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

RDSafe：HSC 捐献者安全和生活质量的多机构研究

• 批准号: 7756660
• 负责人: Michael A Pulsipher
• 金额: $ 62.25万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-01-13 至 2013-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7756660
• 关键词: Acute; Adult; Adverse effects; Age; Blood; Bone Marrow; Caring; Categories; Child; Childhood; Children' s Oncology Group; Cities; Clinical Trials; Collection; Commit; Communities; Data; Donor Exclusions; Donor Selection; Early treatment; Elderly; Emotional; Enrollment; Europe; Evaluation; Event; Health; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Human Resources; Incidence; Informed Consent; International; Left; Marrow; Measurement; Measures; Medical; Methodology; North America; Outcome; Peripheral Blood Stem Cell; Population; Process; Psychological Side Effects; Publishing; Quality of life; Registries; Relative (related person); Reporting; Research; Resources; Risk; Risk Factors; Running; SF-36; Safety; Series; Stem cell transplant; Stem cells; Toxic effect; Transplantation; United States National Institutes of Health; Vulnerable Populations; Work; cohort; design; experience; follow-up; health related quality of life; high risk; information gathering; instrument; international center; member; prevent; programs; prospective; psychologic; psychological outcomes; public health relevance; safety study; symposium; tool; volunteer; willingness

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and readily comparable data regarding the medical and psychological safety of related donors (RDs) is either outdated or lacking, especially in the potentially vulnerable populations of pediatric and older donors (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. PUBLIC HEALTH RELEVANCE: Available studies of related bone marrow or peripheral blood stem cell donor safety provide inadequate information to understand the medical and psychological risks, especially in children and adults above age sixty. This study will take careful measurements of both rare and common medical and psychological side- effects in related donors of all ages and compare the outcomes to unrelated donors to find out if related donors are at increased risk for harm.

描述（由申请人提供）：2006年，北美的8,000多名成人和儿童捐赠了骨髓（BM）或外周血干细胞（PBSC），用于造血干细胞（HSC）移植。这些捐款中的大多数来自接受者（5000/年）的亲戚，这是由关心该收件人的移植中心促进的。其余的捐款来自匿名无关的成人志愿者，由国家骨髓捐助计划（NMDP）进行了协调和仔细的监管。在过去的二十年中，通过NMDP进行的高质量研究产生了有关与HSC捐赠无关的医学和心理毒性的大量数据。相反，有关相关捐助者（RDS）的医学和心理安全性的前瞻性，可量化和易于比较的数据已经过时或缺乏，尤其是在潜在的脆弱的儿科和年长捐助者中（> 60岁）。我们提出的研究（称为RDSAFE，相关的供体安全研究）通过使用NMDP开发的复杂且全面的数据工具将相关HSC捐赠者（UDS）的医学和心理毒性与无关捐助者（UDS）进行比较，从而填补了这一关键数据差距。急性毒性将使用常见（修改的CTC标准）和罕见事件的标准量表进行报告。 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该委员会的当前董事会将与NMDP的供体安全经验和数据工具相结合，由临床研究的统计专业知识，用于血液和骨髓移植（RCI-BMT）。来自儿童肿瘤学组的80多个小儿移植中心已承诺将该队列与同时在70个NMDP供体中心收集的UDS同时进行比较。除其他重大贡献外，从RDSAFE研究中收集的信息将1）定义RDS与UDS的医学和心理结局的差异，并将其与年龄匹配的正：2）分为较低和较高风险的类别，分为较低和较高的风险类别，确定BM VSS的风险差异，3）描述PBS捐赠的风险差异。 RD和5）确定面临医疗或心理风险的捐助者，捐赠会严重损害。公共卫生相关性：有关相关骨髓或外周血干细胞供体安全的可用研究提供了不充分的信息来了解医疗和心理风险，尤其是在60岁以上的儿童和成人中。这项研究将仔细测量各个年龄段的相关捐助者的罕见和常见的医学和心理副作用，并将结果与​​无关的捐助者进行比较，以找出相关捐助者是否有增加伤害风险。