PMID- 30028519
OWN - NLM
STAT- MEDLINE
DCOM- 20190523
LR  - 20210930
IS  - 1097-4598 (Electronic)
IS  - 0148-639X (Print)
IS  - 0148-639X (Linking)
VI  - 58
IP  - 5
DP  - 2018 Nov
TI  - Deflazacort versus prednisone/prednisolone for maintaining motor function and 
      delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
PG  - 639-645
LID - 10.1002/mus.26191 [doi]
AB  - 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-minute walk 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 comparison 
      will better define these differences. Muscle Nerve 58: 639-645, 2018.
CI  - (c) 2018 Wiley Periodicals, Inc.
FAU - Shieh, Perry B
AU  - Shieh PB
AD  - University of California, 300 UCLA Medical Plaza B-200, Los Angeles, Los Angeles, 
      California, USA.
FAU - Mcintosh, Joseph
AU  - Mcintosh J
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Jin, Fengbin
AU  - Jin F
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Souza, Marcio
AU  - Souza M
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Elfring, Gary
AU  - Elfring G
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Narayanan, Siva
AU  - Narayanan S
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Trifillis, Panayiota
AU  - Trifillis P
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Peltz, Stuart W
AU  - Peltz SW
AD  - PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South 
      Plainfield, New Jersey, USA.
FAU - Mcdonald, Craig M
AU  - Mcdonald CM
AD  - University of California Davis School of Medicine, Department of Physical 
      Medicine and Rehabilitation, 4860 Y Street, Suite 3850, Sacramento, California, 
      USA.
FAU - Darras, Basil T
AU  - Darras BT
AD  - Boston Children's Hospital, 300 Longwood Avenue, Boston, Massachusetts, USA.
CN  - THE ACT DMD STUDY GROUP
LA  - eng
GR  - UL1 TR000170/TR/NCATS NIH HHS/United States
PT  - Journal Article
PT  - Randomized Controlled Trial
DEP - 20180927
PL  - United States
TA  - Muscle Nerve
JT  - Muscle & nerve
JID - 7803146
RN  - 0 (Anti-Inflammatory Agents)
RN  - 0 (DMD protein, human)
RN  - 0 (Dystrophin)
RN  - 0 (Pregnenediones)
RN  - 9PHQ9Y1OLM (Prednisolone)
RN  - KR5YZ6AE4B (deflazacort)
RN  - VB0R961HZT (Prednisone)
SB  - IM
MH  - Adolescent
MH  - Age Factors
MH  - Anti-Inflammatory Agents/*therapeutic use
MH  - Child
MH  - Double-Blind Method
MH  - Dystrophin/genetics
MH  - Female
MH  - Humans
MH  - Male
MH  - Muscular Dystrophy, Duchenne/*drug therapy/genetics/*physiopathology
MH  - Prednisolone/*therapeutic use
MH  - Prednisone/*therapeutic use
MH  - Pregnenediones/*therapeutic use
MH  - Walk Test
MH  - Walking/physiology
PMC - PMC6767037
OTO - NOTNLM
OT  - deflazacort
OT  - muscular dystrophy
OT  - prednisolone
OT  - prednisone
OT  - walking
EDAT- 2018/07/22 06:00
MHDA- 2019/05/24 06:00
PMCR- 2019/09/30
CRDT- 2018/07/21 06:00
PHST- 2017/12/11 00:00 [received]
PHST- 2018/06/01 00:00 [revised]
PHST- 2018/06/03 00:00 [accepted]
PHST- 2018/07/22 06:00 [pubmed]
PHST- 2019/05/24 06:00 [medline]
PHST- 2018/07/21 06:00 [entrez]
PHST- 2019/09/30 00:00 [pmc-release]
AID - MUS26191 [pii]
AID - 10.1002/mus.26191 [doi]
PST - ppublish
SO  - Muscle Nerve. 2018 Nov;58(5):639-645. doi: 10.1002/mus.26191. Epub 2018 Sep 27.