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Emcitate (tiratricol)

MCT8 deficiency

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Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Thyroid hormone analogues

3 evidence topics · 3 sources

Guideline recommendations for thyroid hormone analogues

“We recommend treatment with TRIAC (Recommendation: S, Quality of evidence: ØØØO) or DITPA (Recommendation: W, Quality of evidence: ØØOO).” (opens the source at this quote in a new tab)

“We recommend titrating the dosage of TRIAC (or DITPA), aiming to reduce serum T3 concentrations to a target range between 1.4 and 2.5 nmol/L to alleviate the peripheral thyrotoxic state, unless limited by the occurrence of dose-related side effects” (opens the source at this quote in a new tab)

“We recommend long-term therapy with TRIAC (or DITPA) in all patients unless concerns about drug side effects or tolerability arise” (opens the source at this quote in a new tab)

Evidence base for thyroid hormone analogues

“Knowledge of response to thyroxine (alone, or in combination with PTU) and DITPA is limited to case reports and case series, whereas the effects of TRIAC have been studied in a phase 2 clinical trial and a prospective cohort study.” (opens the source at this quote in a new tab)

“Treatment with DITPA (dose range: 2.1–2.5 mg/kg/day) normalized serum T3 and TSH without reduction of T4 concentrations (4/4 cases) and had mixed effects on peripheral symptoms, including a reduction in heart rate (3/4 cases) and improvement in body weight (2/4 cases). Commencement of treatment between 8.5 and 25 months did not improve neurocognitive development” (opens the source at this quote in a new tab)

Mechanism and relative evidence for TRIAC and DITPA

“TRIAC and DITPA are TH analogues that bypass MCT8-dependent transport and activate thyroid hormone receptors (THRs). TRIAC has a high affinity for THR β, reduces serum T3 levels, and ameliorates peripheral thyrotoxic manifestations, with the strongest clinical evidence supporting its use. DITPA similarly lowers serum T3 levels but is supported by less consistent clinical data.” (opens the source at this quote in a new tab)