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Antigen-specific immunotherapy with thyrotropin receptor peptides in Graves' hyperthyroidism: a phase I study

Pearce, Simon H.S., Dayan, Colin ORCID:, Wraith, David C., Barrell, Kevin, Olive, Natalie, Jansson, Lotta, Walker-Smith, Terrie, Carnegie, Christina, Martin, Keith F., Boelaert, Kristien, Gilbert, Jackie, Higham, Claire E., Muller, Ilaria ORCID:, Murray, Robert D., Perros, Petros, Razvi, Salman, Vaidya, Bijay, Wernig, Florian and Kahaly, George J. 2019. Antigen-specific immunotherapy with thyrotropin receptor peptides in Graves' hyperthyroidism: a phase I study. Thyroid 29 (7) , pp. 1003-1011. 10.1089/thy.2019.0036

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Background: Graves' disease is one of the most common autoimmune conditions, but treatment remains imperfect. This study explores the first-in-human use of antigen-specific immunotherapy with a combination of two thyrotropin receptor (TSHR) peptides (termed ATX-GD-59) in Graves' hyperthyroidism. Methods: Twelve participants (11 female) with previously untreated mild to moderate Graves' hyperthyroidism were enrolled in a Phase I open label trial to receive 10 doses of ATX-GD-59 administered intradermally over an 18-week period. Adverse events, tolerability, changes in serum free thyroid hormones, and TSHR autoantibodies were measured. Results: Ten subjects received all 10 doses of ATX-GD-59, five (50%) of whom had free triiodothyronine within the reference interval by the 18-week visit. Two further subjects had improved free thyroid hormones by the end of the study (7/10 responders), whereas three subjects showed worsening thyrotoxicosis during the study. Serum TSHR autoantibody concentrations reduced during the study and correlated with changes in free thyroid hormones (r = 0.85, p = 0.002 for TSHR autoantibody vs. free triiodothyronine). Mild injection-site swelling and pain were the most common adverse events. Conclusions: These preliminary data suggest that ATX-GD-59 is a safe and well-tolerated treatment. The improvement in free thyroid hormones in 70% of subjects receiving the medication suggests potential efficacy as a novel treatment for Graves' hyperthyroidism.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Additional Information: This Open Access article is distributed under the terms of the Creative Commons License
Publisher: Mary Ann Liebert
ISSN: 1050-7256
Date of First Compliant Deposit: 29 July 2019
Last Modified: 04 May 2023 16:39

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