ESPEYB16 3. Thyroid Graves’ Disease (2 abstracts)
3.13. Adjuvant rituximab, a potential treatment for the young patient with Graves' hyperthyroidism (RiGD): study protocol for a single-arm, single-stage, phase II trial
Cole M , Hynes AM , Howel D , Hall L , Abinun M , Allahabadia A , Barrett T , Boelaert K , Drake AJ , Dimitri P , Kirk J , Zammitt N , Pearce S & Cheetham T
To read the full abstract: BMJ Open. 2019;9:e024705.
This protocol paper describes an innovative phase II trial to study the effect of a single low dose of adjuvant rituximab (a chimeric anti-B-cell monoclonal antibody targeting the surface molecule CD20) compared to classical carbimazole therapy in adolescents and young adults with Graves’ disease. Carbimazole treatment will be stopped after 12 months and the primary endpoint remission of Graves’ disease will be analysed at 24 months.
This paper describes in detail the aims, background and expected outcome of the adjuvant administration of a single low dose of rituximab at diagnosis. The immunmodulatory approach in Graves’ disease in children is convincing, based first on current knowledge on the efficacy and safety of rituximab in children with other autoimmune diseases, and second on its effects in adults with Graves’ disease with or without Graves’ orbitopathy.
The results of this proof of concept study will be important before planning phase III controlled randomized trials in larger cohorts. The following limitations are inherent to this study design: small patient number, inclusion of pubertal and post-pubertal patients with potentially different disease course than pre-pubertal children, and short follow-up period. Nevertheless, this combined treatment approach for Graves’ disease could result in a higher rate of remission in children and adolescents due to possible synergistic immunmodulatory effects of rituximab and carbimazole, potentially reduced cumulative carbimazole dose lowering the rate of side effects of carbimazole and, last but not least, fewer patients who will need total thyroidectomy or radioiodine ablation.
We look forward to the results of this first, and hopefully not the last, study investigating this promising new therapeutic approach for children with Graves’ disease.
Volume 16
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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