Real world effectiveness of Mycophenolate-sodium therapy in patients at risk with Graves' orbitopathy

Patients with active, moderate-to-severe Graves' orbitopathy require immunosuppressive treatments to reduce inflammation and morbidity. Since 2021 EUGOGO lists Mycophenolate-sodium (MPS) as first-line-treatment, which lead to a change in treatment regimens. In our center MPS was administered mainly for patients at risk for deterioration (e.g. unstable thyroid function, smoker etc.) or as second-line treatment. To augment the limited data we analyzed our real-world cohort retrospectively.

We analyzed all consecutive patients of our tertiary referral center (2019-2023) with a complete data set, who either received MPS simultaneously with intravenous methylprednisolone (IVMP), or after a first course of IVMP.

We evaluated the data of 172 patients. Ninety-five were eligible for analysis. Clinical Activity Score showed a significant decrease between baseline (BL) and primary endpoint 6 months (3.9 ± 0.9 vs. 2.4 ± 1.4, p < 0.0001). Inactivation was achieved in 60% of all patients at 6 months and in 77% at 12 months. Deviation, motility, upper eye lid retraction and proptosis showed no significant changes after 6 months. TSH-receptor-antibody-levels (TRAb) showed a significant decrease at 3 and 6 months (p < 0.0001). 10.5% developed DON. Multiple logistic regression showed a significant influence of irradiation after BL for inactivation (OR 6.18, 95% CI: 1.08 to 48.99).

While inactivation is most often achieved, the severity of the disease in form of fibrosis (lid retraction, motility) and proptosis is not reversed. Further rehabilitative surgery is needed and patients should still be closely monitored for DON. Other immunosuppressants could be more effective even in IVMP resistant GO and should be subject to randomized head-to-head trials.