Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
Mercuri E, Muntoni F, Osorio AN, Tulinius M, Buccella F, Morgenroth LP, Gordish-Dressman H, Jiang J, Trifillis P, Zhu L, Kristensen A, Santos CL, Henricson EK, Mcdonald CM, Desguerre I, Bernert G, Gosk-Tomek M, Ille A, Kellersmann A, Weiss S, Pilshofer , Balintova Z, Danhofer P, Fabulova P, Jurikova L, Fuchsova P, Haberlova J, Laffargue F, Sarret C, Pontier B, Bellance R, Sarrazin E, Sabouraud P, Magot A, Mercier S, Pereon Y, Cuisset JM, Coopman-Degryse S, Enaud E, Jacquemont ML, Perville A, Renouil M, Trommsdorff , Verheulpen D, Fontaine-Carbonnel S, Vuillerot C, Peudenier S, Ropars J, Audic F, Chabrol B, Chabrier S, Gousse G, Lagrue E, Aragon K, Barnerias C, Brande L, De Lucia S, Desguerre , Gidaro T, Seferian A, Servais L, Laugel , Espil-Taris C, Mecili H, Raffo E, Ragot-Mandry S, Borrell S, Kirschner J, Gangfuss A, Henrich M, Koelbel H, Schara U, Spoenemann N, Temme E, Seeger J, Hirsch A, Denecke J, Johannsen J, Neu A, Osinski D, Ruegner S, Schuessler S, Trollmann R, Kaindl A, Schneider JB, Stoltenburg C, Weiss C, Schreiber G, Hahn A, Grzybowski (Giessen) M, Pavlidou E, Pavlou E, Dobner S, Liptai Z, Dor T, Brogna C, Catteruccia M, D'Amico A, Mercuri E, Pane M, Bello L, Pegoraro E, Semplicini C, Albamonte E, Baranello G, Comi G, Govoni A, Lerario A, Magri F, Masson R, Mauri E, Sansone , Brusa C, Mongini T, Ricci F, Vacchetti M, Bruno C, Paniucci C, Pedemonte M, Giannotta M, Pini A, Messina S, Sframeli M, Vita G, Ruggiero L, Santoro L, Craiu D, Motoescu C, Sandu C, Teleanu RI, Vasile D, Tulinius M, Hughes , Childs AM, Alhaswani Z, Roper H, Parasuraman D, Degoede C, Gowda , Manzur A, Munot P, Sarkokzy A, Charlesworth C, Lemon J, Turner L, Spinty S, Dubrovsky A, Kornberg A, Ryan M, Webster R, Biggar WD, Mcadam LC, Mah JK, Kolski H, Vishwanathan V, Chidambaranathan S, Nevo Y, Gorni K, Carlo J, Mcdonald CM, Henricson EK, Abresch RT, Joyce NC, Cnaan A, Morgenroth LP, Leshner R, Tesi-Rocha C, Thangarajh M, Duong T, Clemens PR, Abdel-Hamid H, Connolly AM, Pestronk A, Teasley J, Harper A, Bertorini TE, Kuntz N, Driscoll S, Day JW, Karachunski P, Lotze T (2020)
Publication Type: Journal article
Publication year: 2020
Journal
Book Volume: 9
Pages Range: 341-360
Journal Issue: 5
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.
Involved external institutions
University of California Davis (UCDAVIS)
United States (USA) (US)
Catholic University of the Sacred Heart / Università Cattolica del Sacro Cuore
Italy (IT)
University College London (UCL)
United Kingdom (GB)
Universitat de Barcelona (UB) / University of Barcelona
Spain (ES)
University of Gothenburg / Göteborgs universitet
Sweden (SE)
Parent Project Italy APS
Italy (IT)
Children’s National Health System
United States (USA) (US)
PTC Therapeutics Inc.
United States (USA) (US)
University of Paris 5 - René Descartes / Université Paris V René Descartes
France (FR)
United States (USA) (US)
Catholic University of the Sacred Heart / Università Cattolica del Sacro Cuore
Italy (IT)
University College London (UCL)
United Kingdom (GB)
Universitat de Barcelona (UB) / University of Barcelona
Spain (ES)
University of Gothenburg / Göteborgs universitet
Sweden (SE)
Parent Project Italy APS
Italy (IT)
Children’s National Health System
United States (USA) (US)
PTC Therapeutics Inc.
United States (USA) (US)
University of Paris 5 - René Descartes / Université Paris V René Descartes
France (FR)
How to cite
APA:
Mercuri, E., Muntoni, F., Osorio, A.N., Tulinius, M., Buccella, F., Morgenroth, L.P.,... Lotze, T. (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://dx.doi.org/10.2217/cer-2019-0171
MLA:
Mercuri, Eugenio, et al. "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 (2020): 341-360.
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