Evidence map›Paper›PMID 22402864›Full record

Trial reportNeurology2012

Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial.

D M Escolar, A Zimmerman, T Bertorini, P R Clemens, A M Connolly, L Mesa, K Gorni, A Kornberg, H Kolski, N Kuntz and 12 more

Open access · bronzeAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Neurology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 2 pooled it
1.1field-weighted citation impact, top 25% 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

16 citing papers in PubMed, 2 syntheses or guidelines pooled it, 25 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Article
  5. Current and Emerging Therapies for Duchenne Muscular Dystrophy.Current treatment options in neurology · 2018
    Review
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Review
  12. Review
  13. Article
  14. Immune-mediated pathology in Duchenne muscular dystrophy.Science translational medicine · 2015
    Review
  15. Article
  16. 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

22 authors at 11 institutions in 4 countries.

D M EscolarChildren’s National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
A Zimmerman
T Bertorini
P R Clemens
A M Connolly
L Mesa
K Gorni
A Kornberg
H Kolski
N Kuntz
Y Nevo
C Tesi-Rocha
K Nagaraju
S Rayavarapu
L P Hache
J E Mayhew
J Florence
F Hu
A Arrieta
E Henricson
R T Leshner
J K Mah
Children's Hospital of Pittsburgh · USRoyal Children's Hospital · AUWashington University in St. Louis · USChildren's National · USFavaloro University · ARMayo Clinic · USMayo Clinic in Arizona · USUniversity of Alberta · CAUniversity of Calgary · CAUniversity of Tennessee at Knoxville · USUniversity of Tennessee Health Science Center · US

Funding

UNIVERSITY OF PITTSBURGH CLINICAL AND TRANSLATIONAL SCIENCE INSTITUTE: BPCAUL1RR024153 · NCRR · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2006 to 2011
$73.9M
University of Pittsburgh Clinical and Translational Science InstituteUL1TR000005 · NCATS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2012 to 2015
$50.8M
Washington University Institute of Clinical and Translational Sciences (UL1)UL1RR024992 · NCRR · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2007 to 2011
$45.4M
ZAP-70 EXPRESSION, CD38 EXPRESSION AND PROGNOSIS IN CHRONIC LYMPHOCYTIC LEUKEMIAM01RR020359 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI MIRZA, NAZRAT · 2005 to 2010
$13.8M
WATER PRECAUTIONS AND TYMPANOSTOMY TUBESM01RR000084 · NCRR · CHILDREN'S HOSP PITTSBURGH/UPMC HLTH SYS · PI PERLMUTTER, DAVID H · 1985 to 2006
$5.6M
Mentored Patient Oriented Research Career DevelopmentK23RR016281 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI ESCOLAR, DIANA M · 2001 to 2005
$622k
NCATS NIH HHS UL1 TR000005NCRR NIH HHS 5M01 RR020359NCRR NIH HHS K23 RR16281-01NCRR NIH HHS M01 RR00084NCRR NIH HHS UL1 RR024153NCRR NIH HHS UL1 RR024992
6 · The paper itself

Abstract

objectiveTo determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD).

methodsThis was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (~20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test.

resultsA total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p = 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs.

conclusionThe addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.

Indexed as

Adrenal Cortex HormonesChildDelayed-Action PreparationsDisease ProgressionDouble-Blind MethodDrug Therapy, CombinationHumansMaleMuscle StrengthMuscular Dystrophy, DuchenneNeurologic ExaminationPentoxifyllinePhosphodiesterase InhibitorsQuality of LifeRespiratory Function TestsSample SizeAdrenal Cortex HormonesDelayed-Action PreparationsPentoxifyllinePhosphodiesterase Inhibitors

Identifiers

PMID22402864
PMCPMC3306159
OpenAlexW1990707185

What OpenQuestion holds

Textmetadata
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.