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Teprotumumab for the treatment of active thyroid eye disease

New England Journal of Medicine Jan 30, 2020

Douglas RS, Kahaly GJ, Patel A, et al. - Since thyroid eye disease is a debilitating, disfiguring, and potentially blinding periocular condition for which no Food and Drug Administration–approved medical therapy is available, researchers conducted a randomized, double-masked, placebo-controlled, phase 3 multicenter trial to evaluate the effect of teprotumumab for the treatment of active thyroid eye disease. They randomized individuals with active thyroid eye disease in a 1:1 ratio to receive intravenous infusions of the IGF-IR inhibitor teprotumumab (10 mg per kilogram of body weight for the first infusion and 20 mg per kilogram for subsequent infusions) or placebo once every 3 weeks for 21 weeks; the last trial visit for this analysis was at week 24. Compared with placebo, teprotumumab resulted in better outcomes with respect to proptosis, Clinical Activity Score, diplopia, and quality of life among individuals with active thyroid eye disease, serious adverse events were uncommon.
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