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.

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study / E. Mercuri, F. Muntoni, A. Osorio, M. Tulinius, F. Buccella, L. Morgenroth, H. Gordish-Dressman, J. Jiang, P. Trifillis, L. Zhu, A. Kristensen, C. Santos, E. Henricson, C. McDonald, I. Desguerre, G. Bernert, M. Gosk-Tomek, A. Ille, A. Kellersmann, S. Weiss, V. Pilshofer, Z. Balintova, P. Danhofer, P. Fabulova, L. Jurikova, P. Fuchsova, J. Haberlova, F. Laffargue, C. Sarret, B. Pontier, R. Bellance, E. Sarrazin, P. Sabouraud, A. Magot, S. Mercier, Y. Pereon, J. Cuisset, S. Coopman-Degryse, E. Enaud, M. Jacquemont, A. Perville, M. Renouil, V. Trommsdorff, D. Verheulpen, S. Fontaine-Carbonnel, C. Vuillerot, S. Peudenier, J. Ropars, F. Audic, B. Chabrol, S. Chabrier, G. Gousse, E. Lagrue, K. Aragon, C. Barnerias, L. Brande, S. De Lucia, I. Desguerre, T. Gidaro, A. Seferian, L. Servais, V. Laugel, C. Espil-Taris, H. Mecili, E. Raffo, S. Ragot-Mandry, S. Borrell, J. Kirschner, A. Gangfuss, M. Henrich, H. Kolbel, U. Schara, N. Sponemann, E. Temme, J. Seeger, A. Hirsch, J. Denecke, J. Johannsen, A. Neu, D. Osinski, S. Rugner, S. Schussler, R. Trollmann, A. Kaindl, J. Schneider, C. Stoltenburg, C. Weiss, G. Schreiber, A. Hahn, M. Grzybowski, E. Pavlidou, E. Pavlou, S. Dobner, Z. Liptai, T. Dor, C. Brogna, M. Catteruccia, A. D'Amico, E. Mercuri, M. Pane, L. Bello, E. Pegoraro, C. Semplicini, E. Albamonte, G. Baranello, G. Comi, A. Govoni, A. Lerario, F. Magri, R. Masson, E. Mauri, V. Sansone, C. Brusa, T. Mongini, F. Ricci, M. Vacchetti, C. Bruno, C. Paniucci, M. Pedemonte, M. Giannotta, A. Pini, S. Messina, M. Sframeli, G. Vita, L. Ruggiero, L. Santoro, D. Craiu, C. Motoescu, C. Sandu, R. Teleanu, D. Vasile, M. Tulinius, I. Hughes, A. Childs, Z. Alhaswani, H. Roper, D. Parasuraman, C. DeGoede, V. Gowda, A. Manzur, P. Munot, A. Sarkokzy, C. Charlesworth, J. Lemon, L. Turner, S. Spinty, A. Dubrovsky, A. Kornberg, M. Ryan, R. Webster, W. Biggar, L. McAdam, J. Mah, H. Kolski, V. Vishwanathan, S. Chidambaranathan, Y. Nevo, K. Gorni, J. Carlo, C. McDonald, E. Henricson, R. Abresch, N. Joyce, A. Cnaan, L. Morgenroth, R. Leshner, C. Tesi-Rocha, M. Thangarajh, T. Duong, P. Clemens, H. Abdel-Hamid, A. Connolly, A. Pestronk, J. Teasley, A. Harper, T. Bertorini, N. Kuntz, S. Driscoll, J. Day, P. Karachunski, T. Lotze. - In: JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH. - ISSN 2042-6305. - 9:5(2020), pp. 341-360. [10.2217/cer-2019-0171]

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

S. De Lucia;V. Sansone;
2020

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.
ataluren; dystrophin; effectiveness; nonsense mutation Duchenne muscular dystrophy; safety; STRIDE Registry
Settore MED/26 - Neurologia
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2434/853343
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