Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
Por:
Mercuri E, Muntoni F, Nascimento-Osorio A, Tulinius M, Buccella F, Morgenroth LP, Gordish-Dressman H, Jiang J, Trifillis P, Zhu J, Kristensen A, Santos CL, Henricson EK, McDonald CM, Desguerre I, STRIDE and CINRG Duchenne Natural History Investigators
Publicada:
1 abr 2020
Ahead of Print:
30 ene 2020
Categoría:
Health policy
Resumen:
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.
Filiaciones:
Mercuri E:
Department of Pediatric Neurology, Catholic University, Rome, Italy
Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy
Muntoni F:
Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK
NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK
Nascimento-Osorio A:
Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain
Tulinius M:
Department of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden
Buccella F:
Parent Project Italy APS, Rome, Italy
Morgenroth LP:
Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA
Gordish-Dressman H:
Center for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA
Jiang J:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Trifillis P:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Zhu J:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Kristensen A:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Santos CL:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Henricson EK:
University of California Davis School of Medicine, Davis, CA, USA
McDonald CM:
University of California Davis School of Medicine, Davis, CA, USA
Desguerre I:
APHP Necker - Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France
hybrid, Green Published, Green Submitted
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