Academic Journal
Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial
| العنوان: | Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial |
|---|---|
| المؤلفون: | Groeneweg, S, Peeters, RP, Moran, C, Stoupa, MA, Auriol, F, Tonduti, D, Dica, A, Paone, L, Rozenkova, K, Malikova, J, van der Walt, A, de Coo, IFM, McGowan, A, Lyons, G, Aarsen, RFK, Barca, PD, van Beynu, IM, van der Knoop, MM, Jansen, J, Manshande, M, Lunsing, RJ, Nowak, S, den Uil, CA, Zillikens, MC, Visser, FE, Vrijoeth, P, de Wit, MCY, Wolf, NI, Zandstra, A, Abegaonkar, G, Singh, Y, de Rijke, YB, Medici, M, Bertini, ES, Depoorter, S, Lebl, J, Cappa, M, De Meirleir, L, Krude, H, Craiu, D, Zibordi, F, Petit, IO, Polak, M, Chatterjee, K, Visser, TJ, Visser, WE |
| المساهمون: | S. Groeneweg, R. Peeter, C. Moran, M. Stoupa, F. Auriol, D. Tonduti, A. Dica, L. Paone, K. Rozenkova, J. Malikova, A. van der Walt, I. de Coo, A. Mcgowan, G. Lyon, R. Aarsen, P. Barca, I. van Beynu, M. van der Knoop, J. Jansen, M. Manshande, R. Lunsing, S. Nowak, C. den Uil, M. Zilliken, F. Visser, P. Vrijoeth, M. de Wit, N. Wolf, A. Zandstra, G. Abegaonkar, Y. Singh, Y. de Rijke, M. Medici, E. Bertini, S. Depoorter, J. Lebl, M. Cappa, L. De Meirleir, H. Krude, D. Craiu, F. Zibordi, I. Petit, M. Polak, K. Chatterjee, T. Visser, W. Visser |
| بيانات النشر: | ELSEVIER SCIENCE INC |
| سنة النشر: | 2019 |
| المجموعة: | The University of Milan: Archivio Istituzionale della Ricerca (AIR) |
| مصطلحات موضوعية: | Settore MED/39 - Neuropsichiatria Infantile |
| الوصف: | Background Deficiency of the thyroid hormone transporter monocarboxylate transporter 8 (MCT8) causes severe intellectual and motor disability and high serum tri-iodothyronine (T-3) concentrations (Allan-Herndon-Dudley syndrome). This chronic thyrotoxicosis leads to progressive deterioration in bodyweight, tachycardia, and muscle wasting, predisposing affected individuals to substantial morbidity and mortality. Treatment that safely alleviates peripheral thyrotoxicosis and reverses cerebral hypothyroidism is not yet available. We aimed to investigate the effects of treatment with the T-3 analogue Triac (3,3',5-tri-iodothyroacetic acid, or tiratricol), in patients with MCT8 deficiency.Methods In this investigator-initiated, multicentre, open-label, single-arm, phase 2, pragmatic trial, we investigated the effectiveness and safety of oral Triac in male paediatric and adult patients with MCT8 deficiency in eight countries in Europe and one site in South Africa. Triac was administered in a predefined escalating dose schedule-after the initial dose of once-daily 350 mu g Triac, the daily dose was increased progressively in 350 mu g increments, with the goal of attaining serum total T-3 concentrations within the target range of 1.4-2.5 nmol/L We assessed changes in several dinical and biochemical signs of hyperthyroidism between baseline and 12 months of treatment. The prespecified primary endpoint was the change in serum T-3 concentrations from baseline to month 12. The co-primary endpoints were changes in concentrations of serum thyroid-stimulating hormone (TSH), free and total thyroxine (T-4), and total reverse T-3 from baseline to month 12. These analyses were done in patients who received at least one dose of Triac and had at least one post-baseline evaluation of serum throid function. This trial is registered with ClinicalTrials.gov, number NCT02060474.Findings Between Oct 15, 2014, and June 1, 2017, we screened 50 patients, all of whom were eligible. Of these patients, four (8%) patients decided not to ... |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| Relation: | info:eu-repo/semantics/altIdentifier/pmid/31377265; info:eu-repo/semantics/altIdentifier/wos/WOS:000482132700020; volume:7; issue:9; firstpage:695; lastpage:706; numberofpages:12; journal:THE LANCET DIABETES & ENDOCRINOLOGY; https://hdl.handle.net/2434/938856; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85070859264 |
| DOI: | 10.1016/S2213-8587(19)30155-X |
| الاتاحة: | https://hdl.handle.net/2434/938856 https://doi.org/10.1016/S2213-8587(19)30155-X |
| Rights: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.2AAEA643 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1016/S2213-8587(19)30155-X |
|---|