Effects of teprotumumab on patients with long-standing, active thyroid eye disease

PURPOSE: Describe five cases of long-standing, active thyroid eye disease that responded to treatment with teprotumumab. OBSERVATIONS: Five patients with a greater than 9-month-history of thyroid eye disease, including two patients who had previously failed orbital radiotherapy, received eight doses of teprotumumab. All five patients, including those with a history of orbital radiotherapy, achieved a proptosis reduction of at least 2 mm in each eye as well as a Clinical Activity Score reduction of at least 2 points. In addition, all cases of diplopia improved and all but one case of lagophthalmos improved. CONCLUSIONS AND IMPORTANCE: Teprotumumab may be a safe and efficacious therapy for active thyroid eye disease that is of longer duration than previously studied in clinical trials, as well as disease refractory to orbital radiotherapy. In addition to robust improvement in proptosis and Clinical Activity Score, data from this series suggests diplopia and lagophthalmos may also respond to teprotumumab. Further study of teprotumumab is needed, but in the meantime these results may encourage providers to consider teprotumumab for their patients with long-standing or previously treated disease.