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Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
06:33

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis

Published on: June 9, 2018

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How did transthyretin amyloid cardiomyopathy progress in patients who took placebo in the study ATTR-ACT? A plain

Jose Nativi-Nicolau1, Daniel P Judge2, James E Hoffman3

  • 1Mayo Clinic, Jacksonville, FL, USA.

Future Cardiology
|March 17, 2022
PubMed

Insights

Tafamidis significantly reduced the risk of death or hospitalization for heart problems in transthyretin amyloid cardiomyopathy (ATTR-CM) patients. This study highlights the benefits of early diagnosis and treatment for this progressive heart condition.

Area of Science:

  • Cardiology
  • Rare Diseases
  • Pharmacology

Background:

  • Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart disease caused by abnormal protein buildup, leading to heart failure.
  • ATTR-CM includes wild-type (age-related) and hereditary (gene variant-related) forms.
  • Understanding ATTR-CM progression without treatment is crucial for evaluating therapeutic benefits.

Purpose of the Study:

  • To evaluate the efficacy of tafamidis in reducing mortality and hospitalizations in patients with ATTR-CM.
  • To assess the natural progression of ATTR-CM in patients not receiving active treatment.

Main Methods:

  • The ATTR-ACT study enrolled 441 patients with wild-type or hereditary ATTR-CM.
  • Participants received either tafamidis or a placebo orally once daily for 30 months.
  • Key endpoints included all-cause mortality, cardiovascular hospitalizations, and functional capacity.

Main Results:

  • Tafamidis treatment reduced the risk of death or cardiovascular hospitalization by approximately one-third compared to placebo.
  • In the placebo group, 40% of patients with wild-type ATTR-CM died, and 60% were hospitalized for heart problems over 30 months.
  • ATTR-CM progression in the placebo group led to worsened heart function and reduced walking distance.

Conclusions:

  • Tafamidis demonstrates significant benefits in managing ATTR-CM, reducing major adverse cardiovascular events.
  • The study underscores the progressive nature of ATTR-CM and the critical need for early diagnosis and intervention.
  • Results from the placebo arm provide valuable insights into the natural history of untreated ATTR-CM.
Abstract

Keywords:
amyloidosiscardiomyopathyheart failurelay summarynatural historyplain language summarytafamidistransthyretin

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