Teprotumumab for chronic thyroid eye disease.

Area of Science:

• Ophthalmology
• Endocrinology
• Immunology

Background:

• Thyroid eye disease (TED) is an autoimmune condition affecting the eye socket.
• Chronic TED with low clinical activity scores (CAS) presents a treatment challenge.
• Teprotumumab is an insulin-like growth factor 1 receptor (IGF-1R) inhibitor.

Purpose of the Study:

• To evaluate the efficacy of teprotumumab in patients with chronic, low CAS TED.
• To assess changes in proptosis and eyelid retraction following teprotumumab treatment.
• To document adverse events associated with teprotumumab in this patient population.

Main Methods:

• Retrospective case series of nine adult patients with chronic TED (duration > 9 months) and low CAS (≤1).
• Patients received a full course of eight teprotumumab infusions.
• Primary outcomes included reduction in proptosis and eyelid retraction; secondary outcomes included CAS and adverse effects.

Main Results:

• Significant reduction in proptosis observed immediately post-treatment (mean 4.0 mm, p=0.02) and at extended follow-up (mean 4.2 mm, p=0.03).
• Eyelid retraction showed a trend toward reduction but was not statistically significant.
• Adverse events included infusion-related myalgias, hair thinning, tinnitus exacerbation, and hyperglycemia.

Conclusions:

• Teprotumumab treatment led to clinically and statistically significant proptosis reduction in patients with chronic, low CAS TED.
• Teprotumumab demonstrates potential as an effective therapeutic option for this specific TED patient cohort.
• Further research may explore long-term outcomes and optimal use in chronic TED.