Valproic Acid Is an Effective Anti-Emetic Treatment in Alexander Disease

    April 2023 in “ Neurology ”
    Emily Sergio, Kathryn Gallison, Giulia Porcari, Joshua Joung, Walter Faig, Geraldine Liu, Amy Waldman
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    TLDR Valproic acid is a more effective treatment for vomiting in Alexander disease than other medications, with few side effects.
    The study investigated the effectiveness of valproic acid (VPA) as an anti-emetic treatment in Alexander disease (AxD) by reviewing medical records of 34 patients under 21 years old. The results showed that 95% of patients treated with VPA experienced a clinically meaningful decrease in emesis, significantly higher than the response rates for anti-reflux medications (48%) and cyproheptadine (32%). Despite some adverse effects like bone fractures, hyperammonemia, and mild hair loss, VPA was generally well tolerated. The findings suggest that VPA is a highly effective treatment for emesis in AxD patients, though monitoring for side effects is necessary.
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