ESPE Yearbook of Paediatric Endocrinology

Nat Rev Endocrinol. 2024 Nov;20(11):647-660. doi: 10.1038/s41574-024-01016-5. PMID: 39039206

Brief Summary: This narrative review summarizes recent advances in the pathogenesis and treatment of Graves’ disease. Drawing on cellular, genetic, and immunological studies, it highlights the central role of autoreactive T and B cells, TSH receptor antibodies and,most importantly, immune tolerance failure. Novel therapeutic approaches targeting these pathways (including monoclonal antibodies and small molecules) are under investigation, not only in mouse models, but also in phase I clinical trials. The review outlines promising directions for individualized and immune-modulating therapies.

Currently, initial treatment of pediatric Graves’ disease is medical: anti-thyroid drug(s) (ATD). When this fails or is not possible, definitive treatment should be considered: radioactive iodine or total thyroidectomy [1]. Although longer ATD treatment probably increases the chance of permanent immunological and biochemical remission to 40-50%, more than half of pediatric patient require thyroid gland destruction or removal and, with that, lifelong thyroxine treatment. Novel therapeutic approaches to further improve the chance of permanent immunological remission are needed.

Reference: 1. Mooij CF, Cheetham TD, Verburg FA, Eckstein A, Pearce SH, Léger J, van Trotsenburg ASP. 2022 European Thyroid Association Guideline for the management of pediatric Graves’ disease. Eur Thyroid J. 2022 Jan 1;11(1):e210073. doi: 10.1530/ETJ-21-0073.21212. PMID: 34981748.