Supplementary tables: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

dataset

posted on 2024-01-08, 18:01 authored by Craig M McDonald, Francesco MuntoniFrancesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin, Heather Gordish-Dressman, Lauren Morgenroth, Christian Werner, James Li, Richard Able, Panayiota Trifillis, Már Tulinius, On behalf of the Study 019 & CINRG Duchenne Natural History Investigators

These are peer-reviewed supplementary tables for the article 'Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients' published in the Journal of Comparative Effectiveness Research.

Supplementary Table 1. Baseline demographics and disease characteristics for Study 019 patients with nmDMD who received ataluren (40 mg/kg/day) plus SoC in at least Study 019 (N = 59) and for propensity-score matched patients with DMD receiving SoC alone in the CINRG DNHS (N = 59), using age at diagnosis for both datasets as the fourth covariate for matching, for the sensitivity analysis of age at LoA.
Supplementary Table 2. Baseline demographics and disease characteristics for non-ambulatory Study 019 patients with nmDMD who received ataluren 40 mg/kg/day plus SoC in at least this study (N = 45) and propensity-score matched patients with DMD who received SoC alone in the CINRG DNHS (N = 45), using age at diagnosis for both datasets as the fourth covariate for matching, for the sensitivity analysis of age at decline in respiratory function.
Supplementary Table 3. Treatment-emergent adverse events† experienced by patients in the as-treated population of Study 019 (N = 94).
Supplementary Table 4. Lipid profile of patients in the as-treated population of Study 019 (N = 94) at baseline and Week 240.
Supplementary Table 5. Hypertensive status of patients in the as-treated population of Study 019 (N = 94) from baseline to Week 240.
Supplementary Table 6. ECG results of patients of patients in the as-treated population of Study 019 (N = 94) from screening to post-treatment.
Supplementary Table 7. Hepatic and renal abnormalities in patients in the as-treated population of Study 019 (N = 94) from screening to post-treatment.

Summary: Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. ClinicalTrials.gov registration: NCT01557400.