Journal article
Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study.
Ferdy S van Geest, Stefan Groeneweg, Erica LT van den Akker, Iuliu Bacos, Diana Barca, Sjoerd AA van den Berg, Enrico Bertini, Doris Brunner, Nicola Brunetti-Pierri, Marco Cappa, Gerarda Cappuccio, Krishna Chatterjee, Alexander D Chesover, Peter Christian, Régis Coutant, Dana Craiu, Patricia Crock, Cheyenne Dewey, Alice Dica, Paul Dimitri Show all
Journal of Clinical Endocrinology and Metabolism | Oxford University Press | Published : 2022
Abstract
CONTEXT: Patients with mutations in thyroid hormone transporter MCT8 have developmental delay and chronic thyrotoxicosis associated with being underweight and having cardiovascular dysfunction. OBJECTIVE: Our previous trial showed improvement of key clinical and biochemical features during 1-year treatment with the T3 analogue Triac, but long-term follow-up data are needed. METHODS: In this real-life retrospective cohort study, we investigated the efficacy of Triac in MCT8-deficient patients in 33 sites. The primary endpoint was change in serum T3 concentrations from baseline to last available measurement. Secondary endpoints were changes in other thyroid parameters, anthropometric parameter..
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Awarded by Wellcome Trust
Funding Acknowledgements
This work was supported by a grant from Eurostars (project number ESTAR E11337) (to W.E.V.) and from the Sherman Foundation (to W.E.V.).