Incorporating patient reported outcomes into individualized prognostication tools for survival and quality of life in transplant patients

将患者报告的结果纳入移植患者生存和生活质量的个体化预测工具中

• 批准号: 9591693
• 负责人: Bronwen Shaw
• 金额: $ 12.38万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-08-01 至 2020-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9591693
• 关键词: Address; Adult; Agreement; Algorithms; Allogenic; Autologous; Blood; Caring; Characteristics; Clinical; Clinical Data; Clinical Research; Clinical Trials; Clinical Trials Network; Collection; Data; Data Set; Demographic Factors; Disease; Elements; Funding; Future; Goals; Heart Diseases; Hematological Disease; Impairment; Intervention; Investigation; Judgment; Knowledge; Late Effects; Life; Long-Term Survivors; Lung diseases; Marrow; Measures; Modernization; National Heart, Lung, and Blood Institute; Outcome; Outcome Measure; Patient Outcomes Assessments; Patients; Predictive Value; Prospective Studies; Quality of life; Reporting; Research; Research Design; Research Personnel; Resources; Respondent; Risk; SF-36; Sampling; Sleep Disorders; Standardization; Survivors; Time; Training; Transplant Recipients; Transplantation; Uncertainty; Validation; Wisconsin; base; cohort; cost; hematopoietic cell transplantation; individual patient; international center; medical schools; novel; patient population; personalized predictions; primary endpoint; prognostic; prognostic of survival; prognostic tool; prospective; secondary analysis; survival outcome; survival prediction; tool; transplant centers; user-friendly

ABSTRACT Hematopoietic cell transplantation (HCT) can cure many patients, but often with the risk of late effects and significantly impaired quality of life (QOL). Whilst tools are available to assist patients and clinicians in predicting individualized outcomes early post-HCT (such as the Center for International Blood and Marrow Research (CIBMTR) 1-year survival calculator), these are currently almost exclusively based on clinical and demographic factors, and predict survival, but not QOL. Despite recent studies showing that pre-HCT patient- reported outcome (PRO)s are significantly predictive of both survival and QOL, few transplant centers routinely collect these. Compelling reasons for the lack of systematic PRO collection include lack of agreement on the most accurate measure, skepticism that PROs offer novel information over clinician judgment, and uncertainty regarding how to integrate them with current HCT tools. The goal of this study is to address these deficits by identifying a short set of discriminating PRO questions, from existing PRO measures, and to incorporate these into tools to predict individualized survival and long term QOL. Our 3 specific aims are: 1) determine the shortest set of pre-transplant PRO elements which will maintain a significant association with survival post- transplant and use this to enhance and extend the existing CIBMTR 1-year survival calculator, 2) develop a new set of calculators which predict 1-year post-HCT QOL, and 3) predict long-term QOL by incorporating both pre-and post-HCT PROs and other post-HCT complications. We will pursue these aims by leveraging the unique PRO data which has been collected on prospective clinical studies performed by the NHLBI-funded CIBMTR and the affiliated Blood and Marrow Transplant Clinical Trials Network (BMT CTN). These organizations strongly encourage secondary analyses and have defined mechanisms by which to achieve this. We will include seven studies which collected PRO data using either the FACT-BMT, SF36 or both. The total sample is 3236, a very large number of patients when considering this rare intervention. This cohort captures a broad representation of adult patients over a 15-year period. In addition to PRO data each study has comprehensive and consistently collected clinical data, including pre-HCT patient and disease variables, transplant characteristics and post-HCT outcomes. Every study includes baseline PRO data (pre-HCT), and PROs collected at multiple post-HCT time points (up to five years). The results from our investigation will have an immediate and significant impact on patients undergoing HCT by enhancing and standardizing HCT tools for individual patient prognostication, both for survival and QOL post-HCT.

抽象的 造血细胞移植（HCT）可以治愈许多患者，但通常存在迟发效应和 生活质量（QOL）显着受损。虽然有工具可以帮助患者和临床医生 HCT 后早期预测个体化结果（例如国际血液和骨髓中心 研究（CIBMTR）1年生存计算器），这些目前几乎完全基于临床和 人口因素，可以预测生存率，但不能预测生活质量。尽管最近的研究表明 HCT 前患者- 报告的结果 (PRO) 可显着预测生存率和生活质量，很少有移植中心定期进行 收集这些。缺乏系统的 PRO 收集的令人信服的原因包括缺乏对 最准确的测量、对 PRO 提供的新颖信息超过临床医生判断的怀疑以及不确定性 关于如何将它们与当前的 HCT 工具集成。本研究的目标是通过以下方式解决这些缺陷 从现有的 PRO 措施中确定一组简短的歧视性 PRO 问题，并将这些问题纳入其中 转化为预测个体生存和长期生活质量的工具。我们的 3 个具体目标是：1) 确定 移植前 PRO 元件的最短集合，将与术后生存保持显着关联 移植并使用它来增强和扩展现有的 CIBMTR 1 年生存计算器，2）开发一个 一套新的计算器，可预测 HCT 后 1 年的生活质量，3) 通过结合两者来预测长期生活质量 HCT 前后的 PRO 以及其他 HCT 后并发症。我们将通过利用 由 NHLBI 资助的前瞻性临床研究收集的独特 PRO 数据 CIBMTR 和附属的血液和骨髓移植临床试验网络 (BMT CTN)。这些 组织强烈鼓励二次分析，并已定义实现这一目标的机制。 我们将纳入七项使用 FACT-BMT、SF36 或两者收集 PRO 数据的研究。总计 样本为 3236 名，在考虑这种罕见的干预措施时，患者数量非常大。该队列捕获了 15 年期间成年患者的广泛代表性。除了 PRO 数据外，每项研究还 全面且一致收集的临床数据，包括 HCT 前患者和疾病变量， 移植特征和 HCT 后结果。每项研究均包括基线 PRO 数据（HCT 前），以及 在 HCT 后的多个时间点（最长五年）收集的 PRO。我们的调查结果将会 通过增强和标准化 HCT 工具，对接受 HCT 的患者产生直接而重大的影响 用于个体患者的 HCT 后生存和生活质量预测。