Teprotumumab Demonstrates Swift Improvements in Thyroid Eye Disease

Fundoscopy showing optic nerve inflammation, a form of opticneuropathy.
Most patients saw a reversal of relative afferent pupillary defect and resolution of color vision deficits, the study says.

The following article is a part of conference coverage from the 2021 meeting of the North American Neuro-Ophthalmology Society, being held virtually from February 20 to 23, 2021. The team at Ophthalmology Advisor will be reporting on the latest news and research conducted by these leading experts in neuro-ophthalmology. Check back for more from the NANOS 2021 Meeting.


Most patients with thyroid eye disease (TED) who underwent treatment with teprotumumab saw a number of improvements, according to a retrospective interventional case series. The research, presented at the North American Neuro-Ophthalmology Society’s 2021 Annual Meeting, shows that the treatment can result in rapid improvements, often after the first or second infusion.

The researchers reviewed 10 patients with a TED diagnosis at 5 facilities. They found that treatment with 10 mg/kg dose at the first treatment and 20 mg/kg dose at subsequent treatments resulted in improvements to patients’ visual acuities as well as a reversal of relative afferent pupillary defect (RAPD), resolution of color vision deficits, improvement of visual field deficits, and improvement in apical compression seen on orbital imaging. 

In TED, insulin-like growth factor type 1 receptors (IGF-1R) and thyrotropin receptors (TSHR) colocalize in orbital fibroblasts, resulting in an inflammatory response, tissue expansion, and remodeling, investigators explain. Teprotumumab blocks IGF-1R and inhibits fibroblast activation via the IGF-1R/TSHR signaling complex at the source of the disease. Before this option became available, patients with TED would be treated with high-dose steroids, orbital radiation, or surgical decompression. 

In this study, a total of 6 patients presented with RAPD. Only 1 patient with RAPD did not achieve complete resolution although that patient did experience some improvements. In addition, 7 patients presented with color vision deficits, and of those, only 1 patient did not reach complete normalization (the same patients in whom RAPD did not fully resolve). However, researchers did note early improvements in that patient’s color vision, too. Improvements were also seen in proptosis and clinical activity scores.

Patients with long-standing TED compressive optic neuropathy (CON), optic atrophy, or globe tenting on imaging were all less likely to achieve significant improvement in vision, the study shows. The research advocates for further investigation into the therapy as a treatment option for TED CON, particularly when other therapies have failed.

Disclosure: Several study authors declared affiliations with the biotech or pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.

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Sears C, Kang J, Wang Y, et al. Effect and durability of teprotumumab for optic neuropathy secondary to thyroid eye disease. Presented at: North American Neuro-Ophthalmology Society Annual Meeting; February 20-23, 2021; Poster 131.