Teprotumumab for the treatment of thyroid eye disease: Clinical experience from expanded access program (EAP)

ABSTRACT

Purpose: Teprotumumab was approved in January of 2020 for the treatment of thyroid eye disease (TED). The purpose of this study was to evaluate additional efficacy parameters in patients receiving teprotumumab in the EAP (NCT04040894), which was initiated prior to the drug's approval by the US. Food and Drug Administration. Methods: This was a retrospective cohort study of patients who received teprotumumab at one study center. Eligible patients included those who were at least 18 years old with a clinical diagnosis of active, moderate-to-severe TED with a clinical activity score (CAS) equal to/greater than 4 with onset of TED within 12 months. Patients were provided 8 infusions (10 mg/kg first infusion, 20 mg/kg thereafter) every 3 weeks over course of 21 weeks. End points included changes in proptosis, intraocular pressure (IOP), extraocular motility deficit, CAS, photophobia score (visual light sensitivity questionnaire-8 [VLSQ-8]), basic secretion test, volumetric analysis of facial compartments (Canfield Vectra H2 camera). Paired t-tests were used to evaluate statistical significance from baseline. Results are presented for the more severe (study) eye. Results: 13 patients (4 males, 9 females) were included in the analysis. 10/13 patients were Caucasian. Average age was 46.5±15.9 years with a mean TED duration of 7.1±3.0 months. 10/13 (77%) received the complete set of 8 infusions (3 discontinued due to COVID-19, personal choice, and hyperglycemia). At week 21, the change from baseline (CFB) was -4.6±2.1 mm (n=11) for proptosis and -4.0±1.6 (n=11) for CAS (both p<0.001), -9.1±5.0 (n=11;p<0.001) for light sensitivity, and -3.6±3.4 mmHg (n=9;p<0.05) for IOP. 6 patients (n=12) had abnormal wetting test at eligibility visit, compared to 3 at week 21 (n=10). 6 patients had manifest strabismus, 5 of who had complete data. Out of the 5 patients, all but one patient had improvements in either extraocular motility or strabismus measurements. Facial volumetric analysis demonstrated reductions in both upper (CFB -1.4±0.7 cc;n=4;p=0.03) and lower lid volume (CFB -2.2±1.3 cc;n=4;p=0.04). Conclusions: These results support previously reported teprotumumab improvements in proptosis and CAS. Data from this cohort of patients suggest that teprotumumab may also be effective in improving light sensitivity, IOP, motility deficit, dry eyes, and periorbital edema.