Abstract
Purpose :
Teprotumumab is approved for the treatment of thyroid eye disease and recent studies suggest efficacy in treating dysthyroid optic neuropathy (DON). This case series reports 4 cases of recurrent or progressive dysthyroid optic neuropathy after treatment with teprotumumab.
Methods :
A single center retrospective chart review was conducted. Patients were included if they were diagnosed with DON, treated with at least 4 infusions of teprotumumab, and developed recurrent DON within 1 year of treatment with teprotumumab. Patient demographics, histories, clinical data, lab values, visual fields, and imaging were analyzed.
Results :
Four female patients were included. Three patients (ages 68, 65, 71) with a history of pre-diabetes presented with recurrent DON symptoms 43.3±0.2 weeks after their first course of 8 infusions. One patient (age 82), despite initial improvement on teprotumumab, experienced worsening of symptoms after 4 infusions. While visual fields and/or color vision improved after initial teprotumumab treatment, all 4 experienced worsening visual fields and/or color vision in the same eye or eyes previously affected.
All patients needed additional treatment to address the recurrent DON. Of the three with recurrent symptoms 43 weeks after treatment, two received an additional course of teprotumumab and one intravenous methylprednisolone (IVMP); all 3 underwent decompression while receiving pharmacologic treatment. The patient who experienced worsening DON after her 4th infusion underwent orbital decompression with perioperative steroids and later completed the remaining 4 teprotumumab infusions.
Conclusions :
A pooled analysis of the pivotal studies for teprotumumab demonstrated that 33% (19/57) and 31% (15/48) of patients developed recurrent proptosis and double vision, after initial response, at 51 week follow up after treatment, respectively. Similarly, providers should be aware that patients with DON treated with teprotumumab may develop recurrent soft tissue expansion which can result in recurrent DON requiring additional therapy. Due to the 20-day half-life of the drug, recurrent disease may occur later than expected and patients should be followed closely during the first year after treatment. These 4 cases demonstrate the challenge in the management of DON with teprotumumab. Further studies are needed to evaluate the long-term efficacy of teprotumumab for DON.
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.