title: Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.
abstract: BACKGROUND
  In this study we performed an open-label, pilot study of an orally administered liquid formulation of immediate-release pentoxifylline (PTX) on patients with Duchenne muscular dystrophy (DMD). Treatment efficacy, safety, and tolerability were assessed.
  
  
  METHODS
  The tolerability and safety of PTX and measures of muscle strength and function were evaluated during 12 months of treatment.
  
  
  RESULTS
  Seventeen boys with DMD, between 4 and 8 years of age, were enrolled at one of five Cooperative International Neuromuscular Research Group (CINRG) centers. Only 9 were able to complete the 12-month PTX treatment phase; the primary reason for discontinuation was adverse events. Intolerable gastrointestinal side effects were experienced by 65% of participants. Two participants had severe leukopenia that resolved with medication withdrawal.
  
  
  CONCLUSIONS
  Open-label treatment with a liquid formulation of immediate-release PTX resulted in a high incidence of adverse events in boys with DMD. Poor tolerability of this PTX formulation precluded adequate assessment of efficacy.
authors.affiliation: Children's National Medical Center, Washington, DC, USA.
authors.forename: Angela
authors.initials: A
authors.lastname: Zimmerman
authors.forename: Paula R
authors.initials: PR
authors.lastname: Clemens
authors.forename: Carolina
authors.initials: C
authors.lastname: Tesi-Rocha
authors.forename: Anne
authors.initials: A
authors.lastname: Connolly
authors.forename: Susan T
authors.initials: ST
authors.lastname: Iannaccone
authors.forename: Nancy
authors.initials: N
authors.lastname: Kuntz
authors.forename: Adrienne
authors.initials: A
authors.lastname: Arrieta
authors.forename: Lauren
authors.initials: L
authors.lastname: Hache
authors.forename: Erik
authors.initials: E
authors.lastname: Henricson
authors.forename: Fengming
authors.initials: F
authors.lastname: Hu
authors.forename: Jill
authors.initials: J
authors.lastname: Mayhew
authors.forename: Diana M
authors.initials: DM
authors.lastname: Escolar
mesh_terms: D000284:Administration, Oral; D002648:Child; D002675:Child, Preschool; D005767:Gastrointestinal Diseases / Q000139:chemically induced; D006801:Humans; D007970:Leukopenia / Q000139:chemically induced; D008297:Male; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D010431:Pentoxifylline / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D010865:Pilot Projects; D016896:Treatment Outcome
chemical_list: D010431:Pentoxifylline