Abstract

BackgroundRiluzole is the only approved oral drug for amyotrophic lateral sclerosis (ALS). We performed a retrospective study including ALS patients treated with riluzole, focusing on adverse events.MethodsPatients diagnosed with ALS according to the revised El Escorial criteria (World Federation of Neurology) in our center and who were administered 50 mg oral riluzole twice daily between January 2011 and September 2017 and followed up for at least 6 months from treatment initiation or until death were included. Data regarding sex, age, disease type, initial symptoms, biochemical analyses performed before and after riluzole administration, and medical history were collected. In case of withdrawal, cause of discontinuation and durations of disease and drug administration were recorded.ResultsA total of 92 cases were enrolled. Riluzole administration was discontinued in 20 cases (21.7%). The most frequent reason for discontinuation was elevated liver enzymes (n = 5, 5.4%), followed interstitial pneumonia (IP), nausea and appetite loss, dizziness, general malaise, tongue paresthesia, and urinary urgency. In two cases, administration was discontinued primarily because of progression of bulbar palsy. All adverse events occurred within 6 months from treatment initiation and improved soon after its discontinuation. Three IP cases developed severe respiratory failure and required steroid treatment.ConclusionRiluzole administration was discontinued in 20 cases among total of 92 cases. Careful follow-up is important for the first six months after the initiation of riluzole administration, including through interviews, chemical analyses, and chest X-rays, as required.

Highlights

  • Riluzole is the only approved oral drug for amyotrophic lateral sclerosis (ALS)

  • No patient characteristics were significantly associated with drug discontinuation (Table 1)

  • All adverse events occurred within 6 months of riluzole initiation, with half of the events occurring within 14 days (Tables 2 and 3)

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Summary

Introduction

Riluzole is the only approved oral drug for amyotrophic lateral sclerosis (ALS). We performed a retrospective study including ALS patients treated with riluzole, focusing on adverse events. Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, primarily affecting upper and lower motor neurons. The only approved oral drug for ALS, is a presynaptic glutamate release inhibitor, which may prevent neural damage and delay muscle-strength deterioration. It decreases peak sodium (Na+) current and mediates voltage-gated Na + channel inactivation, which inhibit persistent Na + current in motor neurons, decreasing neuronal excitability and leading to neural protection [1, 18].

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