The results of a real-world treatment for patients with thyroid eye disease (TED) who also have dysthyroid optic neuropathy (DON) indicated that Tepezza (teprotumumab-trbw) was effective for managing DON, according to recent study results.
Teprotumumab is the first medicine approved by the United States Food and Drug Authority (FDA) to treat patients with TED, which is a serious, progressive, and potentially vision-threatening rare autoimmune disease.
DON is a serious complication of TED, characterised by thyroid-related impairment of visual function that can lead to permanent vision loss related to optic nerve compression.
In a news release,1 Horizon Therapeutics said analysis of the real-world experience of people living with TED and DON, treated with Tepezza between 2020 and 2022 showed “meaningful improvement”.
In two cases, teprotumumab was given as monotherapy, which led to complete resolution of DON
During this period, 24 patients were identified with 22 of those receiving treatment with oral or intravenous steroids, surgery and/or radiation prior to Tepezza.
None of the patients had succeeded with previous treatments of steroids, radiation, and surgery. Following teprotumumab, the DON improved in 19 of 22 patients.
Three patients had longstanding DON with poor vision and optic atrophy and did not show improvement. In two cases, teprotumumab was given as monotherapy, which led to complete resolution of DON.
After Tepezza treatment, meaningful improvement was observed in visual acuity in eyes with DON (mean lines of improvement of 3.7 – 23 patients, range 1–15, mean improvement in the mean deviation on visual field testing of -5.87 – 7 patients); proptosis (mean reduction of 4.4 mm – 19 patients); diplopia (mean improvement of 0.75 grade – eight patients); and Clinical Activity Score (mean reduction of 5.1 – 17 patients). Reported adverse events were consistent with Tepezza clinical trials and included fatigue, dysgeusia, hearing issues, nausea, hyperglycemia, and muscle spasms.
Risk of Blindness
“We know how devastating Thyroid Eye Disease can be for patients, and the risk of blindness in those with dysthyroid optic neuropathy is one of the most worrisome complications, especially when traditional therapies fail,” said study author Associate Professor Madhura Tamhankar, from the Hospital of the University of Pennsylvania.
“These data are encouraging for physicians looking for an option to treat patients at the highest risk of losing their sight and provide evidence that Tepezza can help improve symptoms and preserve visual acuity.”
Horizon Therapeutics said the take-home messages regarding teprotumumab are that the drug is effective for managing DON in most patients in whom the conventional therapies such as steroids, orbital radiation, and orbital decompression caused minimal to no improvement or progressive visual loss; the improvement in vision was sustained in most patients after the teprotumumab infusions were completed; and the two patients who received teprotumumab as the first-line therapy had complete and sustained improvement in DON.
Reference
- Horizon Therapeutics, Real-World Experience Shows TEPEZZA® (teprotumumab-trbw) Was Effective in Treating People With Dysthyroid Optic Neuropathy (DON), news release, 12 March 2023, available at businesswire.com/news/home/20230310005395/en/Real-World-Experience-Shows-TEPEZZA%C2%AE-teprotumumab-trbw-Was-Effective-in-Treating-People-With-Dysthyroid-Optic-Neuropathy-DON [accessed 7 June 2023].
