title: Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial.
abstract: 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.
  
  
  CONCLUSIONS
  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.
  
  
  METHODS
  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.affiliation: Children’s National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
authors.forename: D M
authors.initials: DM
authors.lastname: Escolar
authors.forename: A
authors.initials: A
authors.lastname: Zimmerman
authors.forename: T
authors.initials: T
authors.lastname: Bertorini
authors.forename: P R
authors.initials: PR
authors.lastname: Clemens
authors.forename: A M
authors.initials: AM
authors.lastname: Connolly
authors.forename: L
authors.initials: L
authors.lastname: Mesa
authors.forename: K
authors.initials: K
authors.lastname: Gorni
authors.forename: A
authors.initials: A
authors.lastname: Kornberg
authors.forename: H
authors.initials: H
authors.lastname: Kolski
authors.forename: N
authors.initials: N
authors.lastname: Kuntz
authors.forename: Y
authors.initials: Y
authors.lastname: Nevo
authors.forename: C
authors.initials: C
authors.lastname: Tesi-Rocha
authors.forename: K
authors.initials: K
authors.lastname: Nagaraju
authors.forename: S
authors.initials: S
authors.lastname: Rayavarapu
authors.forename: L P
authors.initials: LP
authors.lastname: Hache
authors.forename: J E
authors.initials: JE
authors.lastname: Mayhew
authors.forename: J
authors.initials: J
authors.lastname: Florence
authors.forename: F
authors.initials: F
authors.lastname: Hu
authors.forename: A
authors.initials: A
authors.lastname: Arrieta
authors.forename: E
authors.initials: E
authors.lastname: Henricson
authors.forename: R T
authors.initials: RT
authors.lastname: Leshner
authors.forename: J K
authors.initials: JK
authors.lastname: Mah
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D002648:Child; D003692:Delayed-Action Preparations; D018450:Disease Progression; D004311:Double-Blind Method; D004359:Drug Therapy, Combination; D006801:Humans; D008297:Male; D053580:Muscle Strength / Q000502:physiology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D009460:Neurologic Examination; D010431:Pentoxifylline / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D010726:Phosphodiesterase Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D011788:Quality of Life; D012129:Respiratory Function Tests; D018401:Sample Size; D016896:Treatment Outcome
chemical_list: D000305:Adrenal Cortex Hormones; D003692:Delayed-Action Preparations; D010726:Phosphodiesterase Inhibitors; D010431:Pentoxifylline