Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
Perry B. Shieh, Gary Elfring, Panayiota Trifillis, Claudio Santos, Stuart W. Peltz, Julie Parsons, Susan Apkon, Basil T. Darras, Craig Campbell, Craig M. McDonald, Richard J. Barohn, Enrico Bertini, Kate Bushby, B. Chabrol, Emma Ciafaloni, Jaume Columer, Giacomi Pietro Comi, Anne M. Connolly, Richard S. Finkel, Kevin M. Flanigan, Nathalie Goemans, Michela Guglieri, Susan T. Iannaccone, Kristi Jones, Petra Kaufmann, Janbernd Kirschner, Jean K. Mah, Katherine D. Mathews, Eugenio Mercuri, Francesco Muntoni, Yoram Nevo, A. Nascimento Osorio, Yann Péréon, Rosaline C. M. Quinlivan, J. Ben Renfroe, Barry S. Russman, Monique M. Ryan, Jacinda B. Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, Douglas M. Sproule, H. Lee Sweeney, M. Tulinius, Juan J. Vílchez, Giuseppe Vita, Thomas Voït, Stephanie Burns Wechsler, Brenda Wong, Ted Abresch, Erik Henricson, Kim Coleman, Michelle Eagle, Julaine Florence, Ed Gappmaier, Craig M. McDonald, Hoda Abdel‐Hamid, Richard J. Barohn, Enrico Bertini, Clemens Bloetzer, Kate Bushby, Russell J. Butterfield, B. Chabrol, Jong‐Hee Chae, Giacomi Pietro Comi, Jahannaz Dastgir, Isabelle Desguerre, Raúl G. Escobar, Erika Finanger, Richard S. Finkel, Kevin M. Flanigan, Nathalie Goemans, Michela Guglieri, Peter Heydemann, Imelda Hughes, Susan T. Iannaccone, Kristi Jones, Anna Kamińska, Peter Karachunski, Janbernd Kirschner, Martin Kudr, Timothy Lotze, Jean K. Mah, Katherine D. Mathews, Eugenio Mercuri, Francesco Muntoni, Yoram Nevo, A. Nascimento Osorio, Yann Péréon, Alexandra Prufer de Queiroz Campos Araújo, J. Ben Renfroe, Maria Bernadete Dutra de Resende, Monique M. Ryan, Jacinda B. Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, H. Lee Sweeney, Gihan Tennekoon, Haluk Topaloğlu
Abstract
Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.