{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T17:34:37Z","timestamp":1793208877369,"version":"4.5.0"},"reference-count":22,"publisher":"Oxford University Press (OUP)","issue":"4","license":[{"start":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T00:00:00Z","timestamp":1774742400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006400","name":"Alnylam","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006400","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008732","name":"Uehara Memorial Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008732","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007263","name":"Astellas Foundation for Research on Metabolic Disorders","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100007263","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Foundation for Medical Technology"},{"name":"Japanese Heart Failure Society Tsuchiya Foundation","id":[{"id":"https:\/\/ror.org\/011r7vr09","id-type":"ROR","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,10,28]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>Vutrisiran was shown to maintain overall health status, as measured by the 23-item Kansas City Cardiomyopathy Questionnaire-overall summary score (KCCQ-OSS), in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in HELIOS-B. We assessed individual KCCQ components to better contextualize changes in KCCQ-OSS in a manner relevant to patients and clinicians.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>In this analysis of HELIOS-B, a double-blind, randomized, placebo-controlled trial of vutrisiran in ATTR-CM, we assessed treatment effect on mean score changes from baseline to 30 months for each 23 KCCQ components (scaled from 0 [worst] to 100 [best]) using a multivariable linear regression model. We further assessed overall changes in KCCQ-OSS as a function of age in both treatment groups to estimate the age-equivalent benefit observed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of 654 participants in HELIOS-B, 486 completed KCCQ assessment both at baseline and 30 months (age: 75 \u00b1 6 years; 93% male; 91% wild-type ATTR; baseline KCCQ-OSS: 74.6 \u00b1 18.9). Vutrisiran, compared with placebo, was associated with more favourable changes in most KCCQ components (median: +3.8, range: \u22120.9 to +7.6). The greater differences favouring vutrisiran were observed in \u2018hurrying or jogging\u2019, \u2018walking on level ground,\u2019 and \u2018discouraged\/down with heart failure\u2019. Similar or greater benefits with vutrisiran were observed in patients not on tafamidis at baseline. KCCQ-OSS at 30 months was inversely related to age, with vutrisiran shifting the age-KCCQ-OSS relationship by approximately 11 years (95%CI: 3\u201320 years).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>In HELIOS-B, vutrisiran was associated with improvement in a wide range of individual KCCQ components, with the greatest benefits observed in physical limitations and quality-of-life domains.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/ejhf\/xuag088","type":"journal-article","created":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T11:35:06Z","timestamp":1774784106000},"page":"787-797","source":"Crossref","is-referenced-by-count":2,"title":["Effect of vutrisiran on components of health status in transthyretin amyloidosis with cardiomyopathy: the HELIOS-B study"],"prefix":"10.1093","volume":"28","author":[{"given":"Yasuhiro","family":"Hamatani","sequence":"first","affiliation":[{"name":"Brigham and Women\u2019s Hospital, Harvard Medical School , 75 Francis St, Boston, MA 02115 ,","place":["USA"]},{"name":"National Hospital Organization Kyoto Medical Center , Kyoto ,","place":["Japan"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian L","family":"Claggett","sequence":"additional","affiliation":[{"name":"Brigham and Women\u2019s Hospital, Harvard Medical School , 75 Francis St, Boston, MA 02115 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0885-1953","authenticated-orcid":false,"given":"Muthiah","family":"Vaduganathan","sequence":"additional","affiliation":[{"name":"Brigham and Women\u2019s Hospital, Harvard Medical School , 75 Francis St, Boston, MA 02115 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Birkhoelzer","sequence":"additional","affiliation":[{"name":"University Hospitals Dorset , Bournemouth ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zi Michael","family":"Miao","sequence":"additional","affiliation":[{"name":"Brigham and Women\u2019s Hospital, Harvard Medical School , 75 Francis St, Boston, MA 02115 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Farooq H","family":"Sheikh","sequence":"additional","affiliation":[{"name":"MedStar Heart & Vascular Institute, Georgetown University School of Medicine , Washington, DC ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emre","family":"Aldinc","sequence":"additional","affiliation":[{"name":"Alnylam Pharmaceuticals , Cambridge, MA ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6174-9232","authenticated-orcid":false,"given":"Julian D","family":"Gillmore","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Division of Medicine, UCL, Royal Free Hospital , London ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianna","family":"Fontana","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Division of Medicine, UCL, Royal Free Hospital , London ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3698-9597","authenticated-orcid":false,"given":"Scott D","family":"Solomon","sequence":"additional","affiliation":[{"name":"Brigham and Women\u2019s Hospital, Harvard Medical School , 75 Francis St, Boston, MA 02115 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