Dados do Trabalho

Título

PULMONARY FUNCTION IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY FROM THE STRIDE REGISTRY AND CINRG NATURAL HISTORY STUDY: A MATCHED COHORT ANALYSIS

Introdução

We investigated if nonsense mutation Duchenne muscular dystrophy (nmDMD) patients receiving ataluren plus standard of care (SoC) in the Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (NCT02369731) 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).

Objetivo

We investigated if nonsense mutation Duchenne muscular dystrophy (nmDMD) patients receiving ataluren plus standard of care (SoC) in the Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (NCT02369731) 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).

Método

STRIDE is an ongoing, multicenter, observational registry providing data on ataluren use in nmDMD patients in routine clinical practice. 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%.

Resultados e Conclusões

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).
These interim registry data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients.

Palavras Chave

Duchenne muscular dystrophy. Nonsense mutation. Pulmonary function.

Declaração de conflito de interesses de TODOS os autores

MT has received lecture fees from Biogen and PTC Therapeutics, and has acted as a
consultant on DMD clinical trials for BioMarin, Catabasis Pharmaceuticals, PTC
Therapeutics, ReveraGen BioPharma and Sarepta Therapeutics, and as an advisory board
member for AveXis, Biogen, Sarepta Therapeutics and PTC Therapeutics.
APC, DSM and AF report no disclosures relevant to this abstract.