Exploring Age Differences in Patient Reported Cognitive Function Trajectories among Hematopoietic Stem Cell Transplant and Chimeric Antigen Receptor T-cell patients and Linking Two Key Measures

探索造血干细胞移植和嵌合抗原受体 T 细胞患者报告的认知功能轨迹的年龄差异，并将两个关键指标联系起来

• 批准号: 10593018
• 负责人: Rachel Nicole Cusatis
• 金额: $ 42.9万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10593018
• 关键词: Address; Adult; Aftercare; Age; Attention; Blood; Bone Marrow Transplantation; CAR T cell therapy; Clinical; Collaborations; Collection; Data; Databases; Disease; Disparity; Elderly; Ensure; Ethnic Origin; European Organization for Research and Treatment of Cancer; Exhibits; Funding; General Population; Health; Hematological Disease; Hematopoietic Stem Cell Transplantation; Household; Impaired cognition; Income; Individual; Information Systems; Infrastructure; Infusion procedures; Link; Lymphoma; Malignant - descriptor; Measurement; Measures; Memory; Mental Depression; Methods; Modeling; Non-Malignant; Outcome; Outcome Measure; Patient Outcomes Assessments; Patients; Pattern; Population; Psyche structure; Psychometrics; Quality of life; Questionnaires; Race; Recommendation; Registries; Reporting; Research; Respondent; Sample Size; Sampling; Sickle Cell Anemia; Site; Socioeconomic Status; Standardization; Subgroup; Survivors; Symptoms; System; Testing; Transplant Recipients; Transplantation; United States; United States National Institutes of Health; Validation; Walking; age difference; chimeric antigen receptor T cells; cognitive function; computerized; curative treatments; ethnic disparity; experience; flexibility; hematopoietic cell transplantation; human old age (65+); international center; leukemia; novel; older patient; patient population; post-transplant; racial disparity; side effect; social; socioeconomic disparity; socioeconomics; survivorship; therapy development; tool; verbal

ABSTRACT Two established therapies to control and/or cure many hematologic diseases are hematopoietic cell transplantation (HCT) and Chimeric antigen receptor t-cell (CAR-T) treatment. In the last few years, there are increasing number of older patients receiving both treatments. Cognitive dysfunction, a common and serious side effect of HCT and CAR-T, was associated with older age. Patient-reported outcomes (PROs), or patient reports of their own symptoms and functioning without interpretation by anyone else, are critical to better understand patients' symptom trajectories, yet very few studies assess PROs among CAR-T patients. Small sample size and single-site studies are limited in their ability to understand potential age differences and even less among racially or socioeconomically diverse older survivors, leading to urgency for PROs from larger more representative data, such as a registry. The Center for International Blood and Marrow Transplant Research (CIBMTR) is an NIH-funded research collaboration that maintains a clinical outcome registry for HCT and CAR-T recipients and, beginning in 2016, developed a strategy and infrastructure for the routine collection of PROs. Patient-Reported Outcomes Measurement Information System (PROMIS) measures were chosen for their flexibility in sets of tools, rigorous testing, and translatability across other diseases and populations. Unlike many other available PRO measures, PROMIS measures are assessed for differences in how items are interpreted across different groups, such as by race/ethnicity and age, known as differential item functioning (DIF). However, the PROMIS cognitive function measure has only been assessed for DIF in 8 of 32 items. Previous research in HCT most often used a different measure to assess cognitive function: European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC). A psychometric analysis known as linking has been used to develop crosswalk tables to directly compare PRO measures, like PROMIS depression, but has yet to link PROMIS to EORTC. Using data on the U.S. general population from an online panel, specific aim 1 will be the first study to use psychometric analysis to assess DIF for the full item bank of PROMIS cognitive function measure by age, race/ethnicity, and income and produce a crosswalk table to directly compare scores on the PROMIS and EORTC cognitive function measures. Using CIBMTR data, specific aim 2 will use linear mixed modeling to assess cognitive function by age and explore potential racial/ethnic and SES disparities among older patients. This project will produce: (1) recommendations for use of cognitive function measures in CIBMTR registry; (2) a crosswalk table directly linking PROMIS and EORTC; (3) a novel description of cognitive function trajectories of HCT and CAR-T patients by age; (4) explore racial/ethnic and socioeconomic disparities in PROs among older HCT and CAR-T patients. This project will provide preliminary data for an R01 proposal to assess disparities in survivorship experience using large registry database and, if necessary, inform intervention development to mitigate such disparities.

抽象的 两种已建立的疗法来控制和/或治愈许多血液疾病是造血细胞 移植（HCT）和嵌合抗原受体T细胞（CAR-T）处理。在过去的几年中，有 接受这两种治疗的老年患者数量增加。认知功能障碍，一种常见和严重的 HCT和CAR-T的副作用与老年有关。患者报告的结果（专业）或患者 他们自身症状和在没有其他任何人解释的情况下发挥作用的报告，对更好的 了解患者的症状轨迹，但很少有研究评估CAR-T患者的专业人士。小的 样本量和单一站点研究的理解能力有限，甚至 在种族或社会经济上多样化的年长幸存者中，较少的人会导致较大的专业人士的紧迫性 更多代表性数据，例如注册表。国际血液和骨髓移植中心 研究（CIBMTR）是一项由NIH资助的研究合作，该合作维持HCT临床结果注册表 和CAR-T接收者，从2016年开始，为常规收集开发了一种策略和基础架构 优点。选择了患者报告的结果测量信息系统（PROMIS）措施 它们在其他疾病和人群中的一组工具，严格的测试以及可翻译性方面的灵活性。与众不同 许多其他可用的Pro措施，对项目的差异进行了评估 跨不同群体解释，例如种族/种族和年龄，称为差异项目功能 （dif）。但是，在32个项目中有8个中，Promis认知函数度量仅对DIF进行了评估。 HCT的先前研究最常使用不同的措施来评估认知功能：欧洲 癌症生活质量调查表（EORTC）研究和治疗组织。心理测量学 被称为链接的分析已用于开发人行横道表，以直接比较Pro措施，例如 Promis抑郁症，但尚未将Promis与EORTC联系起来。使用来自美国普通人群的数据 一个在线面板，特定的目标1将是首次使用心理测量分析评估完整项目DIF的研究 Promis银行认知功能衡量尺寸按年龄，种族/种族和收入量，并产生人行横道表 直接比较Promis和EORTC认知功能度量的分数。使用CIBMTR数据， 特定目标2将使用线性混合建模来评估年龄的认知功能并探索潜力 老年患者的种族/种族和SES差异。该项目将产生：（1）使用建议 CIBMTR注册表中的认知功能度量； （2）直接连接Promis和EORTC的人行横道表； （3）按年龄按HCT和CAR-T患者的认知功能轨迹的新颖描述； （4）探索 老年HCT和CAR-T患者专业人士的种族/种族和社会经济差异。这个项目将 为R01提案提供初步数据，以评估使用大型生存经验的差异 注册表数据库，并在必要时为干预开发提供信息，以减轻此类差异。