Plain Language Summary
What is this summary about?
This summary explains the results of the HELIOS-B study that assessed vutrisiran treatment in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In ATTR-CM, a protein called transthyretin accumulates in clumps called amyloid in different parts of the body, such as the heart and nerves, causing damage. In the heart, this decreases functioning and leads to heart failure and therefore more frequent hospital visits and a greater risk of dying. Vutrisiran lowers the amount of transthyretin in the blood.
In the HELIOS-B study, 654 participants received the drug vutrisiran or placebo (liquid containing no drug), injected under the skin every 12 weeks for up to 3 years. The study assessed the effect of vutrisiran on deaths, cardiac hospitalizations, and urgent doctor visits for heart failure. The study also looked at changes in physical function (ability to walk), quality of life and heart failure symptoms, and levels of proteins linked to heart failure.
What were the results?
Participants taking vutrisiran versus placebo were around 28% less likely to die, or visit the hospital for heart failure and other circulatory system-related problems; they also had less decline in physical function and quality of life. More participants taking vutrisiran versus placebo had stable/improved heart failure symptoms and also had smaller increases in protein levels linked to heart failure. The number of medical problems was similar for both groups.
What do the results of the HELIOS-B study mean?
The study results showed that participants with ATTR-CM saw benefits with vutrisiran in reducing the risk of death and hospitalizations, with less decline in physical function and quality of life.
This is an abstract of the Plain Language Summary of Publication article.
View the full Plain Language Summary PDF of this article to read the full-text
Acknowledgements
The authors would like to thank all the patients and their families for their participation in the trial and the HELIOS-B study collaborators.
Disclosure statement
Marianna Fontana reports research support from Alnylam Pharmaceuticals, AstraZeneca, BridgeBio, and Pfizer; consultancy for Alexion/Caelum Biosciences, Alnylam Pharmaceuticals, AstraZeneca, Attralus, Bayer, BridgeBio/Eidos, Cardior Pharmaceuticals, Intellia Therapeutics, Ionis Pharmaceuticals, Janssen Pharmaceuticals, Lexeo Therapeutics, Mycardium, Novo Nordisk, Pfizer, and Prothena; and ownership of equity in Lexeo Therapeutics and Mycardium. Julian D Gillmore reports research support from Alnylam Pharmaceuticals; consulting fees from Alnylam Pharmaceuticals, AstraZeneca, Bayer, BridgeBio, Intellia Therapeutics, Ionis Pharmaceuticals, and Lycia Therapeutics; and payment for lectures or speaker fees from Alnylam Pharmaceuticals, AstraZeneca, and Bayer. Ronald M Witteles reports personal fees from Alexion, Alnylam Pharmaceuticals, AstraZeneca, BridgeBio, Novo Nordisk, and Pfizer. Ben Laryea and Marc Dworkin are patient authors and have nothing to disclose. Ann Payne is the Clinical Care and Education Manager at the Amyloidosis Research Consortium and has nothing to disclose. Fran Kochman is an employee of Alnylam Pharmaceuticals and the Head of US Patient Advocacy and Engagement. Satish Eraly is an employee of Alnylam Pharmaceuticals. Mathew S Maurer reports receiving research support from Alnylam Pharmaceuticals, Attralus, BridgeBio, Intellia Therapeutics, and Ionis Pharmaceuticals; and consulting fees from Alnylam Pharmaceuticals, AstraZeneca, Intellia Therapeutics, Ionis Pharmaceuticals, Novo Nordisk, and Roche. 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.
Medical writing support for this summary was provided by Kristen Brown, PhD, of Adelphi Communications Ltd (Macclesfield, UK) in accordance with Good Publication Practice guidelines, and funded by Alnylam Pharmaceuticals.
All participants in the HELIOS-B study provided informed written consent. The trial was conducted in accordance with all applicable regulatory requirements, the International Council for Harmonisation Good Clinical Practice guidelines, and the principles of the Declaration of Helsinki.
Patient reviewers on this PLSP have received honoraria from Future Cardiology for their review work but have no other relevant financial relationships to disclose.
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.
Funding
This manuscript was funded by Alnylam Pharmaceuticals, who were the sponsors of the HELIOS-B study.