Evidence map›Paper›PMID 36335191›Full record

Trial reportScientific reports2022

Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy.

Sarah P Sherlock, Jeffrey Palmer, Kathryn R Wagner, Hoda Z Abdel-Hamid, Cuixia Tian, Jean K Mah, Francesco Muntoni, Michela Guglieri, Russell J Butterfield, Lawrence Charnas and 1 more

Registry-linked trialOpen access · goldAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Scientific reports, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02310763 (A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY), which is not on this map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.2field-weighted citation impact, top 21% 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.

NCT02310763 phase2terminatednot on this map

A phase 2 randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of pf-06252616 in ambulatory boys with duchenne muscular dystrophy

TypeinterventionalSponsorPfizerRan2014 to 2018Enrolled121ConditionsDuchenne Muscular DystrophyArmsPF-06252616, Placebo
3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 10 citations in OpenAlex.

  1. Article
  2. Determination of body composition by dual x-ray absorptiometry in persons with haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2024
    Article
  3. Article
  4. Observational
  5. Article
  6. Review
  7. Article
  8. Review
  9. Review
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 8 institutions in 3 countries.

Sarah P SherlockPfizer Inc, Cambridge, MA, USA. Sarah.Sherlock@pfizer.com.
Jeffrey PalmerPfizer Inc, Cambridge, MA, USA.
Kathryn R WagnerKennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Hoda Z Abdel-HamidDivision of Child Neurology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh, PA, USA.
Cuixia TianCincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Jean K MahAlberta Children's Hospital, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Francesco MuntoniDubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, London, UK.
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle, UK.
Russell J ButterfieldUniversity of Utah School of Medicine, Salt Lake City, UT, USA.
Lawrence CharnasPfizer Inc, Cambridge, MA, USA.
Shannon MarraffinoPfizer Inc, Cambridge, MA, USA.
Pfizer (United States) · USAlberta Children's Hospital · CAKennedy Krieger Institute · USNewcastle University · GBUniversity College London · GBUniversity of Cincinnati · USUniversity of Pittsburgh · USUniversity of Utah · US

Funding

Utah Center for Clinical and Translational ScienceUL1TR002538 · NCATS · UNIVERSITY OF UTAH · PI HESS, RACHEL, MAJERSIK, JENNIFER JUHL · 2018 to 2022
$26.0M
NCATS NIH HHS UL1 TR002538
6 · The paper itself

Abstract

We evaluated whether whole-body dual-energy X-ray absorptiometry (DXA) measures of lean body mass can be used as biomarkers for disease progression and treatment effects in patients with Duchenne muscular dystrophy. This post hoc analysis utilized data from a randomized, 2-period study of domagrozumab versus placebo in 120 ambulatory boys with DMD. DXA measures of lean body mass were obtained from the whole body (excluding head), arms, legs and appendicular skeleton at baseline and every 16 weeks. Treatment effects on DXA measures for domagrozumab versus placebo were assessed at Week 49. At Week 49, domagrozumab statistically significantly increased lean body mass versus placebo in the appendicular skeleton (p = 0.050) and arms (p < 0.001). The relationship between lean body mass at Week 49 and functional endpoints at Week 97 was evaluated. Changes in lean body mass at Week 49 in all regions except arms were significantly correlated with percent change from baseline in 4-stair climb (4SC) at Week 97. DXA-derived percent lean mass at Week 49 also correlated with 4SC and North Star Ambulatory Assessment at Week 97. These data indicate that whole-body DXA measures can be used as biomarkers for treatment effects and disease progression in patients with DMD, and warrant further investigation.Trial registration: ClinicalTrials.gov, NCT02310763; registered 8 December 2014.

Indexed as

Muscular Dystrophy, DuchenneAbsorptiometry, PhotonBiomarkersBody CompositionDisease ProgressionHumansMaleBiomarkers

Identifiers

PMID36335191
PMCPMC9637094
OpenAlexW4308496880

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

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.