SFEIES24 Oral Communications Oral Communications (10 abstracts)
1Beacon Hospital, Dublin, Ireland; 2St. Vincents University Hospital, Dublin, Ireland; 3University College Dublin, Dublin, Ireland; 4Institute of Metabolic Science, University of Cambridge, Cambridge, United Kingdom; 5NIHR Cambridge Clinical Research Facility, Cambridge, United Kingdom; 6Clinical Biochemistry, Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom
Background: The treatment of Resistance to Thyroid Hormone beta (RTHbeta) is challenging because no therapy restores the euthyroid state in all tissues. Triac (triiodothyroacetic acid), a centrally-acting thyroid hormone analogue that preferentially activates thyroid hormone receptor beta, is reported to be beneficial in case reports or small case series.
Methods: We have treated a cohort of adult RTHbeta patients, all with heterozygous mutations in THRB and hyperthyroid symptoms, with Triac for up to a decade. Here, we describe the clinical, biochemical, metabolic and cardiac responses to therapy. Patients in whom the HPT axis was altered (due to ATDs, thyroid surgery or radioiodine) were excluded.
Results: A total of eight adult patients (aged 18-54 years, 4 female) were treated with Triac. Median Triac dose used was 2.4 mg per day (range 1.4-3.5 mg) and duration of therapy varied from <1 yr to 12 years. Response to therapy was analysed (Biochemistry, HSS: Hyperthyroid Symptoms Score, SHR: sleeping heart rate, REE: resting energy expenditure). 7 of 8 patients achieved normal circulating FT4 (measured by immunoassay; mean FT4 fell from 31.2 pmol/l to 18.3 pmol/l, RR 10.5-21) and 5 of 6 achieved normal total T3 concentrations (measured by LC-TMS; mean TT3 fell from 2.89 to 1.52 nmol/l, RR 1.09-2.24). Mean reductions in other parameters included; HSS reduction from 17/40 to 9/40, SHR reduction from 60 to 56bpm, REE (measured as Z score) fell from +1.375 to +0.66. Triac was well tolerated and there were no reported side effects. No patients discontinued therapy.
Conclusions: Triac therapy in RTHbeta reduces hyperthyroid symptoms, lowers circulating FT4 and TT3 concentrations and reduces basal metabolic and heart rate effectively, without adverse effects. Whether longer-term Triac treatment alters adverse cardiovascular outcomes recently associated with RTHbeta remains to be determined.