Trial in Progress: Patient-Reported Outcome Measures in the Prospective Observational Cohort Study of Patients at Risk for Chronic Graft-Versus-Host Disease in the United States (THRIVE)

Introduction Chronic graft-versus-host disease (cGVHD) is the leading cause of morbidity and mortality following allogeneic hematopoietic cell transplantation (alloHCT). The pleomorphic nature of cGVHD makes diagnosis and patient (pt) management challenging; thus, pt-reported symptoms often guide diagnosis and determining cGVHD severity. Current understanding of cGVHD is largely based on data from registries, single-institution prospective studies, and the cGVHD Consortium with representation from tertiary care academic centers. Prospective studies including pt-reported outcomes and more representative and generalizable samples of pts with cGVHD are needed. This trial-in-progress focuses on broad pt-centered assessment of post-alloHCT medical and psychosocial complications and pt-reported caregiver involvement in a diverse and contemporary pt population. This study uses multiple organ- or system-directed pt-reported outcome measures (PROMs) to help identify cGVHD symptoms, severity, and impact on pt quality of life, including financial impact of cGVHD. Objectives To assess the clinical course and burden of cGVHD, including diagnosis and severity recorded in electronic case report forms (eCRFs) and PROMs Study Design and Methods THRIVE is a prospective, longitudinal, observational study (NCT05919511) of alloHCT recipients in the United States that uses a hybrid model designed to work with HCT centers and a virtual platform. Eligible pts are ≥18 years old and received alloHCT 90–180 days before enrollment. Up to 1500 alloHCT recipients will be recruited at HCT sites (expected that ∼500 will have cGVHD symptoms at enrollment; ∼500 estimated to develop cGVHD during the course of the study; ∼500 estimated not to develop cGVHD during the study; Figure). Pts have the option to participate on a virtual platform. Pts will be followed for 36 months from date of enrollment or until the earliest of study termination, withdrawal, or death. Data from 13 PROMs divided into 3 groups will be collected every 3 months using a decentralized virtual platform. To reduce survey burden, pts will be randomized into 1 of 6 sequence groups to capture 4–5 PROMs per month in year 1 and every 2 months in years 2 and 3. Clinical data, including medications and adverse events, will be collected and abstracted into eCRFs every 3 months. In general, reporting of results will be descriptive for variables of interest. Summary statistics for continuous variables and for categorical variables will be calculated for all pts as well as for subgroups of interest. Whenever applicable, 95% CIs will be calculated for endpoint estimates. Univariate, multivariate, or repeated-measurement analyses will be performed to assess factors associated with evolution of clinical manifestations of and complications from cGVHD. Recruitment for this study is ongoing.