Long-term teprotumumab regimen: thoughts on future trial design
Author: Steven Leibowitz
Base Hospital / Institution: UCLA Stein Eye Institute
ePoster presentation
Abstract ID: 24-355
Purpose
Teprotumumab, a human monoclonal antibody against IGF-1R, has been shown to be effective at reducing proptosis, diplopia, and inflammation in patients with thyroid eye disease. The indications have broadened in line with published post approval data from active inflammatory moderate, to include a broader spectrum of disease states. The optimal long term treatment paradigm is yet to be determined. We report a treatment responsive patient with multiple relapses following treatment cessation.
Methods
Case report of patient undergoing 4 cycles of teprotumumab. Review of the literature for conditions managed with immunotherapies with substantial clinical trial and real-world data with respect to tailoring long-term dosing regimens.
Results
A 44-year-old Chinese female with severe thyroid eye disease and a history of maximal surgical decompression required 4 courses of teprotumumab, due to repeated relapse following succesful treatment responses. Twelve weeks after completing her third course she experienced recurrence of her usual symptoms consisting of worsening eye pressure sensation, decreased color vision and seeing grey speckles. In addition, she was noted to have worsening clinical findings of decreased visual acuity at 20/40 (-3) and 20/40 (-2) on the right and left compared to 20/25 both sides during treatment, an increased Clinical Activity Score from 3 to 5, constant diplopia and reduction in upgaze by 5 degrees and increased exophthalmometry 1mm bilaterally.
Conclusion
This case demonstrates a chronic relapsing course of TED after cessation of treatment with teprotumumab. Long-term treatment regimens for such cases have not been defined. Re-treatment rate in a study of 5,845 patients was low at 4.9%, correlating with my experience of over 250 cases. Inferences from the clinical trial and real-world experience of more common autoimmune diseases management strategies have some merits, are untested in thyroid eye disease, and may potentially be used to guide future teprotumumab dosing regimen selection in clinical trials. These include dose escalation, treat to target and treat and extend strategies.
Additional Authors
| First name | Last name | Base Hospital / Institution |
|---|---|---|
| Taras | Gout | UCLA Stein Eye Institute |
| Robert | Goldberg | UCLA Stein Eye Institute |
