Paper Details

ABSTRACT

Background & Significance: Although the risk of developing chronic graft-versus-host disease (cGVHD) after hematopoietic cell transplant (HCT) has declined in recent years, important gaps remain in the care of patients living with this condition. Virtually no published studies have tested the effectiveness of interventions to help survivors manage the complex and disruptive medical and psychological 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. 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 for patients with cGVHD. HORIZONS combines medical education and psychosocial skills to improve self-management of cGVHD and quality of life (QOL). This intervention model is scalable, accessible, and evidence-based, with promise to address the immense challenges faced by individuals living with cGVHD. 

Study Design & Methods: We have initiated a multi-site, hybrid type-I randomized effectiveness-implementation trial of HORIZONS compared with minimally enhanced usual care to improve patient QOL (primary outcome), psychological distress, cGVHD symptom burden, coping, and self-efficacy (NCT06910969). The target sample is 350 adult (age ≥ 18) allogeneic HCT survivors with self-reported moderate to severe cGVHD who can participate in an English or Spanish language group telehealth intervention. Patients are being recruited via two academic medical centers (University of Miami Sylvester Comprehensive Cancer Center [UM] and Massachusetts General Hospital [MGH]) and via coordination with patient advocacy and support organizations (e.g., Blood Cancer United, BMT InfoNet, Meredith Cowden Foundation, GVHD Alliance, National Bone Marrow Transplant LINK). Participants complete a baseline survey and then are randomly assigned to the HORIZONS program or enhanced usual care, with randomization stratified by recruitment source (UM, MGH, or patient advocacy and support organizations) and preferred language (English, Spanish). Participants assigned to the HORIZONS program are invited to participate in eight weekly 90-minute structured telehealth intervention sessions (6-8 patients per group). The HORIZONS program is co-led by a behavioral health clinician and a bone marrow transplant clinician. Sessions include psychoeducation about cGVHD and its treatments, skills for managing cGVHD and maintaining QOL, and facilitated peer support, with patients encouraged to set between-session goals. To ensure fidelity, trained study interventionists attend a weekly supervision meeting and complete weekly electronic surveys indicating the topics and exercises addressed during the intervention session. Outcomes are assessed via self-report at baseline, 10, and 18 weeks. We will assess facilitators and barriers to HORIZONS implementation, adoption and maintenance. During the first 10 months of recruitment (8/2025-6/2026), we have enrolled 72 patients (17 UM, 39 MGH, 16 patient advocacy and support organizations; 56% Female; 21% Hispanic/Latino; 72% White, 15% Black, 6% Asian, 3% Middle Eastern, 3% Multi-Racial, 1% Indigenous American). 

Analysis: The primary outcome is patient QOL scores between study groups at the 10-week follow-up using the Functional Assessment of Cancer Therapy-Bone Marrow Transplant measure. Secondary outcomes include psychological distress (Hospital Anxiety and Depression Scale) and self-management targets (coping skills: Measure of Current Status-Part A, self-efficacy: Cancer Self-Efficacy Scale-Transplant, symptom burden: Lee Chronic Graft-Versus-Host Disease Symptom Scale). Analyses will be conducted in accordance with the intention-to-treat principle. We will fit linear mixed models (with random effects accounting for the group-based intervention) for each endpoint at 10 weeks and then will examine change across all timepoints (baseline, 10 weeks, 18 weeks) using longitudinal mixed modeling. We will identify critical facilitators and barriers for HORIZONS implementation and adoption using the RE-AIM QuEST mixed-methods framework. 

Conclusion: This study will provide critical evidence for the effectiveness and implementation of a scalable telehealth intervention to improve survivorship care for patients with cGVHD recruited nationwide.