{"id":64726,"date":"2025-09-15T11:40:23","date_gmt":"2025-09-15T06:10:23","guid":{"rendered":"https:\/\/johnsonfrancis.org\/professional\/?p=64726"},"modified":"2025-09-15T11:40:30","modified_gmt":"2025-09-15T06:10:30","slug":"helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy","status":"publish","type":"post","link":"https:\/\/johnsonfrancis.org\/professional\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\/","title":{"rendered":"HELIOS-B Trial of vutrisiran in transthyretin amyloidosis with cardiomyopathy"},"content":{"rendered":"<iframe loading=\"lazy\" width=\"560\" height=\"315\" src=\"https:\/\/www.youtube.com\/embed\/06HfzlPBE2s?si=KpBBm9f7L1N6dhwN\" title=\"YouTube video player\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe>\n\n<p class=\"wp-block-paragraph\">Transthyretin amyloidosis is a progressive disease due to misfolded protein subunits of transthyretin. Normal transthyretin is a tetrameric protein. Abnormal misfolded subunits accumulate as amyloid fibrils in various organs, including the heart causing cardiomyopathy. Transthyretin amyloid cardiomyopathy (ATTR-CM) manifests as heart failure, arrhythmia, and\/or valvular disease. It is a progressive disease with frequent hospitalizations and finally a fatal outcome within 2-6 years from diagnosis, if untreated [1]. Vutrisiran is a small interfering double stranded RNA (siRNA) which can be administered subcutaneously. It can produce rapid and durable knockdown of circulating TTR protein, which reduces ongoing amyloid deposition and progression of disease.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">HELIOS-B was a double blind, randomized trial with vutrisiran and placebo every 12 weeks for up to 36 months [2]. Primary endpoint was a composite of death from any cause and recurrent cardiovascular events. A total of 655 patients underwent randomization in the HELIOS-B trial. Lower risk of death from any cause and recurrent cardiovascular events was documented in the vutrisiran group. Treatment with vutrisiran resulted in less decline in six minute walk distance. Similar benefits were noted in those not receiving tafamidis at baseline as well. Active treatment group had less decline of quality of life as documented by Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) score.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>References<\/strong><\/p>\n\n\n\n<ol class=\"wp-block-list\">\n<li class=\"\">Maurer MS, Berk JL, Damy T, Sheikh FH, Gonz\u00e1lez-Costello J, Morbach C, Delgado D, Bondue A, Azevedo O, Poulsen SH, Jankowska EA, Yang L, Bender S, Eraly SA, Jay PY, Vest J, Fontana M. Impact of Vutrisiran on Cardiac Biomarkers in Patients With Transthyretin Amyloidosis With Cardiomyopathy From HELIOS-B. J Am Coll Cardiol. 2025 Aug 12;86(6):459-475. doi: 10.1016\/j.jacc.2025.04.055. PMID: 40769675.<\/li>\n\n\n\n<li class=\"\">Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonz\u00e1lez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056\/NEJMoa2409134. Epub 2024 Aug 30. PMID: 39213194.<\/li>\n<\/ol>\n","protected":false},"excerpt":{"rendered":"<p>Transthyretin amyloidosis is a progressive disease due to misfolded protein subunits of transthyretin. Normal transthyretin is a tetrameric protein. Abnormal misfolded subunits accumulate as amyloid fibrils in various organs, including the heart causing cardiomyopathy. Transthyretin amyloid cardiomyopathy (ATTR-CM) manifests as heart failure, arrhythmia, and\/or valvular disease. It is a progressive disease with frequent hospitalizations and [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":64727,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"nf_dc_page":"","footnotes":""},"categories":[9],"tags":[],"class_list":["post-64726","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-general"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>HELIOS-B Trial of vutrisiran in transthyretin amyloidosis with cardiomyopathy - All About Cardiovascular System and Disorders<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/johnsonfrancis.org\/professional\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"HELIOS-B Trial of vutrisiran in transthyretin amyloidosis with cardiomyopathy - All About Cardiovascular System and Disorders\" \/>\n<meta property=\"og:description\" content=\"Transthyretin amyloidosis is a progressive disease due to misfolded protein subunits of transthyretin. Normal transthyretin is a tetrameric protein. Abnormal misfolded subunits accumulate as amyloid fibrils in various organs, including the heart causing cardiomyopathy. Transthyretin amyloid cardiomyopathy (ATTR-CM) manifests as heart failure, arrhythmia, and\/or valvular disease. It is a progressive disease with frequent hospitalizations and [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/johnsonfrancis.org\/professional\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\/\" \/>\n<meta property=\"og:site_name\" content=\"All About Cardiovascular System and Disorders\" \/>\n<meta property=\"article:published_time\" content=\"2025-09-15T06:10:23+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-09-15T06:10:30+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/johnsonfrancis.org\/professional\/wp-content\/uploads\/2025\/09\/HELIOS-B-Trial-of-Vutrisiran.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1203\" \/>\n\t<meta property=\"og:image:height\" content=\"675\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Johnson Francis\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Johnson Francis\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/\"},\"author\":{\"name\":\"Johnson Francis\",\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/#\\\/schema\\\/person\\\/5441d907049b914770f4bd98fb57feec\"},\"headline\":\"HELIOS-B Trial of vutrisiran in transthyretin amyloidosis with cardiomyopathy\",\"datePublished\":\"2025-09-15T06:10:23+00:00\",\"dateModified\":\"2025-09-15T06:10:30+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/\"},\"wordCount\":374,\"publisher\":{\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/#\\\/schema\\\/person\\\/5441d907049b914770f4bd98fb57feec\"},\"image\":{\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/wp-content\\\/uploads\\\/2025\\\/09\\\/HELIOS-B-Trial-of-Vutrisiran.jpg\",\"articleSection\":[\"General Cardiology\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/\",\"url\":\"https:\\\/\\\/johnsonfrancis.org\\\/professional\\\/helios-b-trial-of-vutrisiran-in-transthyretin-amyloidosis-with-cardiomyopathy\\\/\",\"name\":\"HELIOS-B Trial of vutrisiran in transthyretin amyloidosis with cardiomyopathy - 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