Long-term outcome of Graves' orbitopathy following treatment with sirolimus.

Abstract

Sirolimus was found to be associated with a better outcome of Graves' orbitopathy (GO) at 24weeks compared to methylprednisolone. We conducted a retrospective study to investigate its efficacy and safety over a longer period. Data from 40 consecutive patients with moderate-to-severe, active GO, 20 treated with sirolimus and 20 with methylprednisolone, were collected. overall outcome (composite evaluation) of GO at 48weeks. (1) GO outcome at 24weeks, and, at 24 and 48weeks: (2) outcome of single eye features; (3) quality of life (GO-QoL); (4) TSH-receptor antibodies; (5) GO relapse at 48weeks; (6) adverse events. The overall GO outcome at 48weeks did not differ between the two groups (responders: 55% vs 55%). At 24weeks, prevalence of responders was greater in sirolimus group (65% vs 25%; P = 0.01). A reduction ≥ 1 point in clinical activity score (CAS) was more frequent in sirolimus patients at 24 (85% vs 40%; P = 0.005) and 48weeks (75% vs 60%; P = 0.03). The proportion of GO-QoL responders (appearance subscale) at 24weeks was greater in sirolimus group (62.5% vs 26.3%; P = 0.03). No difference was observed for the remaining outcome measures. Treatment with sirolimus is followed by a greater overall response of GO compared with methylprednisolone at 24weeks, but not at 48weeks, when only CAS is affected. A more prolonged period of treatment may be required for a better outcome to be observed over a longer period.