Teprotumumab treatment for dysthyroid optic neuropathy

While previous small studies report positive effects of teprotumumab on thyroid eye disease-associated compressive optic neuropathy (TED-CON), the timing and duration of CON improvement remains unclear. This study analyzes the efficacy, timing, and durability of teprotumumab treatment for TED-CON.

Retrospective study of all patients who completed eight teprotumumab infusions at one institution from January 1 2020 to December 31 2022. Primary outcome was CON resolution rate after teprotumumab. Secondary outcomes included number of infusions to CON improvement or resolution, factors impacting resolution, and recurrence rates at late follow-up.

Of 129 patients who completed teprotumumab, 35 (13.6%) eyes of 21 patients had TED-CON: 4 (11.4%) eyes had disc edema, 15 (42.9%) had dyschromatopsia, and 32 (91.4%) had Humphrey visual field defects. Of 19 patients with CON clinical data after initiating teprotumumab, 100% of eyes had CON improvement after a median of 1.5 infusions (range 1-7), with improvement after only one infusion in 43.8%. CON resolved in 25 (78.1%) eyes of 16 patients after a median of 4 infusions (range 1-8). Despite proptosis regression at most recent follow-up (median 20.4 months) in 11 (55.0%) TED-CON patients averaging 2.9 mm (range 0.5-6.0, SD 1.9), only 3 (15.0%) patients had recurrence of CON.

All patients had CON improvement with teprotumumab, typically over a rapid time course, and the vast majority had CON resolution. Despite proptosis regression, nearly all patients remained CON-free at long-term follow-up. These results suggest that teprotumumab may be an effective treatment for dysthyroid optic neuropathy, especially for patients with mild CON.