Abstract:AIM: To explore the efficacy of teprotumumab N01 (IBI311), an insulin-like growth factor-1 receptor (IGF-1R) inhibitor, in thyroid eye disease (TED) patients who had failed glucocorticoids. METHODS: In this pilot case series study, patients with TED failing intravenous or oral glucocorticoid treatments were treated with teprotumumab N01 (10 mg/kg for the first infusion then 20 mg/kg every 3wk for subsequent 3 infusions). Proptosis, clinical activity score (CAS), Gorman diplopia score, magnetic resonance imaging (MRI), adverse events and laboratory tests were evaluated during the treatment. RESULTS: The patients’ mean age was 38.7y, 6 of 10 were female, and TED duration of these patients ranged from 0.8 to 5y. At week 12, 7 patients had proptosis response, 6 achieved a CAS of 0 or 1 and 5 achieved an overall response. For patients with proptosis improvement, MRI also showed consistent changes including volume decrease of extraocular muscles and orbital fat tissues. The incidence of adverse events was low. CONCLUSION: Teprotumumab N01 infusions result in proptosis response and clinical activity improvements accompanied with favorable safety profile in TED patients who had failed glucocorticoids. Randomized controlled trials will be conducted to further assess the efficacy and safety of teprotumumab N01 in TED patients.
