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  • Long-Term Effectiveness of Teprotumumab

    By Lynda Seminara
    Selected by Stephen D. McLeod, MD, and reviewed by Russell N. Van Gelder, MD, PhD
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    Journal Highlights

    Ophthalmology, April 2022

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    Douglas et al. evaluated the long-term effect of teprotumumab for thyroid eye disease (TED) in an open-label study (OPTIC-X) that lasted 48 weeks beyond the OPTIC 72-week study. They found that 90% of week-72 responders did not require additional treatment in the extension period. In addition, 89% of the OPTIC placebo group became teprotumumab responders in OPTIC-X.

    For this study, participants with a 72-week response to teprotumumab in OPTIC were contacted at 96 and 120 weeks to determine if they required treatment for TED since their initial involvement in OPTIC. Treatment non-responders, patients with a flare, and the placebo group of OPTIC were enrolled in OPTIC-X. Flare was defined as proptosis increase of at least 2 mm, elevation of 2 or more points in clinical activity score (CAS), or both. Main outcome measures were proptosis response and safety findings. Patient-reported quality of life (QoL) was a secondary outcome.

    Overall, 33 (89.2%) of the 37 OPTIC placebo recipients became proptosis responders to teprotumumabin OPTIC-X (–3.5 ± 1.7 mm). The degree of response matched that in OPTIC. Among these responders, the CAS, proptosis, and diplopia responses were maintained through week 48 in 95.2%, 90.6%, and 85.7%, respectively. Of the five initial nonresponders who were re-treated in OPTIC-X, two had a successful proptosis response, one had a 1.5-mm proptosis reduction from OPTIC baseline, and two discontin­ued treatment early. Re-treatment was successful in five of eight OPTIC re­sponders who experienced flare (mean proptosis reduction, 1.9 mm from OPTIC-X baseline and 3.3 mm from OPTIC baseline). No new safety signals were observed in the extension study, but pharmacovigilance continues. Mild hearing impairment was reported by four patients.

    Results indicate that patients with delayed TED treatment respond simi­larly to those with early treatment and that initial nonresponders to teprotu­mumab may benefit from further treat­ment. The findings require validation in larger studies, said the authors.

    The original article can be found here.