Therapy of ATTR Cardiac Amyloidosis: Current Indications

Daniela Di Lisi1, Vincenzo Di Stefano2, Filippo Brighina2

  • 1Division of Cardiology, University Hospital Paolo Giaccone, Palermo, Italy; Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (PROMISE) "G. D'Alessandro", University of Palermo, Palermo, Italy..

Insights

Early recognition and treatment of transthyretin cardiac amyloidosis (ATTR-CA) are crucial for improving prognosis. This review provides a practical guide to managing ATTR-CA, including new RNA-interfering drugs and TTR stabilizers.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Transthyretin cardiac amyloidosis (ATTR-CA) is a restrictive cardiomyopathy caused by transthyretin (TTR) amyloid fibril deposition.
  • ATTR-CA presents in hereditary (ATTRh) and acquired (ATTRwt) forms, with a poor prognosis if untreated.
  • Early diagnosis and prompt treatment are essential for modifying the disease course.

Purpose of the Study:

  • To provide a practical guide for the treatment of ATTR cardiac amyloidosis.
  • To highlight the importance of early recognition and multidisciplinary management.
  • To review current and emerging therapeutic options for ATTR-CA.

Main Methods:

  • Review of current literature on ATTR cardiac amyloidosis diagnosis and treatment.
  • Discussion of therapeutic strategies including TTR stabilizers and RNA-interfering drugs.
  • Emphasis on a multidisciplinary approach involving various medical specialists.

Main Results:

  • Tafamidis is recommended for hereditary and wild-type TTR cardiomyopathy (NYHA Class I/II) to reduce symptoms, hospitalizations, and mortality.
  • RNA-interfering drugs like patisiran are effective for ATTRh amyloidosis, particularly with polyneuropathy.
  • Early intervention with appropriate therapies can significantly alter the prognosis of ATTR-CA.

Conclusions:

  • ATTR cardiac amyloidosis requires prompt diagnosis and a multidisciplinary treatment strategy.
  • Tafamidis and patisiran represent significant advancements in managing ATTR-CA.
  • This review serves as a practical resource for clinicians managing patients with ATTR cardiac amyloidosis.

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