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

Medical College Of Wisconsin, Milwaukee WI

Investigators

Abstract

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.