Teprotumumab study finds mixed response rates for active thyroid eye disease

Teprotumumab therapy for active thyroid eye disease demonstrates variable response rates and limited long-term improvement, based on data from a retrospective case series.

While 33% of patients maintained a response 2 years after treatment, the study reveals that proptosis and clinical activity score (CAS) regression often occur in the context of disease reactivation in a significant portion of cases (80%). In addition, teprotumumab treatment appears to offer minimal long-term improvement in diplopia.

The cohort comprised 21 patients, and the primary outcome measure was reactivation, defined as a regression in proptosis (≥2mm increase in either eye, returning within ≤2mm of pre-treatment levels) and CAS worsening by ≥2 points. The secondary outcome assessed diplopia response.

Results showed that the average long-term improvement in proptosis in the eye with more proptosis after teprotumumab was 1.57mm (range, -3-4 mm). Of the initial responders (17 out of 21 patients), 47% experienced reactivations, while 12% had isolated proptosis regressions. Overall, only 33% of patients demonstrated continued response throughout the 2-year study period. The average time to regression was 12.25 months (range, 2-22.5 months).

There was no statistically significant change in diplopia at the final visit in any subgroup, suggesting teprotumumab treatment offers minimal long-term improvement in diplopia.

Reference
Hwang CJ, Rebollo NP, Mechels KB, Perry JD. Reactivation after teprotumumab treatment for active thyroid eye disease. Am J Ophthalmol. 2023;S0002-9394(23)00504-4. doi: 10.1016/j.ajo.2023.12.001. Epub ahead of print. PMID: 38142982.