Teprotumumab Treatment in Patients with Steroid and Surgery-Resistant Dysthyroid Optic Neuropathy: A Case Series
Authors' conclusion
The data suggest teprotumumab as a promising treatment for steroid- and surgery-resistant DON with rapid symptom improvement and long-lasting recovery.
Study context
- Population
- Patients with confirmed dysthyroid optic neuropathy (DON) (complication of GD orbitopathy) resistant to steroids and orbital decompression, Hospices Civils de Lyon; median age 58 years; median DON duration 2 months (IQR 2.0–6.5)8 eyes
- Participants
- 6 patients
- Outcome
- DON recovery, defined by changes in best-corrected visual acuity (BCVA) and visual field mean deviation (VF-MD)
- Follow-up
- End of teprotumumab treatment
Methodological detailssingle-centre
- Single-centre
Main finding
As reported in the abstract
DON recovery
Teprotumumab
87.5%
7
- Best-corrected visual acuity (BCVA) improvement in all patients, median (logMAR), p = 0.004
- 0.30 logMAR [0.24-0.42]
- Median visual field mean deviation (VF-MD) improvement, p = 0.024
- 66% (46-90)
Observational data: associations, not effects.
Further findings
DON improved after one infusion
- Patients with DON improvement after one infusion
- 3/6
Clinical activity score and proptosis
- All patients showed improvements in clinical activity score and proptosis
Durability over follow-up · median 73.8 weeks from first infusion
- Inflammatory relapse
- 2 patients
- Improvements persisted; no DON relapse
Safety
- Did not complete all infusions due to adverse events
- 2 patients
Original publication
Teprotumumab Treatment in Patients with Steroid and Surgery-Resistant Dysthyroid Optic Neuropathy: A Case Series. Thyroid. Published 28 August 2025. doi:10.
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