94 - TEPROTUMUMAB EFFICACY AND SAFETY IN AN OPEN-LABEL (OL) EXTENSION IN PATIENTS WITH CHRONIC THYROID EYE DISEASE (TED)
1Amgen. 2Sue Anschutz-Rodgers University of Colorado Eye Center, Aurora, CO, USA. 3Washington University School of Medicine, St Louis, MO, USA. 4Bascom Palmer Eye Institute, University of Miami, Miami, FL, USA. 5The University of Tennessee Health Science Center Hamilton Eye Institute, Memphis, TN, USA. 6Department of Ophthalmology, Shiley Eye Institute, University of California San Diego, La Jolla, CA, USA. 7Neuro-Eye Clinical Trials, Inc., Houston, TX, USA. 8Amgen Inc, Thousand Oaks, CA, USA. 9Division of Endocrinology, Diabetes and Metabolism, Mayo Clinic, Rochester, MN, USA.
Introduction: Thyroid Eye Disease (TED) can lead to chronic and symptomatic disease with pain and proptosis. Teprotumumab has demonstrated efficacy in patients with acute and chronic TED. In the first placebo-controlled, double-blind trial (NCT04583735) in patients with chronic TED (2-10 years disease duration and clinical activity score #2 1), teprotumumab improved proptosis and visual function-quality of life.1 We report teprotumumab safety and efficacy in the open-label extension of this trial.
Methods: Proptosis non-responders (< 2 mm improvement) from the randomized period of the trial could receive open-label teprotumumab (8 infusions over a 24-week treatment period).
Results: Of 24 patients in the open-label extension, 12 patients who were previously treated with placebo received a first course of teprotumumab (PBO/TEP) and 12 patients who were previously treated with teprotumumab received a second course (TEP/TEP). At week 24 of the open-label extension, mean (SD) proptosis change from pre-teprotumumab was -2 (1.2) mm for PBO/TEP and -1.6 (1.2) mm for TEP/TEP; and 7/12 (58.3%) PBO/TEP and 5/12 (41.7%) TEP/TEP were proptosis responders. Adverse events (AEs) were reported in 11 (91.7%) PBO/TEP and 8 (66.7%) TEP/TEP patients, with no serious AEs or deaths. No TEP/TEP patients and 3 PBO/TEP patients reported hearing AEs (eustachian tube dysfunction, hypoacusis, tinnitus).
Conclusions: Delayed treatment (PBO/TEP) had similar outcomes as teprotumumab in first 24 weeks. Additional teprotumumab therapy (TEP/TEP) proved beneficial without added safety concern in about 40% of patients with prior non-response.
Presentada previamente en: North American Neuro-Ophthalmology Society (NANOS) Meeting 2025, March 15-20, 2025, Tucson, AZ, Marriott Starr Pass.
