ESPE Yearbook of Paediatric Endocrinology

Endocr Rev. 2020;41:873–84. doi: 10.1210/endrev/bnaa022.

Over the last decades, the treatment of Graves’ disease has been limited to antithyroid drugs, radioactive iodine ablation or surgery. Especially in the paediatric age group, all three therapeutic options have their advantages and side effects.

New therapeutic options focus on the immune-pathophysiology of the disease by targeting either B-lymphocyte function or the TSH-receptor directly. Monoclonal antibodies may cause B-cell depletion, attenuated B-cell activation, inhibition of IgG recycling, or reduced B-cell proliferation and survival and all are under investigation in Phase 2 trials for treatment of Graves’ disease. TSH-receptor directed therapies aiming at directly inhibiting TSHR signalling (small molecule TSHR antagonist or TSHR blocking antibody) or TSHR specific immunotherapy are promising approaches under development in preclinical or Phase 1 trials.

Lane et al. provide a comprehensive overview of all new pharmacological developments for Graves’ disease treatment that promise completely new, innovative and hopefully efficient and safe treatment modalities not only for adults but also children and adolescents.