Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial

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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.

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.

GLOSSARY

AE: adverse event

GLOSSARY

CINRG: Cooperative International Neuromuscular Research Group

GLOSSARY

CTCAE: Common Terminology Criteria for Adverse Events

GLOSSARY

DMD: Duchenne muscular dystrophy

GLOSSARY

MMT: manual muscle testing

GLOSSARY

NCI: National Cancer Institute

GLOSSARY

PFA: platelet function assay

GLOSSARY

PFT: pulmonary function test

GLOSSARY

PT: prothrombin time

GLOSSARY

PTT: partial thromboplastin time

GLOSSARY

PTX: pentoxifylline

GLOSSARY

QMD: quantitative muscle testing

GLOSSARY

TGF-β: transforming growth factor-β

GLOSSARY

TNF-α: tumor necrosis factor-α

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