@article{1834ed73d7d94ab89f1d4803e1b4f8ae,
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 = "Funding Information: We thank the patients and their families for their participation in this study; individuals who were instrumental in the conduct of this study and the collection of data, particularly principal investigators, supporting investigators, clinical coordinators, clinical evaluators, and study coordinators. We thank Robert Weiss (University of Utah, UT, USA) for dystrophin gene sequencing; the patient advocacy organizations (including Valerie Cwik and the Muscular Dystrophy Association, and Patricia Furlong and the Parent Project Muscular Dystrophy) for the collaboration and support that made this trial possible. Medical writing was provided by Annette Skorupa, PhD of EnlightenMed LLC and was funded by PTC Therapeutics, Inc. Funding Information: We thank the patients and their families for their participation in this study; individuals who were instrumental in the conduct of this study and the collection of data, particularly principal investigators, supporting investigators, clinical coordinators, clinical evaluators, and study coordinators. We thank Robert Weiss (University of Utah, UT, USA) for dystrophin gene sequencing; the patient advocacy organizations (including Valerie Cwik and the Muscular Dystrophy Association, and Patricia Furlong and the Parent Project Muscular Dystrophy) for the collaboration and support that made this trial possible. Medical writing was provided by Annette Skorupa, PhD of EnlightenMed LLC and was funded by PTC Therapeutics, Inc. The ACT DMD Study Group: Hoda Z Abdel-Hamid (Children{\textquoteright}s Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA); Susan D Apkon (Seattle Children{\textquoteright}s Hospital, Seattle, WA, USA); Richard J Barohn (University 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",
}