{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T20:45:26Z","timestamp":1792010726092,"version":"4.4.0"},"reference-count":18,"publisher":"The Endocrine Society","issue":"3","license":[{"start":{"date-parts":[[2021,10,22]],"date-time":"2021-10-22T00:00:00Z","timestamp":1634860800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013297","name":"Eurostars","doi-asserted-by":"publisher","award":["ESTAR E11337"],"award-info":[{"award-number":["ESTAR E11337"]}],"id":[{"id":"10.13039\/100013297","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Sherman Foundation"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2022,2,17]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Context<\/jats:title><jats:p>Patients with mutations in thyroid hormone transporter MCT8 have developmental delay and chronic thyrotoxicosis associated with being underweight and having cardiovascular dysfunction.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>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 parameters, heart rate, and biochemical markers of thyroid hormone action.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>From October 15, 2014 to January 1, 2021, 67 patients (median baseline age 4.6 years; range, 0.5-66) were treated up to 6 years (median 2.2 years; range, 0.2-6.2). Mean T3 concentrations decreased from 4.58 (SD 1.11) to 1.66 (0.69) nmol\/L (mean decrease 2.92 nmol\/L; 95% CI, 2.61-3.23; P &amp;lt; 0.0001; target 1.4-2.5 nmol\/L). Body-weight-for-age exceeded that of untreated historical controls (mean difference 0.72 SD; 95% CI, 0.36-1.09; P = 0.0002). Heart-rate-for-age decreased (mean difference 0.64 SD; 95% CI, 0.29-0.98; P = 0.0005). SHBG concentrations decreased from 245 (99) to 209 (92) nmol\/L (mean decrease 36 nmol\/L; 95% CI, 16-57; P = 0.0008). Mean creatinine concentrations increased from 32 (11) to 39 (13) \u00b5mol\/L (mean increase 7 \u00b5mol\/L; 95% CI, 6-9; P &amp;lt; 0.0001). Mean creatine kinase concentrations did not significantly change. No drug-related severe adverse events were reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Key features were sustainably alleviated in patients with MCT8 deficiency across all ages, highlighting the real-life potential of Triac for MCT8 deficiency.<\/jats:p><\/jats:sec>","DOI":"10.1210\/clinem\/dgab750","type":"journal-article","created":{"date-parts":[[2021,10,22]],"date-time":"2021-10-22T20:41:49Z","timestamp":1634935309000},"page":"e1136-e1147","source":"Crossref","is-referenced-by-count":43,"title":["Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study"],"prefix":"10.1210","volume":"107","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6960-5775","authenticated-orcid":false,"given":"Ferdy S","family":"van Geest","sequence":"first","affiliation":[{"name":"Academic Center for Thyroid Diseases, Department of Internal Medicine, Erasmus Medical Center, 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6879-7392","authenticated-orcid":false,"given":"Stefan","family":"Groeneweg","sequence":"additional","affiliation":[{"name":"Academic Center for Thyroid Diseases, Department of Internal Medicine, Erasmus Medical Center, 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5352-9328","authenticated-orcid":false,"given":"Erica L T","family":"van den Akker","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Department of Pediatrics, Erasmus MC-Sophia Children\u2019s Hospital, University Medical Center, 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Iuliu","family":"Bacos","sequence":"additional","affiliation":[{"name":"Centrul Medical Dr. Bacos Cosma, Timisoara 307200, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"Barca","sequence":"additional","affiliation":[{"name":"Carol Davila University of Medicine, Department of Clinical Neurosciences, Paediatric Neurology Discipline II, Bucharest 050474, Romania"},{"name":"Paediatric Neurology Clinic, Reference Center for Rare Paediatric Neurological Disorders, ENDO-ERN member, Alexandru Obregia Hospital, Bucharest 041914, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sjoerd A A","family":"van den Berg","sequence":"additional","affiliation":[{"name":"Diagnostic Laboratory for Endocrinology, Department of Internal Medicine, Erasmus Medical Center , 3015 GD Rotterdam, The Netherlands"},{"name":"Department of Clinical chemistry, Erasmus Medical Center, 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9276-4590","authenticated-orcid":false,"given":"Enrico","family":"Bertini","sequence":"additional","affiliation":[{"name":"Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesu\u2019 Children\u2019s Research Hospital IRCCS, 00165 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doris","family":"Brunner","sequence":"additional","affiliation":[{"name":"Gottfried Preyer\u2019s Children Hospital, 1100 Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6895-8819","authenticated-orcid":false,"given":"Nicola","family":"Brunetti-Pierri","sequence":"additional","affiliation":[{"name":"Department of Translational Medicine, Federico II University, 80131 Naples, Italy"},{"name":"Telethon Institute of Genetics and Medicine, Pozzuoli, 80078 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Cappa","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Bambino Gesu\u2019 Children\u2019s Research Hospital IRCCS, 00165 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerarda","family":"Cappuccio","sequence":"additional","affiliation":[{"name":"Department of Translational Medicine, Federico II University, 80131 Naples, Italy"},{"name":"Telethon Institute of Genetics and Medicine, Pozzuoli, 80078 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Krishna","family":"Chatterjee","sequence":"additional","affiliation":[{"name":"Wellcome Trust-Medical Research Council Institute of Metabolic Science, University of Cambridge, Cambridge CB2 0QQ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander D","family":"Chesover","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, The Hospital for Sick Children and Department of Paediatrics, University of Toronto, Toronto, M5G 1X8, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Christian","sequence":"additional","affiliation":[{"name":"East Kent Hospitals University NHS Foundation Trust, Ashford TN24 0LZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R\u00e9gis","family":"Coutant","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology and Diabetology, University Hospital, 49100 Angers, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dana","family":"Craiu","sequence":"additional","affiliation":[{"name":"Carol Davila University of Medicine, Department of Clinical Neurosciences, Paediatric Neurology Discipline II, Bucharest 050474, Romania"},{"name":"Paediatric Neurology Clinic, Reference Center for Rare Paediatric Neurological Disorders, ENDO-ERN member, Alexandru Obregia Hospital, Bucharest 041914, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia","family":"Crock","sequence":"additional","affiliation":[{"name":"John Hunter Children\u2019s Hospital, New Lambton Heights, NSW 2305, Australia"},{"name":"Hunter Medical Research Institute, University of Newcastle Kookaburra Circuit, New Lambton Heights, NSW 2305, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cheyenne","family":"Dewey","sequence":"additional","affiliation":[{"name":"Genomics Institute Mary Bridge Children\u2019s Hospital, MultiCare Health System Tacoma, WA 98403, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Dica","sequence":"additional","affiliation":[{"name":"Carol Davila University of Medicine, Department of Clinical Neurosciences, Paediatric Neurology Discipline II, Bucharest 050474, Romania"},{"name":"Paediatric Neurology Clinic, Reference Center for Rare Paediatric Neurological Disorders, ENDO-ERN member, Alexandru Obregia Hospital, Bucharest 041914, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7625-6713","authenticated-orcid":false,"given":"Paul","family":"Dimitri","sequence":"additional","affiliation":[{"name":"Sheffield Children\u2019s NHS Foundation Trust, Sheffield Hallam University and University of Sheffield, Sheffield, S10 2TH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachana","family":"Dubey","sequence":"additional","affiliation":[{"name":"Medanta Superspeciality Hospital, Indore 800020, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anina","family":"Enderli","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics, University Children\u2019s Hospital Zurich, Steinwiesstrasse 75, 8032 Z\u00fcrich, Switzerland"},{"name":"Neurology Department, Children\u2019s Hospital, St. Gallen, 9000, 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Torino, University of Torino, Torino 10126,Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Reinauer","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children\u2019s Hospital, Medical Faculty, Duesseldorf 40225, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yolanda B","family":"de Rijke","sequence":"additional","affiliation":[{"name":"Department of Clinical chemistry, Erasmus Medical Center, 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rowen","family":"Seckold","sequence":"additional","affiliation":[{"name":"John Hunter Children\u2019s Hospital, New Lambton Heights, NSW 2305, Australia"},{"name":"Hunter Medical Research Institute, University of Newcastle Kookaburra Circuit, New Lambton Heights, NSW 2305, 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(Center for Diagnosis and Treatment of Leukodystrophies), V. Buzzi Children\u2019s Hospital, Milano 20154, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hans","family":"van Toor","sequence":"additional","affiliation":[{"name":"Diagnostic Laboratory for Endocrinology, Department of Internal Medicine, Erasmus Medical Center , 3015 GD Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5172-5402","authenticated-orcid":false,"given":"Serap","family":"Turan","sequence":"additional","affiliation":[{"name":"Marmara University School of Medicine Department of Pediatric Endocrinology, Istanbul 34854, Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Vanderniet","sequence":"additional","affiliation":[{"name":"John Hunter Children\u2019s Hospital, New Lambton Heights, NSW 2305, Australia"},{"name":"Hunter Medical Research Institute, University of Newcastle Kookaburra Circuit, New Lambton Heights, NSW 2305, 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