Teprotumumab for Thyroid-Associated Ophthalmopathy

Title: Teprotumumab for Thyroid-Associated Ophthalmopathy

Smith, TJ et al.


What You Need to Know:

Treatment with teprotumumab, a human monoclonal antibody inhibitor of insulin-like growth factor I receptor (IGF-IR), was more effective vs. placebo in reducing proptosis and the Clinical Activity Score in patients with active ophthalmopathy.

Trial Design:

  • Multicenter, double-masked, randomized, placebo-controlled trial conducted to evaluate teprotumumab in patients with active, moderate-to-severe ophthalmopathy
  • 88 patients randomized to placebo or active drug given intravenously (IV) once every 3 weeks for 8 infusions
  • Primary endpoint: response in the study eye defined as reduction of ≥2 points in the Clinical Activity Score (0-7) and a reduction of ≥2mm in proptosis at Week 24
  • Secondary endpoints: proptosis, Clinical Activity Score, results on Graves’ ophthalmopathy-specific quality-of-life questionnaire

Key Outcomes:

  • 69% of teprotumumab patients (29/42) had a response at Week 24 (P<0.001) vs. 20% of placebo patients (9/45)
  • 43% of teprotumumab patients (18/42) had a response at Week 6 (P<0.001) vs. 4% of placebo patients (2/45)
  • Differences between treatment arms increased at subsequent time points
  • Hyperglycemia was reported in patients with diabetes but was controlled by adjusting antidiabetic medications