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我们的初步工作的基础上，并大大扩展了我们的初步工作，重点是 同胞儿童造血干细胞（HSC）供体的健康相关生活质量（HRQoL）。 虽然使用未成年人作为HSC供体在医学上和法律上都是可以接受的，但最近的政策声明， 美国儿科学会和我们自己的初步工作，发现儿科供体HRQoL缺陷 这表明在这一领域开展更多研究至关重要。我们的初步研究发现，约20%的健康 儿科供体在移植前不久和移植后一年内出现了临床上重要的HRQoL缺陷 捐赠。此外，父母高估了捐赠儿童的HRQoL，这种高估是 对于自我报告的HRQoL最差的儿童尤其如此。本建议的总体目标是 研究的目的是确定一些同胞儿童HSC供体中HRQoL较差的预测因素。此外，本发明还提供了一种方法， 我们将确定最有成效的潜在干预目标和内容， 改善HRQoL。这项研究将产生一个新的，大的兄弟姐妹儿科捐赠者，他们的受体和 护理人员和三个非捐赠者对照组。我们将在捐赠前和4周内进行面试， 409名同胞儿童HSC供体、其受体和父母在捐献后6个月和1年， 三个关键的比较组-来自同一家庭的所有5 - 18岁的非供体兄弟姐妹， 接受无关移植的患者，以及健康对照的匹配样本。医疗数据从 将收集捐赠者、接受者和移植中心的数据，并与访谈数据合并。分析重点 假设来自现有的文献，我们自己的初步工作，和一个多变量模型， 确定哪些供体、受体、家庭和中心特征可预测供体HRQoL。这次调查 这是重要的，因为（a）儿科HSC捐赠正在增加，（B）它专注于健康的弱势群体， 儿童被要求承受潜在的医疗/心理风险，没有直接的自身利益，（c）它将 提供关于儿童供体中HRQoL较差预测因素的关键信息，（d）它将解开 观察到的HRQoL缺陷是否是由于捐赠过程，家庭动力学和/或其他一些原因 （e）它将为指导方针/干预措施提供基础，以减轻 供体HRQoL较差的风险。这项调查是创新的，因为它将是第一个（a）审查 在全国范围内的大样本中，儿科HSC供体HRQoL，（B）提供了重要和新颖的比较， 非供体儿童，（c）使用基于组的轨迹建模的创新应用程序来检查供体 HRQoL，（d）全面评估供者/受者/家庭/移植因素对儿童的影响 （e）包括一个大的种族多样性和讲西班牙语的样本。这次调查将 提高我们对同胞儿童HSC供体及其家属HRQoL的理解，并提供 为这一群体制定指导方针/干预措施提供重要信息。

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.