Quality of Life (QoL) in Pediatric Patients Treated with Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease (aGVHD) Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT): Analysis of Msb-GVHD001 Phase 3 Study

Abstract

Introduction MSB-GVHD001, a single-arm phase 3 study, evaluated the efficacy of up to 8 weeks of remestemcel-L treatment in 54 pediatric patients with aGVHD following HSCT. Objective This is a post-hoc analysis of health-related QoL data. Methods QoL was assessed in 24 patients using the Pediatric Quality of Life Inventory 3.0 Cancer Module Scale (PedsQL™) and Karnofsky/Lansky performance status (K/LPS) at baseline (BL) and days 28, 56, and 100. PedsQL consists of 27 items divided over 8 domains: Pain/Hurt, Nausea, Procedural Anxiety, Treatment Anxiety, Worry, Cognitive Problems, Perceived Physical Appearance, and Communication. All scores for PedsQL were transformed to a 0 to 100 scale, and descriptive statistics were generated at each visit, by dimension and globally; higher results indicate better QoL. The relationship between overall response rate (ORR) and QoL at Visit 28 was evaluated using one-way analysis of variance (ANOVA). The difference between groups was based on pairwise comparison (t-distribution) of marginal linear predictions. Results PedsQL scores at baseline were poor compared with published values in children with cancer, with >10 points difference in the Pain/Hurt, Nausea, and Worry domains, highlighting the additional burden of GVHD. Performance status scores at BL showed a high proportion of children experiencing moderate to severe restrictions of activities (43% in 8-12 year-olds, 58% in 13-17 year-olds). Remestemcel-L treatment was associated with improvements in Pain/Hurt, Nausea (>10 points), and Procedural Anxiety, while scores in the remaining domains were relatively stable, except for decline in Cognitive Problems. Of the 24 patients, 71% achieved ORR at Day 28 and experienced an improvement in Global QoL score (70.6) vs. deterioration in patients without response (62.6) vs. BL (66.4). ANOVA analysis showed >10 points difference in patients with vs. without ORR in Procedural Anxiety, Treatment Anxiety, Worry, and Communication. Conclusion Patients with steroid-refractory aGVHD demonstrating ORR while on remestemcel-L treatment achieved improvements in QoL from BL to day 28. Prior publications reported similar changes in PedsQL domain scores for children who rated their health as improved during remission 1 year after treatment. Results should be interpreted with caution due to the small sample size. Sponsorship Mesoblast