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，但通常具有迟到的风险和 显着损害生活质量（QOL）。虽然可以使用工具来帮助患者和临床医生 预测HCT早期的个性化结果（例如国际血液和骨髓中心 研究（CIBMTR）1年生存计算器）目前几乎完全基于临床和 人口统计学因素，预测生存，但不能预测QOL。尽管最近的研究表明，HCT患者 - 报告的结果（PRO）s均显着预测生存和QOL，几乎没有移植中心常规 收集这些。缺乏系统性专业收集的令人信服的原因包括对 最准确的衡量标准，专业人士提供了有关临床医生判断的新信息和不确定性 关于如何将它们与当前的HCT工具集成在一起。这项研究的目的是通过 从现有的PRO措施中识别一组简短的区分Pro问题，并将其纳入这些问题 变成预测个性化生存和长期质量的工具。我们的三个具体目标是：1）确定 最短的移植前元素集将与生存保持显着关联。 移植并使用它来增强和扩展现有的CIBMTR 1年生存计算器，2） 新的一组计算器，可以预测HCT QOL后1年，3）通过合并两者来预测长期QoL HCT前专业人士和其他HCT后并发症。我们将通过利用 已收集的独特Pro数据，该数据已根据NHLBI资助的前瞻性临床研究收集 CIBMTR以及附属的血液和骨髓移植临床试验网络（BMT CTN）。这些 组织强烈鼓励次要分析，并确定了实现这一目标的机制。 我们将包括七项研究，这些研究使用FACT-BMT，SF36或两者收集了PRO数据。总计 样本为3236，在考虑这种极少数干预措施时，患者数量很大。这个队列捕获了 成年患者在15年内的广泛代表。除专业数据外，每个研究都有 全面且一致收集的临床数据，包括HCT患者和疾病变量， 移植特征和HCT后结果。每项研究都包括基线Pro数据（前HCT）和 优点在多个HCT时间点（最多五年）收集。我们调查的结果将有 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 用于个人预测，包括生存和QOL后HCT。