The effect of long-term tafamidis treatment on quality of life in people with transthyretin amyloid cardiomyopathy (ATTR-CM): A plain language summary

Summary

What is this summary about?

This summary explains some results of a study called ATTR-ACT and its ongoing long-term extension study that were published in the European Journal of Heart Failure. The purpose of ATTR-ACT was to find out if a drug called tafamidis is an effective treatment for people with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). People took tafamidis or placebo for up to 2.5 years in ATTR-ACT (the initial study). A placebo looks like the study medicine but does not contain any active ingredients. After people completed the initial study, they could take part in the extension study. An extension study allows people to continue receiving treatment after the original clinical study ends and helps researchers understand how well a treatment works over a longer time period. This extension study allows people to receive tafamidis for up to an additional 5 years. People who took placebo in the initial study now receive tafamidis. People who took tafamidis in the initial study continue tafamidis treatment. Researchers looked at changes in peoples' ability to enjoy life (‘quality of life’) and heart failure symptoms since they started ATTR-ACT. Results are available for the first 2.5 years of the extension study.

What are the key takeaways?

During the initial study, there was less worsening of quality of life and heart failure symptoms in people who took tafamidis compared to people who took placebo. In the extension study, quality of life and heart failure symptoms were maintained or nearly maintained in people who took tafamidis in the initial study. In people who started tafamidis in the extension study, quality of life and heart failure symptoms continued to worsen, but the worsening slowed down.

What were the main conclusions reported by the researchers?

Tafamidis slows the worsening of quality of life and heart failure symptoms in people with ATTR-CM. People with ATTR-CM should start treatment early to receive the most benefit.

Clinical Trial Registration: NCT01994889 (ATTR-ACT) (ClinicalTrials.gov)

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Financial disclosure

ATTR-ACT and the extension study were sponsored by Pfizer. Martha Grogan reports grants, participation in advisory boards, and consultancy fees paid to her institution from Alnylam, Eidos, Janssen, Novo Nordisk, and Pfizer. Margot K Davis reports honoraria for advisory board participation from Akcea, Alnylam, AstraZeneca, Bayer, Boehringer Ingelheim, Ferring, Ionis, Janssen, and Pfizer; consulting fees from Janssen and Novo Nordisk; speaking fees from Bayer, Ferring, Janssen, and Pfizer; and research funding from Pfizer. Maria G Crespo-Leiro reports grants from Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares (CIBERCV) and Pfizer to her institution, honorary for advisory board participation from Novo Nordisk, travel grants from Pfizer and personal fees for lectures in scientific meetings from Astellas Pharma, AstraZeneca, Boehringer Ingelheim, Novartis, and Vifor Pharma. Marla B Sultan reports former employment with Pfizer and current employment with Johnson & Johnson. Balarama Gundapaneni and Franca Stedile Angeli report current or former full-time employment with Pfizer and stock and/or stock options at Pfizer. Mazen Hanna reports honoraria for advisory board participation from Alexion Pharmaceuticals, Alnylam, Akcea, BridgeBio, and Pfizer, and served as a speaker for a scientific meeting session funded by Alnylam. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Competing interests disclosure

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript.

Writing disclosure

Writing support for this summary was provided by Emily Balevich, PhD, at Engage Scientific Solutions, and was funded by Pfizer. Authors of the full article were involved in preparing this summary.