Use of the insulin-like growth factor I receptor teprotumumab led to long-term improvements in individuals with thyroid eye disease (TED), said Raymond Douglas, MD, PhD, during AAO 2020 Virtual. The director of the Orbital and Thyroid Eye Disease Program at Cedars-Sinai Medical Center in Los Angeles presented results from a phase 3 multicenter trial, which showed that teprotumumab produced better outcomes versus placebo for proptosis, clinical activity score, diplopia, and quality of life.
Participants received either teprotumumab (n = 41) or placebo (n = 42) once every 3 weeks for 21 weeks. After 24 weeks, the treatment group’s proptosis response was higher (83% vs 10%), as were the cohort’s overall response (78% vs 7%) and diplopia response (68% vs 29%) levels. Six in 10 of those receiving teprotumumab achieved a clinical activity score of 0 or 1, versus 2 in 10 in the placebo contingent. Graves’ ophthalmopathy-specific quality-of-life score was also superior in the treatment group (13.79 points vs 4.43 points).
In a 48-week off-treatment follow-up trial, 58% of patients who received the study drug who had ≥1 grade diplopia improvement at week 24 maintained that response at week 72 without additional TED treatment.
By all accounts, teprotumumab appears to be a breakthrough medication. In a press release issued last year, Dr Douglas said that “Currently, patients with Active TED suffer through life-altering symptoms and – once the active phase of disease ends – are often left with permanent damage.” Indeed, individuals with TED typically experience long-term functional, psychological, and economic burdens, including the inability to work and perform activities of daily living. Many with the condition withdraw from social obligations and stay indoors because of how they look, and because they cannot protect their bulging eyes from outdoor elements.
For them, teprotumumab is a potential game-changer. Dr Douglas categorizes the reduction in proptosis as “unprecedented.” In an interview earlier this year, he noted that the drug has resulted in “a remarkable improvement for patients. It’s amazing how life-transforming this has been….They are back to normal and do not have to worry about losing their vision, getting double vision, or other things that come with this disease such as eye-bulging and facial disfigurement.”