Teprotumumab 500mg powder for solution for infusion vials

ImportanceDysthyroid optic neuropathy (DON) is a sight-threatening complication of thyroid eye disease (TED). The pivotal teprotumumab trials excluded patients with DON, and the only prior treatment meta-analysis addressed intravenous glucocorticoids and orbital decompression, leaving the role of teprotumumab in DON unsynthesised.ObjectiveTo systematically review and, where possible, meta-analyse visual and orbital outcomes following teprotumumab treatment for DON.MethodsPubMed, Ovid (MEDLINE/Embase) and Web of Science were searched from inception to 7 June 2026, following PRISMA 2020 guidelines. Eligible studies enrolled adults with DON treated with teprotumumab. Single-arm random-effects meta-analysis (inverse-variance, REML) pooled the proportion with DON resolution and mean changes in proptosis and best-corrected visual acuity (BCVA); risk of bias was assessed with the ROBINS-I tool.ResultsOf 310 unique records, 4 reports met eligibility. Four multi-patient series/cohorts (40 patients) were meta-analyzed. The pooled proportion with DON resolution was 0.77 (95% CI 0.58-0.89; k = 4, I2 = 0%). Proptosis fell by a pooled -4.80 mm (95% CI - 6.69 to -2.90; k = 3) but with substantial heterogeneity (I2 = 87%), with a two-study BCVA estimate of -0.56 logMAR. Visual fields, color vision and relative afferent pupillary defects improved in the large majority of eyes, often within one to two infusions; DON recurred in approximately 15% at long-term follow-up.ConclusionThe available data on teprotumumab for DON come from only a few small retrospective series without comparator groups, leaving certainty low to very low; even so, treated eyes showed reproducible and frequently early recovery of vision, reversal of optic neuropathy, and reversal of proptosis, and this lack of definitive trial evidence should not be seen as absence of benefit. Because the antibody acts directly on the orbital tissue expansion that compresses the optic nerve, it is a plausible first-line medical option for DON where it is accessible and patients can be monitored closely, provided that eyes failing to improve promptly are escalated without delay to orbital decompression and that emergency surgery remains the response to rapidly worsening sight loss. Adequately designed prospective comparative studies, with longer follow-up of recurrence, safety, and cost, are needed to establish teprotumumab's place in the DON treatment pathway.

Abstract licence: CC BY-NC-ND

BackgroundThyroid eye disease (TED) is a disabling, organ-specific autoimmune disease that is a global health concern. Recently, certain biological agents have demonstrated unique advantages for the treatment of TED. Teprotumumab is an emerging biological agent used for TED treatment. This study assessed whether teprotumumab can serve as an effective and safe treatment for active TED through a meta-analysis of the literature.MethodsWe searched 4 databases (PubMed, The Cochrane Library, Web of Science, and Embase) for randomized controlled trials regarding the treatment of Graves' ophthalmopathy by teprotumumab by March 31, 2024. We screened the literature library and extracted the data according to the inclusion and exclusion criteria.ResultsOur study included 5 articles that involved 411 cases. Significant differences were reported in the change from baseline in proptosis (proptosis vs baseline), diplopia response at week 24, and clinical activity score of 0 or 1 at week 24 in the teprotumumab versus placebo group. The teprotumumab group reported no significant risk of adverse events or serious adverse events during the intervention.ConclusionTeprotumumab significantly decreased proptosis and clinical activity score and improved diplopia response in patients with TED, with fewer adverse effects. Therefore, it is a promising biological agent. However, this conclusion should be further validated by high-quality, long-term randomized controlled trials with large sample sizes.

Abstract licence: CC BY