{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T20:51:08Z","timestamp":1791924668532,"version":"4.3.4"},"reference-count":26,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"8","content-domain":{"domain":["www.ahajournals.org"],"crossmark-restriction":true},"short-container-title":["JAHA"],"published-print":{"date-parts":[[2024,4,16]]},"abstract":"<jats:sec sec-type=\"background\" xml:lang=\"en\">\n                    <jats:title>Background<\/jats:title>\n                    <jats:p xml:lang=\"en\">The phase 2 PIONEER\u2010HCM (Phase 2 Open\u2010label Pilot Study Evaluating Mavacamten in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction) study showed that mavacamten improved left ventricular outflow tract\u00a0gradients, exercise capacity, and symptoms in patients with obstructive hypertrophic cardiomyopathy (HCM), but the results of longer\u2010term treatment are less well described. We report interim results from the PIONEER\u2010OLE (PIONEER Open\u2010Label Extension) study, the longest\u2010term study of mavacamten in patients with symptomatic obstructive HCM.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec xml:lang=\"en\">\n                    <jats:title>Methods and Results<\/jats:title>\n                    <jats:p xml:lang=\"en\">Patients who previously completed PIONEER\u2010HCM (n=20) were eligible to enroll in PIONEER\u2010OLE. Patients received oral mavacamten, 5\u2009mg once daily (starting dose), with individualized dose titration at week 6. Evaluations included serial monitoring of safety, echocardiography, Kansas City Cardiomyopathy Questionnaire\u2013Overall Summary Score, and serum NT\u2010proBNP (N\u2010terminal pro\u2010B\u2010type natriuretic peptide) levels. Thirteen patients enrolled and received mavacamten (median study duration at data cutoff, 201\u2009weeks). Most patients (92.3%) received \u03b2\u2010blockers concomitantly. Treatment\u2010emergent adverse events were predominantly mild\/moderate. One patient had an isolated reduction in left ventricular ejection fraction to 47%, which recovered and remained normal with continued treatment at a reduced dose. At week 180, mavacamten was associated with New York Heart Association class improvements from baseline (class II to I, n=9; class III to II, n=1; and unchanged, n=2), sustained reductions in left ventricular outflow tract gradients (mean [SD] change from baseline: resting, \u221250 [55] mm\u2009Hg; Valsalva, \u221270 [41] mm\u2009Hg), and serum NT\u2010proBNP levels (median [interquartile range] change from baseline: \u2212498 [\u22122184 to \u221276] ng\/L), and improved Kansas City Cardiomyopathy Questionnaire\u2013Overall Summary Score (mean [SD] change from baseline: +17 [16]).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec xml:lang=\"en\">\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p xml:lang=\"en\">This long\u2010term analysis supports the continued safety and effectiveness of mavacamten for &gt;3\u2009years in obstructive HCM.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec xml:lang=\"en\">\n                    <jats:title>Registration<\/jats:title>\n                    <jats:p xml:lang=\"en\">\n                      URL:\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/www.clinicaltrials.gov\">https:\/\/www.clinicaltrials.gov<\/jats:ext-link>\n                      ; Unique identifier: NCT03496168.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1161\/jaha.123.030607","type":"journal-article","created":{"date-parts":[[2024,4,9]],"date-time":"2024-04-09T05:41:21Z","timestamp":1712641281000},"update-policy":"https:\/\/doi.org\/10.1161\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":41,"title":["Long\u2010Term Safety and Efficacy of Mavacamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Interim Results of the PIONEER\u2010OLE Study"],"prefix":"10.1161","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6390-6526","authenticated-orcid":false,"given":"Ahmad","family":"Masri","sequence":"first","affiliation":[{"name":"Division of Cardiology, Hypertrophic Cardiomyopathy Center, School of Medicine Oregon Health &amp; Science University Portland OR"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven J.","family":"Lester","sequence":"additional","affiliation":[{"name":"Department of Cardiovascular Diseases Mayo Clinic Arizona Phoenix AZ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1568-9280","authenticated-orcid":false,"given":"John C.","family":"Stendahl","sequence":"additional","affiliation":[{"name":"Section of Cardiovascular Medicine, Department of Internal Medicine Yale School of Medicine New Haven CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8157-8899","authenticated-orcid":false,"given":"Sheila M.","family":"Hegde","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine Brigham and Women\u2019s Hospital Boston MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1912-1710","authenticated-orcid":false,"given":"Amy J.","family":"Sehnert","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb Princeton NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ganesh","family":"Balaratnam","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb Princeton NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashish","family":"Shah","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb Princeton NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shawna","family":"Fox","sequence":"additional","affiliation":[{"name":"IQVIA Durham NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8729-0933","authenticated-orcid":false,"given":"Andrew","family":"Wang","sequence":"additional","affiliation":[{"name":"Duke Cardiology Duke University Hospital Durham NC"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIR.0000000000000937"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehu284"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1710575"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00424-019-02259-2"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.106.644682"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1503\/cmaj.120138"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphys.2022.975076"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1007\/s12551-017-0274-6"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad3456"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M117.776815"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1809540115"},{"key":"e_1_3_2_13_2","unstructured":"CAMZYOS\u2122 (mavacamten) . 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