Patient-Reported Outcomes From a Phase 3 Study of Givinostat in Patients With Duchenne Muscular Dystrophy
Author(s)
Laura McAdam, MSc, MD, FRCPC1, Federica Alessi, MSc2, Nuria Muelas Gómez, MD3, Liesbeth De Waele, Prof. Dr.4, Katiana Gruppioni, MPH5, Sara Cazzaniga, Msc2, Paolo Bettica, MD, PhD2, Craig Zaidman, MD6;
1Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada, 2Italfarmaco SpA., Milan, Italy, 3Hospital Universitari I Politècnic La Fe, Neuromuscular Diseases Unit, Neurology Department, Valencia, Spain, 4University Hospitals Leuven, Neuromuscular Reference Centre for Children, Department of Paediatrics, Leuven, Belgium, 5ITF Therapeutics, Concord, MA, USA, 6Washington University School of Medicine, Department of Neurology, St Louis, MO, USA
1Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada, 2Italfarmaco SpA., Milan, Italy, 3Hospital Universitari I Politècnic La Fe, Neuromuscular Diseases Unit, Neurology Department, Valencia, Spain, 4University Hospitals Leuven, Neuromuscular Reference Centre for Children, Department of Paediatrics, Leuven, Belgium, 5ITF Therapeutics, Concord, MA, USA, 6Washington University School of Medicine, Department of Neurology, St Louis, MO, USA
Presentation Documents
OBJECTIVES: Givinostat, an oral histone deacetylase inhibitor, was recently approved in the US and UK for Duchenne muscular dystrophy (DMD) treatment in patients aged ≥6 years. These analyses evaluated patient-reported outcome (PRO) data from the double-blind, randomized, phase 3 EPIDYS study, which compared the efficacy and safety of givinostat versus placebo in ambulant boys (aged ≥6 years) with DMD (NCT02851797).
METHODS: The PRO instrument in EPIDYS was the Pediatric Outcome Data Collection Instrument (PODCI), commonly used to evaluate quality of life (QoL) in patients with DMD. The PODCI comprises a global function score and 5 subscales: upper extremity (UE) function, transfer and basic mobility (TBM), sports/physical function (SPF), pain/comfort (PC), and happiness (HAP). Scores range from 0 to 100, with higher scores indicating better QoL. PODCI was completed by participants or proxy in EPIDYS at baseline and months 12 and 18. Results are based on descriptive statistics only. All patients received corticosteroids during the study.
RESULTS: The baseline mean (SD) PODCI global function scores were 77.2 (12.5) for givinostat (n=81) and 76.9 (12.9) for placebo (n=39). At month 18, mean (SD) PODCI global function scores were 72.7 (15.3) for givinostat and 69.0 (15.9) for placebo, indicating less decline for givinostat-treated patients compared to the placebo group. At 18 months, mean (SD) subscale scores were higher for givinostat than placebo for UE (80.7 [14.4] vs 76.7 [15.6], respectively), TBM (80.5 [16.4] vs 76.7 [17.1]), SPF (49.9 [21.6] vs 46.1 [22.2]), PC (79.6 [19.5] vs 76.6 [20.8]), and HAP (73.4 [19.3] vs 68.8 [25.2]).
CONCLUSIONS: Patients treated with givinostat showed smaller reductions in QoL across all PODCI global scores and subscales compared with placebo. These data suggest that givinostat provides a benefit in slowing down the decline in QoL in patients with DMD.
METHODS: The PRO instrument in EPIDYS was the Pediatric Outcome Data Collection Instrument (PODCI), commonly used to evaluate quality of life (QoL) in patients with DMD. The PODCI comprises a global function score and 5 subscales: upper extremity (UE) function, transfer and basic mobility (TBM), sports/physical function (SPF), pain/comfort (PC), and happiness (HAP). Scores range from 0 to 100, with higher scores indicating better QoL. PODCI was completed by participants or proxy in EPIDYS at baseline and months 12 and 18. Results are based on descriptive statistics only. All patients received corticosteroids during the study.
RESULTS: The baseline mean (SD) PODCI global function scores were 77.2 (12.5) for givinostat (n=81) and 76.9 (12.9) for placebo (n=39). At month 18, mean (SD) PODCI global function scores were 72.7 (15.3) for givinostat and 69.0 (15.9) for placebo, indicating less decline for givinostat-treated patients compared to the placebo group. At 18 months, mean (SD) subscale scores were higher for givinostat than placebo for UE (80.7 [14.4] vs 76.7 [15.6], respectively), TBM (80.5 [16.4] vs 76.7 [17.1]), SPF (49.9 [21.6] vs 46.1 [22.2]), PC (79.6 [19.5] vs 76.6 [20.8]), and HAP (73.4 [19.3] vs 68.8 [25.2]).
CONCLUSIONS: Patients treated with givinostat showed smaller reductions in QoL across all PODCI global scores and subscales compared with placebo. These data suggest that givinostat provides a benefit in slowing down the decline in QoL in patients with DMD.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR230
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)