Bilateral dysthyroid compressive optic neuropathy responsive to teprotumumab

Abstract

Thyroid eye disease is an auto-immune mediated orbitopathy which can cause dysthyroid compressive optic neuropathy. Traditional management of active thyroid eye disease includes temporizing high-dose steroids, orbital radiation and surgical decompression, which each possess significant limitations and/or side effects. Teprotumumab is an IGF-IR inhibitor recently FDA-approved for active thyroid eye disease. The authors report reversal of bilateral dysthyroid compressive optic neuropathy managed medically utilizing teprotumumab.

Updates on the understanding and management of thyroid eye disease

“…Several case reports describe its efficacy treating severe TED cases with CON, with improvements in VA, relative afferent pupillary defect (RAPD), CAS, Humphrey visual fields, proptosis, and extraocular muscle size after two to three infusions. 92 – 95 In a case series of 10 patients with active and chronic TED and CON, 70% of patients showed improvement of VA, RAPD, or both after two infusions. 96 On average, patients had 4.7 mm of proptosis reduction and 5.25 points of CAS improvement, and of the seven patients with color vision deficits, six had normalization of their color vision.…”

Efficacy and Safety of Teprotumumab in Thyroid Eye Disease

“…Of the 15 reported cases, 9 had complete resolution of CON while 6 cases had improved CON. [31][32][33][34][35][36] The onset of improvement was as early as after 2 doses of teprotumumab in 6 of these cases. 32,36 Data on proptosis reduction and CAS improvement were also available in 13 of the 15 reported cases.…”

“…After only 2 doses, proptosis reduction was noted in 5 of 13 cases and CAS improvement in 9 of 13 cases. [31][32][33][34]36 In addition, these responses persisted at 3-33 weeks after the final 8th dose. [31][32][33]36 Radiographic data was available for 7 cases, and all showed reduction in both extraocular muscle size and apical crowding, supporting the clinical evidence of CON resolution or improvement.…”

“…[31][32][33][34]36 In addition, these responses persisted at 3-33 weeks after the final 8th dose. [31][32][33]36 Radiographic data was available for 7 cases, and all showed reduction in both extraocular muscle size and apical crowding, supporting the clinical evidence of CON resolution or improvement. While these reports suggest that teprotumumab may be an effective therapy for CON, further studies should help identify clinical factors that would be predictive of a positive response to teprotumumab, including duration and/or severity of CON.…”

“…Nevertheless, since the FDA approval of teprotumumab, 6 publications have reported on 15 cases demonstrating favorable effects of teprotumumab for CON (Table 1). [31][32][33][34][35][36] All 15 patients had failed to improve with or were not candidates for conventional treatments including systemic corticosteroids, orbital radiotherapy, and/or surgical decompression. All had poor optic nerve function, which was characterized by decreased visual acuity (VA) and/or color vision, presence of relative afferent pupillary defect (RAPD), and visual field (VF) defect.…”