Purpose

Thyroid-related orbitopathy (TRO) is a complex autoimmune disorder associated with substantial morbidity. Although systemic corticosteroids and teprotumumab are established treatments for moderate-to-severe active disease, some patients remain refractory to steroids and do not have access to teprotumumab. Tocilizumab, a monoclonal antibody targeting the interleukin-6 (IL-6) receptor, has shown early promise as a therapy. This study evaluates the efficacy and safety of tocilizumab in a 19-patient case series representing the largest North American cohort reported to date.

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Methods

This retrospective case series evaluated 19 patients with active, moderate-to-severe TRO refractory to steroids who were treated with tocilizumab. Efficacy was assessed by comparing ocular symptoms and objective clinical measures between baseline and post-treatment follow-up. Safety was evaluated through reported adverse events and treatment discontinuations.

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Results

We studied 19 patients (16 female, 3 male). Mean age was 60 years (SD 8.8). Mean duration of treatment was 17 months (SD 11.9) with follow up length 3.7 years (SD 2.1). Treatment was stopped due to possible treatment related adverse events (hair loss, hepatotoxicity) in 2 (11%) patients. Meaningful improvement from baseline to post-treatment follow-up was observed in mean Clinical Activity Score (2.7 to 0.4), proptosis (6, 33%), diplopia (7, 39%), and resolution of periorbital edema (13, 72%), and ocular pain (7, 39%). No patients experienced clinically significant vision loss, and visual acuity improved in 6 (32%) patients and was unchanged in 13 (68%) patients.

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Conclusion

Our experience demonstrates that tocilizumab is effective in improving the clinical symptoms and inflammatory activity of TRO, while maintaining a favorable safety profile with no significant or unexpected adverse effects.

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Additional Authors

First name	Last name	Base Hospital / Institution
Adam	Wandzura	University of Alberta
Kelsey	Roelofs	University of Alberta
Ezekiel	Weis	University of Alberta

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