Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Eugenio Mercuri, Francesco Muntoni, Andrés Nascimento Osorio, Már Tulinius, Filippo Buccella, Lauren P. Morgenroth, Heather Gordish-Dressman, Joel Jiang, Panayiota Trifillis, Jin Zhu, Allan Kristensen, Claudio L. Santos, Erik K. Henricson, Craig M. McDonald, Isabelle Desguerre

Research output: Contribution to journalArticle

2 Scopus citations

Abstract

Aim: 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.

Original languageEnglish (US)
Pages (from-to)341-360
Number of pages20
JournalJournal of Comparative Effectiveness Research
Volume9
Issue number5
DOIs
StatePublished - Apr 2020

Keywords

  • ataluren
  • dystrophin
  • effectiveness
  • nonsense mutation Duchenne muscular dystrophy
  • safety
  • STRIDE Registry

ASJC Scopus subject areas

  • Health Policy

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    Mercuri, E., Muntoni, F., Osorio, A. N., Tulinius, M., Buccella, F., Morgenroth, L. P., Gordish-Dressman, H., Jiang, J., Trifillis, P., Zhu, J., Kristensen, A., Santos, C. L., Henricson, E. K., McDonald, C. M., & Desguerre, I. (2020). Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. Journal of Comparative Effectiveness Research, 9(5), 341-360. https://doi.org/10.2217/cer-2019-0171