Diiodothyropropionic acid (DITPA) in the treatment of MCT8 deficiency

Journal of Clinical Endocrinology and Metabolism

Volumn 97, Issue 12, 2012, Pages 4515-4523

  Verge, Charles F ^a,b   Konrad, Daniel ^c   Cohen, Michal ^d   Di Cosmo, Caterina ^e   Dumitrescu, Alexandra M ^e   Marcinkowski, Teresa ^e   Hameed, Shihab ^a,b   Hamilton, Jill ^d   Weiss, Roy E ^e   Refetoff, Samuel ^e  

^a SYDNEY CHILDREN'S HOSPITAL   (Australia)

^b UNIVERSITY OF NEW SOUTH WALES   (Australia)

^c UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e UNIVERSITY OF CHICAGO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DIIODOTHYROPROPIONIC ACID; FERRITIN; LIOTHYRONINE; MONOCARBOXYLATE TRANSPORTER 8; SEX HORMONE BINDING GLOBULIN; THYROMIMETIC AGENT; THYROTROPIN; THYROXINE; UNCLASSIFIED DRUG;

ARTICLE; CHILD; CLINICAL ARTICLE; COMPASSIONATE USE; DRUG BLOOD LEVEL; DRUG DOSE INCREASE; DRUG EFFICACY; FREE THYROXINE INDEX; GASTROINTESTINAL SYMPTOM; GENE MUTATION; HEART RATE; HUMAN; HYPERMETABOLISM; INFANT; LIOTHYRONINE BLOOD LEVEL; MALE; MCT8 GENE; MONOCARBOXYLATE TRANSPORTER 8 DEFICIENCY; MUSCLE HYPOTONIA; PRESCHOOL CHILD; PRIORITY JOURNAL; PSYCHOMOTOR DEVELOPMENT; SIDE EFFECT; THYROID DISEASE; THYROTROPIN BLOOD LEVEL; WEIGHT REDUCTION;

BRAIN; CARDIOTONIC AGENTS; CHILD DEVELOPMENT; CHILD, PRESCHOOL; COMPASSIONATE USE TRIALS; DIIODOTHYRONINES; DISEASES IN TWINS; HUMANS; INFANT; MALE; MENTAL RETARDATION, X-LINKED; MONOCARBOXYLIC ACID TRANSPORTERS; MULTICENTER STUDIES AS TOPIC; MUSCLE HYPOTONIA; MUSCULAR ATROPHY; PROPIONATES; PSYCHOMOTOR PERFORMANCE; THYROID FUNCTION TESTS;

MUS;

EID: 84870728589     PISSN: 0021972X     EISSN: 19457197     Source Type: Journal    
DOI: 10.1210/jc.2012-2556     Document Type: Article

References (23)

1. Dumitrescu AM, Liao XH, Best TB, Brockmann K, Refetoff S 2004 A novel syndrome combining thyroid and neurological abnormalities is associated with mutations in a monocarboxylate transporter gene. Am J Hum Genet 74:168-175 (Pubitemid 38085248)
2. Friesema EC, Grueters A, Biebermann H, Krude H, von Moers A, Reeser M, Barrett TG, Mancilla EE, Svensson J, Kester MH, Kuiper GG, Balkassmi S, Uitterlinden AG, Koehrle J, Rodien P, Halestrap AP, Visser TJ 2004 Association between mutations in a thyroid hormone transporter and severe X-linked psychomotor retardation. Lancet 364:1435-1437 (Pubitemid 39370719)
3. Friesema EC, Ganguly S, Abdalla A, Manning Fox JE, Halestrap AP, Visser TJ 2003 Identification of monocarboxylate transporter 8 as a specific thyroid hormone transporter. J Biol Chem 278:40128-40135 (Pubitemid 37248580)
4. Schwartz CE, May MM, Carpenter NJ, Rogers RC, Martin J, Bialer MG, Ward J, Sanabria J, Marsa S, Lewis JA, Echeverri R, Lubs HA, Voeller K, Simensen RJ, Stevenson RE 2005 Allan-Herndon-Dudley syndrome and the monocarboxylate transporter 8 (MCT8) gene. Am J Hum Genet 77:41-53 (Pubitemid 40848035)
5. Allan W, Herndon CN, Dudley FC 1944 Some examples of the inheritance of mental deficiency: apparently sex-linked idiocy and microcephaly. Am J Ment Defic 48:325-334
6. Schwartz CE, Stevenson RE 2007 The MCT8 thyroid hormone transporter and Allan-Herndon-Dudley syndrome. Best Pract Res Clin Endocrinol Metab 21:307-321 (Pubitemid 46888197)
7. Visser WE, Friesema EC, Visser TJ 2011 Minireview: thyroid hormone transporters: the knowns and the unknowns. Mol Endocrinol 25:1-14
8. Heuer H, Visser TJ 2009 Minireview: pathophysiological importance of thyroid hormone transporters. Endocrinology 150:1078-1083
9. Dumitrescu AM, Liao XH, Weiss RE, Millen K, Refetoff S 2006 Tissue specific thyroid hormone deprivation and excess in Mct8 deficient mice. Endocrinology 147:4036-4043 (Pubitemid 44268357)
10. Trajkovic M, Visser TJ, Mittag J, Horn S, Lukas J, Darras VM, Raivich G, Bauer K, Heuer H 2007 Abnormal thyroid hormone metabolism in mice lacking the monocarboxylate transporter 8. J Clin Invest 117:627-635 (Pubitemid 46348521)
11. Di Cosmo C, Liao XH, Dumitrescu AM, Weiss RE, Refetoff S 2009 A thyroid hormone analogue with reduced dependence on the monocarboxylate transporter 8 (MCT8) for tissue transport. Endocrinology 150:4450-4458
12. Morkin E, Pennock G, Spooner PH, Bahl JJ, Underhill Fox K, Goldman S 2002 Pilot studies on the use of 3,5-diiodothyropropionic acid, a thyroid hormone analog, in the treatment of congestive heart failure. Cardiology 97:218-225
13. Goldman S, McCarren M, Morkin E, Ladenson PW, Edson R, Warren S, Ohm J, Thai H, Churby L, Barnhill J, O'Brien T, Anand I, Warner A, Hattler B, Dunlap M, Erikson J, Shih MC, Lavori P 2009 DITPA (3,5-diiodothyropropionic acid), a thyroid hormone analog to treat heart failure. Phase II Trial Veterans Affairs Cooperative Study. Circulation 119:3093-3100
14. Ladenson PW, McCarren M, Morkin E, Edson RG, Shih MC, Warren SR, Barnhill JG, Churby L, Thai H, O'Brien T, Anand I, Warner A, Hattler B, Dunlap M, Erikson J, Goldman S 2010 Effects of the thyromimetic agent diiodothyropropionic acid on body weight, body mass index, and serum lipoproteins: a pilot prospective, randomized, controlled study. J Clin Endocrinol Metab 95:1349-1354
15. Wémeau JL, Pigeyre M, Proust-Lemoine E, d'Herbomez M, Gottrand F, Jansen J, Visser TJ, Ladsous M 2008 Beneficial effects of propylthiouracil plus L-thyroxine treatment in a patient with a mutation in MCT8. J Clin Endocrinol Metab 93:2084-2088 (Pubitemid 351831519)
16. Chapo J, Peng Y, Pitts KR 2007 A phosphorimager-based filter binding thyroid hormone receptor competition assay for chemical screening. J Pharmacol Toxicol Methods 56:28-33 (Pubitemid 46829928)
17. Pennock GD, Raya TE, Bahl JJ, Goldman S, Morkin E 1992 Cardiac effects of 3,5-diiodothyropropionic acid, a thyroid hormone analog with inotropic selectivity. J Pharmacol Exp Ther 263:163-169
18. Mousa SA, O'Connor L, Davis FB, Davis PJ 2006 Proangiogenesis action of the thyroid hormone analog 3,5-diiodothyropropionic acid (DITPA) is initiated at the cell surface and is integrin mediated. Endocrinology 147:1602-1607
19. Mizuno H, Sugiyama Y, Nishi Y, Ueda N, Ohro Y, Togari H 2006 Elevation of serum creatine kinase in response to medical treatment of Graves' disease in children. Acta Paediatr 95:243-245 (Pubitemid 43189021)
20. Di Cosmo C, Liao XH, Dumitrescu AM, Philp NJ, Weiss RE, Refetoff S 2010 Mice deficient in MCT8 reveal a mechanism regulating thyroid hormone secretion. J Clin Invest 120:3377-3388
21. Trajkovic-Arsic M, Visser TJ, Darras VM, Friesema EC, Schlott B, Mittag J, Bauer K, Heuer H 2010 Consequences of monocarboxylate transporter 8 deficiency for renal transport and metabolism of thyroid hormones in mice. Endocrinology 151:802-809
22. Hernandez A, Quignodon L, Martinez ME, Flamant F, St. Germain DL 2010 Type 3 deiodinase deficiency causes spatial and temporal alterations in brain T3 signaling that are dissociated from serum thyroid hormone levels. Endocrinology 151:5550-5558
23. Hernandez A, Morte B, Belinchón MM, Ceballos A, Bernal J 2012 Critical role of types 2 and 3 deiodinases in the negative regulation of gene expression by T(3) in the mouse cerebral cortex. Endocrinology 153:2919-2928