Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study.
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van Geest Ferdy S, Groeneweg Stefan, van den Akker Erica L T, Bacos Iuliu, Barca Diana, van den Berg Sjoerd A A, Bertini Enrico, Brunner Doris, Brunetti-Pierri Nicola, Cappa Marco, Cappuccio Gerarda, Chatterjee Krishna, Chesover Alexander D, Christian Peter, Coutant Régis, Craiu Dana, Crock Patricia, Dewey Cheyenne, Dica Alice, Dimitri Paul, Dubey Rachana, Enderli Anina, Fairchild Jan, Gallichan Jonathan, Garibaldi Luigi R, George Belinda, Hackenberg Annette, Heinrich Bianka, Huynh Tony, Kłosowska Anna, Lawson-Yuen Amy, Linder-Lucht Michaela, Lyons Greta, Monti Lora Felipe, Moran Carla, Müller Katalin E, Paone Laura, Paul Praveen G, Polak Michel, Porta Francesco, Reinauer Christina, de Rijke Yolanda B, Seckold Rowen, Menevşe Tuba Seven, Simm Peter, Simon Anna, Spada Marco, Stoupa Athanasia, Szeifert Lilla, Tonduti Davide, van Toor Hans, Turan Serap, Vanderniet Joel, de Waart Monique, van der Wal Ronald, van der Walt Adri, van Wermeskerken Anne-Marie, Wierzba Jolanta, Zibordi Federica, Zung Amnon, Peeters Robin P, Visser W Edward
Аннотация
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 parameters, heart rate, and biochemical markers of thyroid hormone action.
RESULTS: 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 < 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) µmol/L (mean increase 7 µmol/L; 95% CI, 6-9; P < 0.0001). Mean creatine kinase concentrations did not significantly change. No drug-related severe adverse events were reported.
CONCLUSIONS: Key features were sustainably alleviated in patients with MCT8 deficiency across all ages, highlighting the real-life potential of Triac for MCT8 deficiency.