Randomized Trial of a Multi-Disciplinary Intervention for Patients with Chronic Grant-Versus-Host Disease

PROJECT SUMMARY Overview: The goal of this study is to demonstrate the efficacy of a multidisciplinary group-based telehealth intervention (HORIZONS) compared to minimally enhanced usual for hematopoietic stem cell transplant (HCT) survivors living with chronic graft-versus-host disease (cGVHD). Background: Allogeneic HCT is a commonly used treatment that offers a potentially curative therapy for patients with hematologic malignancies. Over half of long-term HCT survivors develop cGVHD, a debilitating immunologic syndrome that attacks multiple organs and is a major cause of post-HCT morbidity and mortality. Virtually no published studies have tested the effectiveness of interventions to assist long term survivors in coping with the complex and disruptive sequelae of cGVHD. Moreover, immune vulnerability, symptom burden, and geographic distance from transplant centers challenge the implementation of on-site survivorship care for this population. This study addresses a priority research gap identified by the National Institutes of Health consensus panel on HCT late effects: the need to assist HCT survivors with self-management. Based on our prior work and a conceptual self-management framework, we developed and established the feasibility and preliminary efficacy of HORIZONS, a multidisciplinary group-based telehealth intervention combining medical and psychosocial expertise to improve self-management skills and quality of life in patients with cGVHD. Research Plan: We propose to conduct a hybrid type I multi-site randomized effectiveness-implementation trial in 350 patients with cGVHD to test the efficacy of HORIZONS vs. minimally enhanced usual care for improving patient QOL, psychological distress, cGVHD symptom burden, coping and self-efficacy. We will also identify critical facilitators and barriers for HORIZONS implementation and adoption using the RE-AIM QuEST mixed- methods framework. Environment: This project will be conducted with demographically and geographically diverse patients recruited via a national advocacy organization (Blood & Marrow Transplant Information Network [BMT InfoNet]) and transplant centers at two academic medical centers (University of Miami [Sylvester] and Massachusetts General Hospital). These sites have the supportive oncology research infrastructure, transplant experience, and processes to ensure the success of this trial. The research team has the expertise in developing and testing multi-site palliative and supportive care interventions for patients with hematologic malignancies and those undergoing HCT to ensure the successful implementation and evaluation of HORIZONS across study sites. Relevance of Research: This project will establish the essential foundation for a future implementation and dissemination trial of HORIZONS into clinical practice to transform survivorship care across regions and settings.