Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy

Angela Zimmerman, Paula R. Clemens, Carolina Tesi-Rocha, Anne Connolly, Susan T. Iannaccone, Nancy Kuntz, Adrienne Arrieta, Lauren Hache, Erik Henricson, Fengming Hu, Jill Mayhew, Diana M. Escolar

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


Introduction: 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.

Original languageEnglish (US)
Pages (from-to)170-173
Number of pages4
JournalMuscle and Nerve
Issue number2
StatePublished - Aug 2011


  • Clinical trial
  • Duchenne
  • Leukopenia
  • Muscular dystrophy
  • Pentoxifylline

ASJC Scopus subject areas

  • Clinical Neurology
  • Physiology (medical)
  • Cellular and Molecular Neuroscience
  • Physiology


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