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Teprotumumab - Interpreting the Clinical Trials in the Context of Thyroid Eye Disease

Article Title: Teprotumumab: interpreting the clinical trials in the context of thyroid eye disease pathogenesis and current therapies

Article Summary:

Teprotumumab is a monoclonal antibody targeted against the insulin-like growth factor-1 (IGF-1) receptor and is used for the treatment of thyroid eye disease (TED). Two multicenter, randomized, double-masked, clinical trials (phase II and III) evaluated its efficacy in patients with recent-onset TED. Teprotumumab infusions were given every 3 weeks for a total of 8 infusions in comparison to placebo (n=170). Teprotumumab was found to be superior to placebo in both studies and reduced proptosis significantly by more than 2 mm. It also resulted in a greater number of patients with a clinical activity score (used to quantify inflammatory signs of the disease) of 0 or 1, a higher diplopia responder rate, and overall improvement in the Grave’s Ophthalmopathy Quality of Life score. Adverse side effects included muscle spasms, nausea, alopecia, diarrhea, fatigue, hearing impairments, and hyperglycemia. Limitations included contraindications in individuals who are pregnant or diagnosed with inflammatory bowel disease. It should also be noted that this drug is not yet available in Canada.

Summary By: Sarah Yeo

Article Published: April 2021

Reference: Winn BJ, Kersten RC. Teprotumumab: Interpreting the Clinical Trials in the Context of Thyroid Eye Disease Pathogenesis and Current Therapies. Ophthalmology. 2021 Nov;128(11):1627-1651. doi: 10.1016/j.ophtha.2021.04.024. Epub 2021 Apr 28. (


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