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Tafamidis in Hereditary ATTR-CM — EECC MCQ

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ModerateCardiomyopathyTafamidis in Hereditary ATTR-CMEECC

A 38-year-old woman with known hereditary ATTR amyloidosis (Val30Met mutation) and early cardiac involvement (septal thickness 13 mm, LVEF 58%, mildly elevated NT-proBNP) is started on tafamidis. What is the mechanism and evidence for this treatment?

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Correct answer: D — Tafamidis stabilises the transthyretin tetramer, preventing dissociation into amyloidogenic monomers; the ATTR-ACT trial showed reduction in mortality and cardiovascular hospitalisations

Tafamidis is a TTR stabiliser that binds to the thyroxine-binding sites on the TTR tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils. The ATTR-ACT trial demonstrated that tafamidis 80 mg (or tafamidis meglumine 20 mg) reduced all-cause mortality by 30% and cardiovascular hospitalisations by 32% in ATTR cardiac amyloidosis (both wild-type and hereditary). The ESC 2023 Cardiomyopathy Guidelines recommend tafamidis for ATTR-CM (Class I, LOE B). It is most effective when initiated early in the disease course (NYHA I-II), before significant myocardial damage. Tafamidis does not remove existing amyloid deposits; TTR gene silencers (patisiran, inotersen) reduce TTR production and are used primarily in hereditary ATTR with polyneuropathy.

Reference: ESC (2023): Guidelines on Cardiomyopathies; ATTR-ACT Trial