Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis



Poster Number: 116


Christian Werner, Dr. med., PTC Therapeutics, Már Tulinius, Department of Pediatrics, Gothenburg University, Queen Silvia Children’s Hospital, Filippo Buccella, Parent Project APS, Isabelle Desguerre, MD, PhD, Hôpital Necker Enfants Malades, APHP, Janbernd Kirschner, PhD, University Medical Center Freiburg, Eugenio Mercuri, MD, PhD, Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Francesco Muntoni, MD, UCL Institute of Child Health and Great Ormond Street Hospital for Children, Andres Nascimento Osorio, MD, Hospital Sant Joan de Déu. U.B., Lauren Morgenroth, MS, CGC, TRiNDS, Heather Gordish-Dressman, PhD, Children’s National Hospital, Shelley Johnson, DBA, MBA, PTC Therapeutics Inc, Pannie (Panayiota) Trifillis, PhD, PTC Therapeutics, Inc., Craig McDonald, MD, University of California Davis Health

Background: Strategic Targeting of Registries and International Database of Excellence (STRIDE; NCT02369731) is an ongoing, multicenter, observational registry providing data on ataluren use in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients in routine clinical practice.

Objectives: We investigated if nmDMD patients receiving ataluren plus standard of care (SoC) in the STRIDE Registry experienced a lesser decline in pulmonary function versus DMD patients receiving SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Natural History Study (NCT00468832).

Methods: Data were extracted on January 31, 2022. Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use. Patients from CINRG who had received investigational drugs for DMD were excluded from this analysis. Kaplan–Meier analyses were used to estimate ages at %-predicted forced vital capacity (FVC) <60% and <30%. Results: The mean (standard deviation) ages at onset of first symptoms (STRIDE vs CINRG; N=260 per cohort) were 2.8 (1.7) and 2.8 (1.5) years, respectively. Most patients (STRIDE vs CINRG) received corticosteroids for ≥12 months (85.0% vs 83.8%), with a similar proportion receiving deflazacort (47.7% vs 44.2%) or other corticosteroids (41.9% vs 43.5%). Median (95% confidence interval [CI]) ages at %-predicted FVC <60% (STRIDE vs CINRG) were 17.7 (16.8, not estimable) and 15.3 (14.9, 16.5) years, respectively (p=0.0053). Median (95% CI) ages at %-predicted FVC <30% (STRIDE vs CINRG) were not estimable and 22.5 (20.3, 25.4) years, respectively (p=0.0008). Conclusions: These interim registry data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients.