient. The remaining donations came from anonymous unrelated adult volunteers, coordinated and carefully regulated by the National Marrow Donor Program (NMDP). Over the past two decades, high quality studies run through the NMDP have resulted in a wealth of data regarding medical and psychological toxicities associated with unrelated HSC donation. Conversely, prospective, quantifiable, and readily comparable data regarding the medical and psychological safety of related donors (RDs) is either outdated or lacking, especially in the potentially vulnerable populations of pediatric and older donors (>60yrs). Our proposed study (called RDSafe, related donor safety study) fills this critical data gap by comparing medical and psychological toxicities of related HSC donors with unrelated donors (UDs) using sophisticated and comprehensive data tools developed by the NMDP. Acute toxicities will be reported using standard scales for common (Modified CTC criteria) and uncommon events. Well-validated, standardized health related quality of life (HRQoL) instruments (SF-36 for adults and PedsQL" 4.0 for children) will be used to assess donors and comparisons will be made with age- matched normals and related versus unrelated donors. The study has been developed by members of the Donor Health and Safety Working Committee of the Center for International Blood and Marrow Transplant Research (CIBMTR): the PI of this proposal is a current Co-Chair of this committee. The donor safety experience and data tools of the NMDP will be combined with statistical expertise provided by the Resource for Clinical Investigations in Blood and Marrow Transplant (RCI-BMT) of the CIBMTR. The study has been designed to enhance accrual of pediatric and older RDs, in order to gather sufficient data to provide toxicity risk profiles specific to these groups. Forty CIBMTR transplant centers and over 80 pediatric transplant centers from the Children's Oncology Group have committed to enroll RDs. This cohort will be compared to a concurrently enrolled cohort of UDs collected at over 70 NMDP donor centers. Among other significant contributions, the information gathered from the RDSafe study will 1) define differences in the medical and psychological outcomes of RDs vs. UDs and compare them with age-matched normals, 2) segment the RD population into categories of lower and higher risk, identifying vulnerable populations of donors, 3) describe differences in risk of BM vs. PBSC donation in RD of all ages, 4) define standards for informed consent of RD, and 5) identify donors who are at medical or psychological risk to be significantly harmed by donation.