Skill Checkup: A 68-Year-Old Man With Acute Decompensated Heart Failure, Worsening Dyspnoea and Lower Extremity Oedema

Marco Luigetti, MD, PhD


August 18, 2022

The only approved agent for ATTR-CM (either hereditary or wild-type) is tafamidis. Its approval was based on a randomised controlled trial, the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT). The ATTR-ACT study enrolled 441 patients with ATTR amyloidosis (either hereditary or wild-type).

In patients with ATTR-CM, tafamidis resulted in a 13.4% absolute reduction in all-cause mortality and a 32% absolute reduction in yearly cardiovascular hospitalization at 30 months compared with placebo. Tafamidis is also associated with improvements in HF-related quality of life and functional status in some patients.

Diflunisal, an NSAID, is also a transthyretin stabiliser but is not approved for ATTR; it is often given off-label. In general, NSAID therapy is associated with a risk for gastrointestinal, renal and cardiac side effects and is contraindicated in patients with renal failure or HF. Therefore, the use of diflunisal may be limited in hATTR patients who have severe hATTR-CM.


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