Evidence map›Paper›PMID 30028519›Full record

Trial reportMuscle & nerve2018

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.

Perry B Shieh, Joseph Mcintosh, Fengbin Jin, Marcio Souza, Gary Elfring, Siva Narayanan, Panayiota Trifillis, Stuart W Peltz, Craig M Mcdonald, Basil T Darras and 1 more

Open access · hybridAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Muscle & nerve, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed, 3 pooled it
3.0field-weighted citation impact, top 8% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

32 citing papers in PubMed, 3 syntheses or guidelines pooled it, 59 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Trial
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Article
  11. Article
  12. Review
  13. Current Strategies of Muscular Dystrophy Therapeutics: An Overview.Methods in molecular biology (Clifton, N.J.) · 2023
    Review
  14. Article
  15. Review
  16. Article
  17. Review
  18. Review
  19. Theragnosis for Duchenne Muscular Dystrophy.Frontiers in pharmacology · 2021
    Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 4 institutions in 1 country.

Perry B ShiehUniversity of California, 300 UCLA Medical Plaza B-200, Los Angeles, Los Angeles, California, USA.
Joseph McintoshPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Fengbin JinPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Marcio SouzaPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Gary ElfringPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Siva NarayananPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Panayiota TrifillisPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Stuart W PeltzPTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, South Plainfield, New Jersey, USA.
Craig M McdonaldUniversity of California Davis School of Medicine, Department of Physical Medicine and Rehabilitation, 4860 Y Street, Suite 3850, Sacramento, California, USA.
Basil T DarrasBoston Children's Hospital, 300 Longwood Avenue, Boston, Massachusetts, USA.
THE ACT DMD STUDY GROUP
PTC Therapeutics (United States) · USBoston Children's Hospital · USUniversity of California, Davis · USUniversity of California, Los Angeles · US

Funding

Harvard Clinical and Translational Science Center (UL1)UL1TR000170 · NCATS · HARVARD MEDICAL SCHOOL · PI NADLER, LEE MARSHALL · 2012 to 2013
$31.4M
NCATS NIH HHS UL1 TR000170
6 · The paper itself

Abstract

introductionACT 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.

methodsPatients 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).

resultsMean 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).

conclusionsThis 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.

Indexed as

AdolescentAge FactorsAnti-Inflammatory AgentsChildDouble-Blind MethodDystrophinFemaleHumansMaleMuscular Dystrophy, DuchennePrednisolonePrednisonePregnenedionesWalkingWalk TestAnti-Inflammatory AgentsdeflazacortDMD protein, humanDystrophinPrednisolonePrednisonePregnenedionesdeflazacortmuscular dystrophyprednisoloneprednisonewalking

Identifiers

PMID30028519
PMCPMC6767037
OpenAlexW2882978393

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.