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 A. Parsons, Susan Apkon, Basil T. Darras, Craig Campbell, Craig M. McDonald, Richard J. Barohn, Enrico Bertini, Kate Bushby, Brigitte Chabrol, Emma Ciafaloni, Jaume Columer, Giacomi Pietro Comi, Anne Connolly, Richard S. Finkel, Kevin M. FlaniganNathalie Goemans, Michela Guglieri, Susan T. Iannaccone, Kristi J. Jones, Petra Kaufmann, Janbernd Kirschner, Jean K. Mah, Katherine Mathews, Eugenio Mercuri, Francesco Muntoni, Yoram Nevo, Andrés Nascimento Osorio, Yann Péréon, Rosaline Quinlivan, J. Ben Renfroe, Barry Russman, Monique Ryan, Jacinda Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, Douglas M. Sproule, H. Lee Sweeney, Már Tulinius, Juan J. Vilchez, Giuseppe Vita, Thomas Voit, Stephanie Burns-Wechsler, Brenda Wong, Ted Abresch, Erik K. Henricson, Kim Coleman, Michelle Eagle, Julaine Florence, Ed Gappmaier, Craig McDonald, Hoda Z. Abdel-Hamid, Richard J. Barohn, Clemens Bloetzer, Kate Bushby, Russell J. Butterfield, Brigitte Chabrol, Jong Hee Chae, Giacomi Pietro Comi, Jahannaz Dastgir, Isabelle Desguerre, Raul G. Escobar, Erika Finanger, Richard S. Finkel, Nathalie Goemans, Michela Guglieri, Peter Heydemann, Imelda Hughes, Susan T. Iannaccone, Kristi J. Jones, Anna Kaminska, Peter Karachunski, Janbernd Kirschner, Martin Kudr, Timothy Lotze, Jean K. Mah, Katherine Mathews, Francesco Muntoni, Yoram Nevo, Andrés Nascimento Osorio, Yann Péréon, Alexandra Prufer De Queiroz Campos Araujo, J. Ben Renfroe, Maria Bernadete Dutra De Resende, Monique Ryan, Jacinda Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, H. Lee Sweeney, Gihan Tennekoon, Haluk Topaloglu, Ricardo Erazo Torricelli, Már Tulinius, Giuseppe Vita, Thomas Voit, Brenda Wong, Lindsay N. Alfano, Michelle Eagle, Meredith K. James, Linda Lowes, Anna Mayhew, Elena S. Mazzone, Leslie Nelson, Kristy J. Rose

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1337-1347
Number of pages11
JournalJournal of Comparative Effectiveness Research
Volume10
Issue number18
DOIs
StatePublished - Dec 1 2021

Keywords

  • Duchenne muscular dystrophy
  • deflazacort
  • meta-analysis
  • nonsense mutation Duchenne muscular dystrophy
  • prednisolone
  • prednisone

ASJC Scopus subject areas

  • Health Policy

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