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: 7581672
• 负责人: Michael A Pulsipher
• 金额: $ 70.14万
• 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/7581672
• 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and readily comparable data regarding the medical and psychological safety of related donors (RDs) is either outdated or lacking, especially in the potentially vulnerable populations of pediatric and older donors (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，北美超过8,000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。这些捐赠大部分来自接受者的亲属（每年 5000 份），并由照顾该接受者的移植中心提供便利。其余的捐赠来自匿名、无关的成年志愿者，由国家骨髓捐赠计划 (NMDP) 协调和仔细监管。在过去的二十年中，通过 NMDP 进行的高质量研究已经获得了大量有关与无关的 HSC 捐赠相关的医学和心理毒性的数据。相反，有关相关捐赠者 (RD) 医疗和心理安全的前瞻性、可量化和易于比较的数据要么已经过时，要么缺乏，特别是在儿科和老年捐赠者（> 60 岁）的潜在弱势群体中。我们提出的研究（称为 RDSafe，相关捐献者安全研究）通过使用 NMDP 开发的复杂且全面的数据工具比较相关 HSC 捐献者与无关捐献者 (UD) 的医疗和心理毒性，填补了这一关键数据空白。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来报告急性毒性。将使用经过充分验证的标准化健康相关生活质量 (HRQoL) 工具（针对成人的 SF-36 和针对儿童的 PedsQL" 4.0）来评估捐赠者，并将与年龄匹配的正常人以及相关与不相关的捐赠者进行比较。该研究由国际血液和骨髓移植研究中心 (CIBMTR) 捐赠者健康与安全工作委员会的成员开发：该提案的 PI 是该委员会的现任联合主席。 NMDP 的捐赠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。该研究旨在提高儿科和老年 RD 的累积率。为了收集足够的数据来提供针对这些群体的毒性风险概况，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招募 RD。与在 70 多个 NMDP 捐赠中心收集的同时登记的 UD 队列进行比较。除其他重大贡献外，从 RDSafe 研究收集的信息将 1) 定义 RD 与 UD 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 人群分为较低风险和较高风险类别，识别捐赠者的弱势群体，3）描述所有年龄段的 RD 中 BM 与 PBSC 捐赠风险的差异，4）定义 RD 知情同意的标准，以及 5）识别处于医疗或捐赠造成严重伤害的心理风险。公共卫生相关性：现有的相关骨髓或外周血干细胞捐献者安全性研究提供的信息不足以了解医疗和心理风险，特别是对于六十岁以上的儿童和成人。这项研究将仔细测量所有年龄段的相关捐赠者的罕见和常见的医疗和心理副作用，并将结果与​​非相关捐赠者进行比较，以确定相关捐赠者受到伤害的风险是否增加。