Teprotumumab use in a real-world setting: Expanded access program findings

Purpose : Teprotumumab was recently approved in January of 2020 to treat thyroid eye disease (TED). The purpose of this study was to evaluate the safety profile and Graves' ophthalmopathy quality of life questionnaire (GO-QOL) for a group of patients in which compassionate use of teprotumumab was deemed appropriate, prior to U.S. Food and Drug Administration (FDA) approval. Methods : Adults with active, moderate-to-severe TED were scheduled to receive 8 infusions (10 mg/kg first infusion, 20 mg/kg thereafter) of teprotumumab over 21 weeks. Adverse events (AEs), lab assessments, vitals and GO-QOL (max QOL=100) were assessed. Results : 22 patients (52.4±16.2 years, 64% female, 91% non-smokers, 7.2±3.0 months TED duration) from 8 sites were treated. 19/22 (86%) received 8 infusions (3 discontinued treatment due to COVID-19, personal choice, and hyperglycemia). Baseline Total GO-QOL was 47.8±21.4 (appearance [AP]: 42.9±26.7, visual function [VF]: 52.9±24.6). At Week 21, Total GO-QOL improved from baseline by 24.9±21.0 points (AP improved by 23.8±26.2, VF improved by 25.7±25.8), all large changes. All patients reported an AE. One patient suffered from appendicitis, which was deemed unrelated. Other AEs (>2 patients) included muscle spasms (n=11), fatigue (n=10), hypoacusis (n=5), headache (n=5), nausea (n=5), extremity pain (n=4), alopecia (n=4), hypertension (n=4), dry skin (n=3), diarrhea (n=3), tinnitus (n=3), myalgia (n=3), increased lacrimation (n=3), and hypogeusia (n=3). No new safety concerns were identified. Conclusions : Teprotumumab resulted in large QOL improvements as demonstrated previously in controlled clinical trials. Safety findings were consistent with the previously established teprotumumab profile.