Endocrine Abstracts

Purpose: To describe pre- to post-treatment changes in clinical activity score (CAS) in patients with Graves orbitopathy treated with tocilizumab (TCZ).

Introduction: Graves’ ophthalmopathy (GO) is an autoimmune disorder that affects 30-50% of patients with Graves’ disease. The first-line treatment of moderate to severe GO according to EUGOGO is intravenous glucocorticoids, but 20-30% of the cases appear to be corticosteroid refractory. Steroid-resistant GO constitutes a major clinical and therapeutic challenge but promising results have been described with TCZ as a second-line moderate to severe GO treatment. We report a case series of severe GO treated with tocilizumab.

Case report 1: 41-year-old man, former smoker who was diagnosed two years ago with hyperthyroidism and GO, presented in our hospital for reevaluation. The patient was in a thyrotoxic state and he was treated with methimazole and with intravenous methylprednisolone (cumulative dose 4.1 gr) with only partial response. He presented with unilateral eyelid swelling, orbital pain, redness and tearing. His CAS was 5 out of 7. A MRI orbits scan showed enlargement of the extraocular muscles, more prominent in the right orbit. The patient rejected radiotherapy. We initiated therapy with TCZ, a monoclonal antibody that inhibits the IL-6 receptor. ‘Off-label’ authorization for the use of tocilizumab was asked to Pharmacy Committee of our center. After its approbation, he received 4 intravenous doses (8 mg/kg) every 28 days, according to Perez-Moreiras RCT. On follow up, symptoms were improved impressively, and CAS score regressed to 1/7.

Case report 2: 52-year-old men diagnosed with Graves’ hyperthyroidism and GO one year ago, presented in our hospital for corticosteroid resistant GO. The patient had no general symptoms of thyroid disease. He previously received bolus of methylprednisolone with a weak response. He presented with bilateral eyelid swelling, orbital pain, redness and visual loss on the left side. His CAS was 4 out of 7. Due to the severity and the progression of the disease despite the corticosteroid treatment, we decided to initiate the treatment with TCZ. He received 4 intravenous doses (8 mg/kg) every 28 days. On follow up, symptoms were significantly improved including improved vision in his left eye, and CAS score regressed to 1/7.

Conclusions: We present this cases to raise awareness for the relative efficacy and tolerability profile of intravenous tocilizumab in the management of moderate to severe, corticosteroid resistant or refractory GO, of recent-onset, with lower cost compared to other novel therapies such as teprotumumab.