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

确定儿童造血干细胞捐献者健康相关生活质量不佳的预测因素

• 批准号: 10319994
• 负责人: Michael A Pulsipher
• 金额: $ 67.32万
• 依托单位: UNIVERSITY OF PITTSBURGH AT PITTSBURGH
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-01-06 至 2024-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10319994
• 关键词: Academy; Address; Advocate; Affect; Age; American; Anxiety; Area; Blood; Blood Component Removal; Caregivers; Cells; Characteristics; Child; Child Health; Childhood; Chronic; Clinical; Critical Illness; Data; Development; Disease; Ethnic Origin; Family; Family Characteristics; Family dynamics; Funding; Gender; Goals; Guidelines; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Infusion procedures; Intervention; Interview; Investigation; Legal; Literature; Medical; Mental Depression; Minor; Modeling; National Heart, Lung, and Blood Institute; Operative Surgical Procedures; Outcome; Parents; Patient Self-Report; Patients; Pattern; Pediatrics; Policies; Predictive Factor; Procedures; Process; Publishing; Quality of life; Race; Reporting; Research; Review Literature; Risk; Safety; Sampling; Self-Direction; Siblings; Single Parent; Stress; Stressful Event; Telephone Interviews; Time; Transplantation; Treatment/Psychosocial Effects; Umbilical Cord Blood Transplantation; Vulnerable Populations; Work; base; chronic graft versus host disease; cohesion; cohort; compare effectiveness; comparison group; disorder risk; donor stem cell; emotional experience; ethnic diversity; experience; group intervention; health related quality of life; improved; innovation; mortality; novel; post-transplant; psychologic; psychosocial; stem cells; therapy design; transplant centers; treatment comparison

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.

抽象的 这项拟议的纵向调查基于并大大扩展了我们的初步工作 兄弟小儿造血干细胞（HSC）供体中与健康相关的生活质量（HRQOL）。 尽管将未成年人用作HSC捐助者是在医学和法律上接受的，但最近的政策声明 美国儿科学院和我们自己的初步工作，发现儿科供体HRQOL定义 表明该领域的其他研究至关重要。我们的初步工作发现约有20％的健康 小儿捐赠者经历了临床上重要的HRQOL在不久和后不久的一年中定义的 捐款。此外，父母过高估计的捐赠者的hrqol，这高估了 对于自我报告的HRQOL最贫穷的儿童来说，特别是很大。该提议的总体目标 研究是为了确定一些兄弟小儿HSC捐赠者中HRQOL差的预测因子。此外， 我们将确定干预措施或指南的最大潜在目标和内容的更改 改善HRQOL。这项研究将产生一个新的，大量的兄弟姐妹小儿捐助者，他们的接受者和 护理人员和三个非主持人比较组。我们将在预期和4周的情况下进行采访 奉献后6个月零1年，有409个兄弟小儿HSC捐助者，他们的收件人和父母， 三个关键的比较组 - 所有非副兄弟姐妹5-18岁，来自同一家庭 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 将收集捐助者，接收者和移植中心数据，并与访谈数据合并。分析重点 关于来自现有文献，我们自己的初步工作以及多变量模型的假设 确定哪个捐助者，接受者，家庭和中心特征可以预测供体HRQOL。这项调查 之所以重要，是因为（a）小儿HSC捐赠正在增加，（b）它的重点是脆弱的健康组 被要求儿童在没有直接自我效力的情况下经历潜在的医疗/心理风险，（c） 提供有关小儿捐赠者中HRQOL差的预测指标的关键信息，（d）将解开 观察到的HRQOL是否定义是由于捐赠过程，家庭动态和/或其他一些 患有重病的兄弟姐妹的方面，（e）它将为减轻指导/干预的基础 捐助者HRQOL的风险。投资具有创新性，因为它将是第一个（a）检查 小儿HSC供体HRQOL在全国范围的大型样本中，（b）提供了重要而新颖的比较 Nondonor儿童，（c）使用基于组的轨迹建模的创新应用来考试捐助者 HRQOL，（d）全面评估供体/接受者/家庭/移植因子对小儿的影响 捐助者HRQOL和（e）包括一个大量的种族多样性和讲西班牙语的样本。这项调查会 促进我们对兄弟小儿HSC捐助者及其家人中HRQOL的了解，并提供 为该小组制定准则/干预措施的关键信息。

项目成果

Pediatric HCT in Florida (2014 -2016): A report from the FPBCC.

佛罗里达州儿科 HCT（2014 年 -2016 年）：来自 FPBCC 的报告。

• DOI: 10.1111/petr.13931
• 发表时间: 2021
• 期刊: Pediatric transplantation
• 影响因子: 1.3
• 作者: Chellapandian,Deepak;Sunkersett,Gauri;Oshrine,Benjamin;GalvezSilva,Jorge;Ziga,Edward;Alperstein,Warren;Joyce,Michael;Katzenstein,Howard;Godder,Kamar;Castillo,Paul;Barredo,Julio;Fort,John;Shaw,PeterH;Nieder,MichaelL;Cline,Je
• 通讯作者: Cline,Je