Long-term real world teprotumumab outcomes

Abstract

Purpose : Teprotumumab, an IGF-IR inhibitor, was approved for the treatment of thyroid eye disease (TED) in January 2020. Two clinical trials demonstrated efficacy in patients with active moderate-to-severe TED and excluded patients with other types of TED or previous therapy with orbital irradiation, surgery, glucocorticoid use (cumulative dose > 1 gm), or biologic treatments. This study is the first to report the long-term real world outcomes in patients treated with teprotumumab for TED.

Methods : The pooled data of consecutive patients treated with teprotumumab for TED from 3 academic institutions were reviewed. Patient data were collected at baseline, post-treatment, and at long-term follow-up. All patients who completed treatment with ≥4 infusions were included. The percentage of proptosis responders (≥2 mm reduction) and mean changes from baseline in proptosis, Gorman diplopia score (GDS), clinical activity score (CAS), and Graves' ophthalmopathy quality-of-life (GOQoL) score at post-treatment and long-term were calculated. The percentage of patients who had proptosis regression (≥2 mm increase from post-treatment to long-term follow-up) was also calculated.

Results : 48 patients with a mean age of 57.9 (range 30-90) and mean CAS of 4.1 (range 1-7) were included (Fig 1). The mean post-treatment and long-term follow-ups were at 28.9±SD 11.0 and 57.44±11.1 weeks after the first infusion, respectively. Compared to baseline, post-treatment metrics demonstrated a significant decrease in mean proptosis (2.91±2.18, n=47), GDS (0.67±1.22, n=43), and CAS (3.32±1.37, n=44), all p<0.002. No significant difference occurred in these values between post-treatment and long-term (n = 40, 39, 34 respectively). The mean GOQoL gain between baseline and post-treatment (19.8±17.6, n=15, p<0.001) was maintained long-term (22.7±14.8, n=11). 70.2% were proptosis responders (n=47) post-treatment, but 28.2% (n=39) had proptosis regression. Of the regressors, 81.8% (n=11) continued to have inactive disease (CAS of 0 or 1) (Fig 2).

Conclusions : Our results confirm the efficacy and durability of teprotumumab in consecutive patients with TED using the metrics reported in clinical trials. Although regression of the initially robust proptosis response may occur, most patients do not develop reactivation of active TED. Further investigation on the long-term durability of proptosis response and reason for proptosis regression in a portion of patients is warranted.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.