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  • Teprotumumab for Inactive TED

    By Lynda Seminara
    Selected By: Prem S. Subramanian, MD, PhD
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    Journal Highlights

    Eye
    Published online Nov. 21, 2020

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    Teprotumumab, an insulin-like growth factor 1 receptor (IGF-1R) inhibitor, has been found to reduce proptosis and inflammation in active thyroid eye dis­ease (TED). Ugradar et al. investigated whether the drug also could clinically benefit patients with inactive TED characterized by progressive strabis­mus, proptosis, or optic neuropathy but no inflammation—a group for whom there is no accepted medical treatment.

    They found that teprotumumab reduced IGF-1R expression in patients with in­active disease relative to controls. They also determined that overexpression of IGF-1R was significantly more perva­sive in cases of inactive and active TED than in controls.

    In this clinical series, consecutive patients who had noninflammatory TED for at least four months (clinical activity score [CAS] ≤1) received a full course of teprotumumab (eight infu­sions total; 10 mg/kg initially, followed by 20 mg/kg every three weeks). The primary outcome measure was propto­sis response at week 24. In addition, orbital tissues from patients with either stage of TED and healthy controls were histologically analyzed for expression of IGF-1R α and β.

    A total of four patients (age range, 21-47 years) met the study’s eligibility criteria. Before treatment, their mean (standard deviation [SD]) CAS was 0 (0). After treatment, their mean (SD) proptosis value decreased by 2.6 mm (1.2; p < .01), but their CAS and diplo­pia scores remained at 0.

    For the histological analysis, samples were obtained from five patients with noninflammatory TED, five patients with inflammatory TED, and five con­trols (age range, 29-70 years). The mean numbers of regions staining positive for IGF-1R α and β antibodies differed significantly between noninflammatory and inflammatory TED (both p < .01) and between noninflammatory TED and controls (both p < .01).

    Despite the small sample size, the significant reduction in proptosis at­tained with teprotumumab in nonin­flammatory TED suggests its potential as medical therapy, said the authors.

    The original article can be found here.