Targeted biological therapies for Graves' disease and thyroid-associated ophthalmopathy. Focus on B-cell depletion

Laszlo Hegedüs1, Terry J Smith, Raymond S Douglas

  • 1Department of Endocrinology and Metabolism, Odense University Hospital, University of Southern Denmark, Denmark. laszlo.hegedus@ouh.regionsyddanmark.dk

Based on experience from the treatment of other autoimmune diseases and because of the limitations imposed by existing therapeutic options for Graves' disease (GD) and thyroid-associated ophthalmopathy (TAO), rituximab (RTX) was recently proposed as a novel therapy option. Here, we summarize the rationale for using RTX; give an overview of the possible mechanisms of action; and give an account of its effects and side-effects when used in GD and TAO. Scant evidence, originating from only a few methodologically inhomogeneous studies, suggests that RTX may prolong remission for hyperthyroidism over that seen with antithyroid drugs, at least in mild GD. Furthermore, in patients with TAO, who are unresponsive to conventional immunosuppressive therapy, RTX seems efficacious. As we wait for larger-scale randomized studies, RTX, should be considered experimental and reserved for patients who do not respond favourably to conventional therapy. It is the first in what is likely to be a series of new and emerging treatments specifically targeting relevant components of the immune system. Further studies will hopefully lead to improved and better tailored, individualized therapy for GD and especially TAO.

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