Rapamycin for treatment of refractory dysthyroid compressive optic neuropathy.

We report a patient with dysthyroid optic neuropathy refractory to steroids and orbital decompression treated with rapamycin, a fibroblast and T cell inhibitor. Symptoms, visual acuity, color plate testing, and visual fields improved. Aside from hypercholesterolemia, no complication related to this therapy was observed. By addressing the pathogenesis of thyroid eye disease, rapamycin may represent an alternative when standard treatments fail. Further investigation of rapamycin for treatment of dysthyroid orbitopathy is warranted.