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Pentoxifylline in ALS: a double-blind, randomized, multicenter, placebo-controlled trial
V Meininger1, B Asselain, P Guillet
1Fédération des Maladies du Système Nerveux, Pavillon Paul Castaigne, AP-HP, Hôpital de la Salpétrière, Paris, France. vincent.meininger@psl.aphp.fr
Neurology
|January 13, 2006
Summary
Pentoxifylline did not improve survival in patients with amyotrophic lateral sclerosis (ALS) treated with riluzole. This drug should be avoided in ALS patients due to lack of benefit and potential harm.
Area of Science:
- Neurology
- Pharmacology
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
- Riluzole is a standard treatment for ALS.
- Pentoxifylline is a US Food and Drug Administration-approved drug with potential therapeutic properties.
Purpose of the Study:
- To evaluate the efficacy and safety of pentoxifylline in ALS patients already receiving riluzole.
- To determine if pentoxifylline impacts survival or functional decline in ALS.
Main Methods:
- A double-blind, randomized, placebo-controlled, multicenter trial involving 400 ALS patients.
- Patients received either placebo or 1.2 g of pentoxifylline daily.
- Primary outcome was all-cause mortality; secondary outcomes included functional and muscle strength deterioration.
Main Results:
- Pentoxifylline did not significantly improve survival; adjusted risk of death was higher in the pentoxifylline group (adjusted risk 1.43, p = 0.02).
- Secondary outcome analyses did not reveal a similar negative effect on functional measures.
- Adverse events like nausea and dysphagia were reported but were reversible.
Conclusions:
- Pentoxifylline offers no benefit for ALS patients treated with riluzole and should be avoided.
- Discrepancies between survival and functional outcomes highlight the need for careful endpoint selection in ALS clinical trials.