Table 2.2. Recommendations for specific treatment for cardiac transthyretin amyloidosis (ATTR-CA).

Recommendation	Class	LE	Comment	Table 2018	Ref.
Tafamidis 80 mg/day for treatment of patients with cardiac transthyretin amyloidosis patients in order to reduce mortality and cardiovascular hospitalizations.	I	B	NEW: A multicenter randomized clinical trial supports the recommendation.	New	28
Several steps of amiloid fibers formation constitute therapeutic targets in transthyretin amyloidosis (ATTR). The first disease-modifying therapy to show any evidence of benefit in patients with amyloid cardiomyopathy is tafamidis, a TTR tetramer stabilizer. Tafamidis was tested in a multicenter, placebo-controlled, randomized trial involving 441 patients with cardiac amyloidosis patients, of which 264 were assigned to receive tafamidis at doses of 20 mg or 80 mg daily (ATTR-ACT [Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy] study).28 The primary results showed that the use of tafamidis was associated with a 30% reduction in all-cause mortality (RR = 0.70 [95% CI: 0.51-0.96]) and 32% reduction of cardiovascular-related hospitalizations (RR = 0.68 [95% CI: 0.56 −0.81]), in addition to reduced worsening of functional capacity and quality of life. Based on these results, tafamidis was approved by ANVISA in Brazil for treatment of CA-ATTR, at a dose of 80 mg / day.28

ATTR: transthyretin amyloidosis; RR: relative risk