Identifying Predictors of Poor Health-Related Quality-of-life among Pediatric Hematopoietic Stem Cell Donors

摘要 这项拟议的纵向调查建立在并极大地扩展了我们的初步工作，重点是 同胞儿科造血干细胞捐献者的健康相关生活质量(HRQOL)。 尽管使用未成年人作为造血干细胞捐赠者在医学上和法律上都是可以接受的，但最近美国政府的政策声明 美国儿科学会和我们自己的初步工作发现儿科捐赠者的HRQOL缺陷 这表明在这一领域进行更多的研究是至关重要的。我们的初步工作发现，~20%的健康 儿科捐赠者在前不久和一年后经历了临床上重要的HRQL缺陷 捐款。此外，父母高估了捐献儿童的HRQL，这种高估是 尤其是对于自我报告的HRQOL最差的儿童。这项提议的首要目标是 调查的目的是在一些兄弟姐妹的儿科HSC捐献者中确定不良的HRQOL的预测因素。此外, 我们将确定最具生产力的潜在目标，以及干预措施或指南更改的内容 提高人力资源质量。这项研究将产生一个新的、大量的兄弟姐妹儿科捐赠者、他们的接受者和 照顾者和三个非捐赠者对照组。我们将在捐赠前和4周内进行采访， 捐献后6个月和1年，409名兄弟姐妹的儿科HSC捐赠者、他们的接受者和父母以及 三个关键的比较组-来自同一家庭的所有5-18岁的非捐赠者兄弟姐妹， 接受无关移植的患者，以及健康比较的匹配样本。医疗数据来自 捐赠者、接受者和移植中心的数据将被收集并与采访数据合并。分析焦点 关于现有文献中的假设，我们自己的初步工作，以及一个多变量模型 确定哪些捐赠者、受赠者、家庭和中心特征可以预测捐赠者的HRQL。这项调查 之所以意义重大，是因为(A)儿童造血干细胞捐赠正在增加，(B)它侧重于健康的弱势群体 儿童被要求在没有直接利益的情况下承受潜在的医疗/心理风险；(C) 提供有关儿科捐赠者的不良HRQOL预测因素的关键信息，(D)将理清 观察到的HRQOL缺陷是否是由于捐赠过程、家庭动态和/或其他原因 (E)这将为指导方针/干预措施提供基础，以减轻 捐献者不良的HRQOL风险。这项调查是创新的，因为它将是第一次审查 在全国范围内的大样本中的儿童造血干细胞捐献者的HRQOL，(B)提供了与 非捐献者儿童，(C)使用基于群体的轨迹建模的创新应用程序来检查捐赠者 (D)综合评价供者/受者/家庭/移植因素对儿童的影响 捐助者的HRQOL和(E)包括大量不同族裔和讲西班牙语的样本。这项调查将 促进兄弟姐妹儿科HSC捐献者及其家人对HRQOL的了解并提供 为这一群体制定指导方针/干预措施的关键信息。

ABSTRACT This proposed longitudinal investigation builds on, and significantly extends, our preliminary work focused on health-related quality-of-life (HRQoL) among sibling pediatric hematopoietic stem cell (HSC) donors. Although the use of minors as HSC donors is medically and legally accepted, recent policy statements by the American Academy of Pediatrics and our own preliminary work which found pediatric donor HRQoL deficits indicate that additional research in this area is critical. Our preliminary work found that ~20% of healthy pediatric donors experienced clinically important HRQoL deficits shortly before and up to one year following donation. In addition, parents over-estimated HRQoL of the donor child and this overestimation was particularly large for children with the poorest self-reported HRQoL. The overarching goal of this proposed investigation is to identify predictors of poor HRQoL among some sibling pediatric HSC donors. In addition, we will identify the most productive potential targets for, and content of, interventions or guideline changes to improve HRQoL. The study will generate a new, large cohort of sibling pediatric donors, their recipients and caregivers and three non-donor comparison groups. We will conduct interviews at pre-donation, and 4 weeks, 6 months and 1 year post-donation with 409 sibling pediatric HSC donors, their recipients and parents and three key comparison groups – all nondonor siblings age 5-18 from the same family, nondonor siblings of patients receiving unrelated transplants, and a matched sample of healthy comparisons. Medical data from donors, recipients and transplant center data will be collected and merged with interview data. Analyses focus on hypotheses derived from the existing literature, our own preliminary work, and a multivariable model to determine which donor, recipient, family and center characteristics predict donor HRQoL. This investigation is significant because (a) pediatric HSC donation is increasing, (b) it focuses on a vulnerable group of healthy children being asked to undergo potential medical/psychological risk with no direct self-benefit, (c) it will provide critical information about predictors of poor HRQoL among pediatric donors, (d) it will disentangle whether observed HRQoL deficits are due to the donation process, to family dynamics, and/or to some other aspect of having a critically ill sibling, and (e) it will provide the basis for guidelines/interventions to mitigate the risk of poor donor HRQoL. The investigation is innovative because it will be the first to (a) examine pediatric HSC donor HRQoL in a large nationwide sample, (b) provide important and novel comparisons with nondonor children, (c) use an innovative application of group-based trajectory modeling to examine donor HRQoL, (d) comprehensively evaluate the effects of donor/recipient/family/transplant factors on pediatric donor HRQoL and (e) include a large ethnically diverse and Spanish-speaking sample. This investigation will advance our understanding of HRQoL among sibling pediatric HSC donors and their families and provide crucial information for the development of guidelines/interventions for this group.