Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
2020; Future Medicine; Volume: 9; Issue: 5 Linguagem: Inglês
10.2217/cer-2019-0171
ISSN2042-6313
AutoresEugenio Mercuri, Francesco Muntoni, A. Nascimento Osorio, M. Tulinius, Filippo Buccella, Lauren P. Morgenroth, Heather Gordish‐Dressman, Joel Jiang, Panayiota Trifillis, Jin Zhu, Allan Kristensen, Claudio Santos, Erik Henricson, Craig M. McDonald, Isabelle Desguerre,
Tópico(s)Genetic Neurodegenerative Diseases
ResumoAim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
Referência(s)