@article{e4a1e5660b944d44910a11f324efcd64,
title = "DEFLAZACORT VERSUS PREDNISONE/PREDNISOLONE FOR MAINTAINING MOTOR FUNCTION AND DELAYING LOSS OF AMBULATION: A POST HOC ANALYSIS FROM THE ACT DMD TRIAL",
abstract = "Introduction: ACT DMD was a 48-week trial of ataluren for nonsense mutation Duchenne muscular dystrophy (nmDMD). Patients received corticosteroids for ≥6 months at entry and stable regimens throughout study. This post hoc analysis compares efficacy and safety for deflazacort and prednisone/prednisolone in the placebo arm. Methods: Patients received deflazacort (n = 53) or prednisone/prednisolone (n = 61). Endpoints included change from baseline in 6-minutewalk distance (6MWD), timed function tests, estimated age at loss of ambulation (extrapolated from 6MWD). Results: Mean changes in 6MWD were -39.0 m (deflazacort; 95%confidence limit [CL], -68.85, -9.17) and -70.6 m (prednisone/prednisolone; 95% CL, -97.16, -44.02).Mean changes in 4-stair climb were 3.79 s (deflazacort; 95% CL, 1.54, 6.03) and 6.67 s (prednisone/prednisolone; 95% CL, 4.69, 8.64). Conclusions: This analysis, limited by its post hoc nature, suggests greater preservation of 6MWD and 4-stair climb with deflazacort vs. prednisone/prednisolone. A head-to-head comparisonwill better define these differences.",
keywords = "Deflazacort, Muscular dystrophy, Prednisolone, Prednisone, Walking",
author = "{THE ACT DMD STUDY GROUP} and Shieh, {Perry B.} and Joseph McIntosh and Fengbin Jin and Marcio Souza and Gary Elfring and Siva Narayanan and Panayiota Trifillis and Peltz, {Stuart W.} and McDonald, {Craig M.} and Darras, {Basil T.} and Abdel-Hamid, {Hoda Z.} and Apkon, {Susan D.} and Barohn, {Richard J.} and Enrico Bertini and Clemens Bloetzer and Butterfield, {Russell J.} and Craig Campbell and Brigitte Chabrol and Chae, {Jong Hee} and Comi, {Giacomi Pietro} and Darras, {Basil T.} and Jahannaz Dastgir and Isabelle Desguerre and Escobar, {Raul G.} and Erika Finanger and Finkel, {Richard S.} and Flanigan, {Kevin M.} and Nathalie Goemans and Michela Guglieri and Peter Heydemann and Imelda Hughes and Iannaccone, {Susan T.} and Jones, {Kristi J.} and Anna Kaminska and Peter Karachunski and Janbernd Kirschner and Martin Kudr and Timothy Lotze and Mah, {Jean K.} and Katherine Mathews and Eugenio Mercuri and Francesco Muntoni and Yoram Nevo and Osorio, {Andr{\'e}s Nascimento} and Julie Parsons and Yann P{\'e}r{\'e}on and Araujo, {Alexandra Prufer De Queiroz Campos} and Renfroe, {J. Ben} and {De Resende}, {Maria Bernadete Dutra} and Monique Ryan",
note = "Publisher Copyright: {\textcopyright} 2018 The Authors Muscle & Nerve Published by Wiley Periodicals, Inc.",
year = "2018",
month = nov,
doi = "10.1002/mus.26191",
language = "English (US)",
volume = "58",
pages = "639--645",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "5",
}