A Systematic Review of Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy

Bishnu Mohan Singh; Narayan Bohara; Kamal Gautam; Madan Basnet; Sistu Kc; Binod Kc; Anuradha Raut; Abisha Phudong; Jeevan Gautam

doi:10.7759/cureus.18221

A Systematic Review of Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy

Cureus. 2021 Sep 23;13(9):e18221. doi: 10.7759/cureus.18221. eCollection 2021 Sep.

Authors

Bishnu Mohan Singh^{1

2}, Narayan Bohara³, Kamal Gautam⁴, Madan Basnet⁵, Sistu Kc⁶, Binod Kc⁷, Anuradha Raut⁸, Abisha Phudong⁹, Jeevan Gautam¹⁰

Affiliations

¹ Division of Clinical and Translational Research, Larkin Community Hospital, Miami, USA.
² Emergency and General Medicine, Patan Academy of Health Sciences, Lalitpur, NPL.
³ Cardiology, Oxford University Clinical Research Unit, Patan Academy of Health Sciences, Lalitpur, NPL.
⁴ Internal Medicine, Oxford University Clinical Research Unit, Patan Academy of Health Sciences, Lalitpur, NPL.
⁵ Medicine, Nepalese Army Institute of Health Sciences, Kathmandu, NPL.
⁶ Internal Medicine, Patan Academy of Health Sciences, Lalitpur, NPL.
⁷ Internal Medicine, Upstate University Hospital, New York, USA.
⁸ Internal Medicine, Nepal Medical College and Teaching Hospital, Kathmandu, NPL.
⁹ Emergency Department, Care and Cure Multispeciality Hospital, Lalitpur, NPL.
¹⁰ Internal Medicine, Tribhuvan University Teaching Hospital, Kathmandu, NPL.

Abstract

Transthyretin amyloid cardiomyopathy disease burden is increasing daily due to advancements in diagnostic and imaging modalities in the modern world. Tafamidis is one of many therapeutic options. The main objective of this review is to study the role of Tafamidis in slowing the progression of transthyretin cardiomyopathy (TTR-CM) by analyzing randomized controlled trials (RCTs) and non-RCTs of Tafamidis. We searched for published papers of Tafamidis in the English language in electronic databases like Google Scholar, PubMed, Cochrane Library, and PubMed Central. We imported the resulting articles from our search to Mendeley software. Four reviewers removed the duplicates and performed title and abstract screening of the articles. The same reviewers obtained the full-text of relevant articles and did full-text screening based on eligibility criteria. Finally, five reviewers performed a quality assessment of RCTs using the Cochrane risk of bias assessment and of non-RCTs by a checklist prepared by Downs and Black. Any disagreements about any process were resolved by a discussion with other authors. One RCT and five non-RCTs of Tafamidis were included in this systematic review. From the non-RCTs, stability was observed in different parameters like echocardiographic findings, cardiac biomarkers, and ECG in patients with transthyretin cardiomyopathy during the study duration with Tafamidis. ATTR-ACT (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial) trial demonstrated reduction of cardiovascular events and all-cause mortality in the Tafamidis group in comparison to placebo. In both RCT and non-RCTs, Tafamidis was established as a safe and tolerable drug for patients with TTR-CM. Our study proved the role of Tafamidis in reducing cardiovascular events, all-cause mortality, and the progression of cardiac disease in TTR-CM patients. In addition to five non-RCTs, current evidence is based on the findings of only one RCT of Tafamidis. Hence, evidence from additional RCTs is required to strongly support the stability of parameters like echocardiographic findings, cardiac biomarkers, and ECG with Tafamidis use.

Keywords: amyloid; cardiac biomarkers; cardiomyopathies; echocardiography; heart failure; outcomes; randomized controlled trial; tafamidis; transthyretin amyloid cardiomyopathy; ttr-stabilizing drug.

Publication types

Review