Document Detail


Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial.
MedLine Citation:
PMID:  22402864     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
OBJECTIVE: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD).
METHODS: This 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.
RESULTS: A 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.
CONCLUSION: The 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.
Authors:
D M Escolar; 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
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Publication Detail:
Type:  Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, Non-P.H.S.     Date:  2012-03-07
Journal Detail:
Title:  Neurology     Volume:  78     ISSN:  1526-632X     ISO Abbreviation:  Neurology     Publication Date:  2012 Mar 
Date Detail:
Created Date:  2012-03-20     Completed Date:  2012-05-01     Revised Date:  2013-06-26    
Medline Journal Info:
Nlm Unique ID:  0401060     Medline TA:  Neurology     Country:  United States    
Other Details:
Languages:  eng     Pagination:  904-13     Citation Subset:  AIM; IM    
Affiliation:
Children’s National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
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MeSH Terms
Descriptor/Qualifier:
Adrenal Cortex Hormones / therapeutic use
Child
Delayed-Action Preparations
Disease Progression
Double-Blind Method
Drug Therapy, Combination
Humans
Male
Muscle Strength / physiology
Muscular Dystrophy, Duchenne / drug therapy*,  physiopathology,  psychology
Neurologic Examination
Pentoxifylline / administration & dosage,  adverse effects,  therapeutic use*
Phosphodiesterase Inhibitors / administration & dosage,  adverse effects,  therapeutic use*
Quality of Life
Respiratory Function Tests
Sample Size
Treatment Outcome
Grant Support
ID/Acronym/Agency:
5M01 RR020359/RR/NCRR NIH HHS; K23 RR16281-01/RR/NCRR NIH HHS; M01 RR00084/RR/NCRR NIH HHS; UL1 RR024153/RR/NCRR NIH HHS; UL1 RR024992/RR/NCRR NIH HHS; UL1 RR024992/RR/NCRR NIH HHS; UL1 TR000005/TR/NCATS NIH HHS
Chemical
Reg. No./Substance:
0/Adrenal Cortex Hormones; 0/Delayed-Action Preparations; 0/Phosphodiesterase Inhibitors; 6493-05-6/Pentoxifylline
Comments/Corrections

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