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.
Abstract:
Transthyretin cardiac amyloidosis is a restrictive cardiomyopathy caused by extracellular deposition in the heart of amyloid fibrils derived from plasma transthyretin (ATTR), either in its hereditary (ATTRh) or acquired (ATTRwt) forms. Cardiac amyloidosis has a very poor prognosis if therapy is not started promptly. Therefore, it is very important to recognize cardiac amyloidosis early in order to immediately start a treatment capable of modifying the prognosis. Treatment of cardiac amyloidosis is not easy, often requiring a multidisciplinary team. New RNA-interfering drugs (such as patisiran) have been devised and are effective in the treatment of ATTRh amyloidosis. Tafamidis (a stabilizer of the native tetramer structure of TTR) is recommended to treat patients with genetic testing-proven hereditary hTTR-cardiomyopathy or wild-type TTR cardiomyopathy and NYHA Class I or II to reduce symptoms, CV hospitalization and mortality (Class I, level of evidence B). Patisiran should be considered in ATTRh cardiomyopathy with polyneuropathy. Thus, this review is intended to be a simple practical guide for the treatment of ATTR cardiac amyloidosis.
More Related Videos
Related Concept Videos
Cardiomyopathy V: Interprofessional Care
Myocarditis III: Medical Management
Cardiomyopathy II: Dilated Cardiomyopathy
Cardiomyopathy IV: Restrictive Cardiomyopathy
Heart Failure VI: Adjunct Therapies
Cardiomyopathy III: Hypertrophic Cardiomyopathy


