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

ABSTRACT: 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.The tolerability and safety of PTX and measures of muscle strength and function were evaluated during 12 months of treatment.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.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.

SUBMITTER: Zimmerman A

PROVIDER: S-EPMC3136640 | biostudies-literature | 2011 Aug

REPOSITORIES: biostudies-literature

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

Zimmerman Angela A Clemens Paula R PR Tesi-Rocha Carolina C Connolly Anne A Iannaccone Susan T ST Kuntz Nancy N Arrieta Adrienne A Hache Lauren L Henricson Erik E Hu Fengming F Mayhew Jill J Escolar Diana M DM

Muscle & nerve 20110614 2

<h4>Introduction</h4>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.<h4>Methods</h4>The tolerability and safety of PTX and measures of muscle strength and function were evaluated during 12 months of treatment.<h4>Results</h4>Seventeen boys with DMD, between 4 and 8 years of age, were enrolled a ...[more]

PMID: 21674534

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Project description:Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in childhood. It is caused by mutations of the DMD gene, leading to progressive muscle weakness, loss of independent ambulation by early teens, and premature death due to cardiorespiratory complications. The diagnosis can usually be made after careful review of the history and examination of affected boys presenting with developmental delay, proximal weakness, and elevated serum creatine kinase, plus confirmation by muscle biopsy or genetic testing. Precise characterization of the DMD mutation is important for genetic counseling and individualized treatment. Current standard of care includes the use of corticosteroids to prolong ambulation and to delay the onset of secondary complications. Early use of cardioprotective agents, noninvasive positive pressure ventilation, and other supportive strategies has improved the life expectancy and health-related quality of life for many young adults with DMD. New emerging treatment includes viral-mediated microdystrophin gene replacement, exon skipping to restore the reading frame, and nonsense suppression therapy to allow translation and production of a modified dystrophin protein. Other potential therapeutic targets involve upregulation of compensatory proteins, reduction of the inflammatory cascade, and enhancement of muscle regeneration. So far, data from DMD clinical trials have shown limited success in delaying disease progression; unforeseen obstacles included immune response against the generated mini-dystrophin, inconsistent evidence of dystrophin production in muscle biopsies, and failure to demonstrate a significant improvement in the primary outcome measure, as defined by the 6-minute walk test in some studies. The long-term safety and efficacy of emerging treatments will depend on the selection of appropriate clinical end points and sensitive biomarkers to detect meaningful changes in disease progression. Correction of the underlying mutations using new gene-editing technologies and corticosteroid analogs with better safety profiles offers renewed hope for many individuals with DMD and their families.

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

Publications

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

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