Farmaci biologici per l’orbitopatia basedowiana: recenti sviluppi tra realtà e speranze

Graves’ orbitopathy is the most common extrathyroidal manifestation/complication of Graves’ disease. High-dose intravenous glucocorticoids have been used for decades for the treatment of moderate to severe and active orbitopathy, with positive results on inflammatory signs and symptoms, and recent onset diplopia. However, their efficacy may be transient and is low on exophthalmos and diplopia. Nonsteroidal immunosuppressive agents may offer additional benefits, although often rehabilitative surgery is eventually required. Biological agents targeting key disease pathogenetic steps represent promising options. This review covers new treatments, including teprotumumab, an IGF-1R antagonist, approved in the U.S. for Graves’ orbitopathy, and rituximab, tocilizumab, and belimumab, in phase 3 studies. Novel molecules in trials include TSH receptor inhibitors, anti-VEGF therapy, additional IGF-1R antagonists, and modified TSH receptor peptides. However, results of studies are preliminary or not available yet.