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Brain & Heart

REVIEW ARTICLE
Unlocking the potential of tafamidis in treating

transthyretin cardiac amyloidosis: A systematic
review

1
Heet N. Desai * , Riti Sanghvi , and Sarthak H. Dhruv 3
2
1 Department of Internal Medicine, California Institute of Behavioral Neurosciences and Psychology,
United States of America
2 Department of Medicine, Grant Government Medical College, Mumbai, Maharashtra, India
3 Department of Medicine, Dhruv Hospital, Rajkot, Gujarat, India

Abstract
Cardiac amyloidosis has gained increasing attention in recent years due to substantial
advancements in diagnostic techniques and treatment options. Among the most
notable developments in this field is the approval of tafamidis by the U.S. Food and
Drug Administration in May 2019 for treating transthyretin amyloid cardiomyopathy
(ATTR-CM). This marked a pivotal step forward in managing this complex condition.
This systematic review aimed to comprehensively analyze the efficacy of tafamidis in
treating ATTR-CM and determine its broader applications, with a focus on its impact
on patient outcomes, optimal dosing strategies, and side effect profiles. We strictly
adhered to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses
2020 guidelines, ensuring that the methodology was transparent and rigorous. This
*Corresponding author: review involved an extensive search of multiple databases, including PubMed and
Heet N. Desai
(hdesai879@gmail.com) Google Scholar, for relevant articles published between January 1, 2014 and January
9, 2024. After a comprehensive screening and quality assessment process, 16 high-
Citation: Desai HN, Sanghvi quality articles were identified and included in the final analysis. These articles
R, Dhruv SH. Unlocking the
potential of tafamidis in treating provided abundant data on the clinical outcomes associated with tafamidis use in
transthyretin cardiac amyloidosis: ATTR-CM. The findings of our review highlight the transformative role of tafamidis in
A systematic review. Brain & Heart. the treatment of ATTR-CM. The drug not only improves the quality of life of patients
2024;2(4):4250.
doi: 10.36922/bh.4250 by significantly alleviating symptoms but also markedly reduces hospitalization
rates and all-cause mortality. Tafamidis showed an impressive safety profile across
Received: July 15, 2024
different dosage levels, representing a key therapeutic option. As the use of tafamidis
Accepted: September 24, 2024 continues to expand in clinical practice, its potential to improve short- and long-term
Published Online: November 5, outcomes in patients with ATTR-CM becomes increasingly evident.
2024
Copyright: © 2024 Author(s). Keywords: Tafamidis; Benzoxazole; Cardiac amyloidosis; Transthyretin amyloid
This is an Open-Access article
distributed under the terms of the cardiomyopathy
Creative Commons Attribution
License, permitting distribution,
and reproduction in any medium,
provided the original work is
properly cited. 1. Introduction
Publisher’s Note: AccScience Cardiac amyloidosis (CA) is a type of heart disease characterized by abnormal deposition
Publishing remains neutral with of amyloid fibrils within the myocardial tissue, leading to heart failure (HF). In the early
regard to jurisdictional claims in
published maps and institutional stages, this condition often presents as HF with a preserved ejection fraction. However,
affiliations. as the disease progresses, patients may experience HF with a reduced ejection fraction

Volume 2 Issue 4 (2024) 1 doi: 10.36922/bh.4250