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Adjuvant Rituximab-Exploratory Trial in Young People With Graves Disease

Lookup NU author(s): Professor Timothy Cheetham, Mike Cole, Dr Mario Abinun, Dr Sonya Carnell, Jon Prichard, Gillian WatsonORCiD, Professor Sophie HambletonORCiD, Professor John MatthewsORCiD, Professor Simon PearceORCiD



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


© The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. CONTEXT: Remission rates in young people with Graves hyperthyroidism are less than 25% after 2 years of thionamide antithyroid drug (ATD). OBJECTIVE: We explored whether rituximab (RTX), a B-lymphocyte-depleting agent, would increase remission rates when administered with a short course of ATD. METHODS: This was an open-label, multicenter, single-arm, phase 2 trial in young people (ages, 12-20 years) with Graves hyperthyroidism. An A'Hern design was used to distinguish an encouraging remission rate (40%) from an unacceptable rate (20%). Participants presenting with Graves hyperthyroidism received 500 mg RTX and 12 months of ATD titrated according to thyroid function. ATDs were stopped after 12 months and primary outcome assessed at 24 months. Participants had relapsed at 24 months if thyrotropin was suppressed and free 3,5,3'-triiodothyronine was raised; they had received ATD between months 12 and 24; or they had thyroid surgery/radioiodine. RESULTS: A total of 27 participants were recruited and completed the trial with no serious side effects linked to treatment. Daily carbimazole dose at 12 months was less than 5 mg in 21 of 27 participants. Thirteen of 27 participants were in remission at 24 months (48%, 90% one-sided CI, 35%-100%); this exceeded the critical value (9) for the A'Hern design and provided evidence of a promising remission rate. B-lymphocyte count at 28 weeks, expressed as a percentage of baseline, was related to likelihood of remission. CONCLUSION: Adjuvant RTX, administered with a 12-month course of ATD, may increase the likelihood of remission in young people with Graves hyperthyroidism. A randomized trial of adjuvant RTX in young people with Graves hyperthyroidism is warranted.

Publication metadata

Author(s): Cheetham TD, Cole M, Abinun M, Allahabadia A, Barratt T, Davies JH, Dimitri P, Drake A, Mohamed Z, Murray RD, Steele CA, Zammitt N, Carnell S, Prichard J, Watson G, Hambleton S, Matthews JNS, Pearce SHS

Publication type: Article

Publication status: Published

Journal: Journal of Clinical Endocrinology and Metabolism

Year: 2022

Volume: 107

Issue: 3

Pages: 743-754

Print publication date: 01/03/2022

Online publication date: 23/10/2021

Acceptance date: 18/10/2021

Date deposited: 14/03/2022

ISSN (print): 0021-972X

ISSN (electronic): 1945-7197

Publisher: Oxford University Press


DOI: 10.1210/clinem/dgab763

PubMed id: 34687316


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