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

摘要 造血细胞移植(HCT)可以治愈许多患者，但通常存在晚期效应和 严重损害生活质量(QOL)。虽然可以使用工具来帮助患者和临床医生 预测HCT后早期个体化结局(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 人口统计因素，并预测生存，但不是生活质量。尽管最近的研究表明，HCT前患者- 报道的结果(PRO)S对生存和生活质量都有显著的预测作用，很少有移植中心经常这样做 把这些收集起来。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，怀疑专业人士提供的关于临床医生判断的新信息，以及不确定性 关于如何将它们与当前的HCT工具相结合。这项研究的目标是通过以下方式解决这些不足 从现有的PRO措施中找出一小组有区别的PRO问题，并纳入这些问题 变成预测个性化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 移植前最短的一组促进因子将与移植后的存活率保持显著的相关性 移植并使用它来增强和扩展现有的CIBMTR一年生存计算器，2)开发一个 一套新的计算器，可以预测HCT后一年的生活质量，以及3)通过将两者结合起来预测长期生活质量 HCT前后的优点和其他HCT后的并发症。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 各组织大力鼓励进行二次分析，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或两者都收集了PRO数据。总数 样本为3236例，患者数量非常多，考虑这种罕见的干预措施。这个队列捕获了一个 15年内成人患者的广泛代表性。除了PRO数据外，每项研究都有 全面和持续收集的临床数据，包括HCT前患者和疾病变量， 移植的特点和HCT后的结果。每项研究都包括基线PRO数据(Hct前)，以及 在多个HCT后时间点收集的专业知识(最多五年)。我们的调查结果将会有 通过加强和标准化HCT工具，对接受HCT的患者产生直接和重大的影响 对于个体患者的预测，包括对HCT后生存和生活质量的预测。

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.