Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
Authors: Shieh Perry B , Elfring Gary , Trifillis Panayiota , Santos Claudio , Peltz Stuart W , Parsons Julie A , Apkon Susan , Darras Basil T , Campbell Craig , McDonald Craig M , Barohn Richard J , Bertini Enrico , Bushby Kate , Chabrol Brigitte , Ciafaloni Emma , Columer Jaume , Comi Giacomi Pietro , Connolly Anne , Finkel Richard S , Flanigan Kevin M , Goemans Nathalie , Guglieri Michela , Iannaccone Susan T , Jones Kristi J , Kaufmann Petra , Kirschner Janbernd , Mah Jean K , Mathews Katherine , Mercuri Eugenio , Muntoni Francesco , Nevo Yoram , Osorio Andrés Nascimento , Péréon Yann , Quinlivan Rosaline , Renfroe J. Ben , Russman Barry , Ryan Monique , Sampson Jacinda , Schara Ulrike , Selby Kathryn , Sejersen Thomas , Sproule Douglas M , Sweeney H. Lee , Tulinius Már , Vilchez Juan J , Vita Giuseppe , Voit Thomas , Burns-Wechsler Stephanie , Wong Brenda , Abresch Ted , Henricson Erik K , Coleman Kim , Eagle Michelle , Florence Julaine , Gappmaier Ed , McDonald Craig , Abdel-Hamid Hoda Z , Barohn Richard J , Bertini Enrico , Bloetzer Clemens , Bushby Kate , Butterfield Russell J , Chabrol Brigitte , Chae Jong-Hee , Comi Giacomi Pietro , Dastgir Jahannaz , Desguerre Isabelle , Escobar Raul G , Finanger Erika , Finkel Richard S , Flanigan Kevin M , Goemans Nathalie , Guglieri Michela , Heydemann Peter , Hughes Imelda , Iannaccone Susan T , Jones Kristi J , Kaminska Anna , Karachunski Peter , Kirschner Janbernd , Kudr Martin , Lotze Timothy , Mah Jean K , Mathews Katherine , Mercuri Eugenio , Muntoni Francesco , Nevo Yoram , Osorio Andrés Nascimento , Péréon Yann , de Queiroz Campos Araujo Alexandra Prufer , Renfroe J. Ben , Dutra de Resende Maria Bernadete , Ryan Monique , Sampson Jacinda , Schara Ulrike , Selby Kathryn , Sejersen Thomas , Sweeney H. Lee , Tennekoon Gihan , Topaloglu Haluk , Torricelli Ricardo Erazo , Tulinius Már , Vilchez Juan J , Vita Giuseppe , Voit Thomas , Wong Brenda , Alfano Lindsay N , Eagle Michelle , James Meredith K , Lowes Linda , Mayhew Anna , Mazzone Elena S , Nelson Leslie , Rose Kristy J 
Published: October 2021

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.