{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Rampal RK"],"funding":["Constellation Pharmaceuticals, a Novartis Company","NCI NIH HHS"],"pagination":["1531-1538"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12092244"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["31(5)"],"pubmed_abstract":["Janus kinase (JAK) inhibitors provide limited depth and durability of response in myelofibrosis. We evaluated pelabresib-a bromodomain and extraterminal domain (BET) inhibitor-plus ruxolitinib (a JAK inhibitor) compared with placebo plus ruxolitinib as first-line therapy. In this phase 3 study (MANIFEST-2), JAK inhibitor-naive patients with myelofibrosis were randomized 1:1 to pelabresib 125 mg once daily (QD; 50-175 mg QD permitted) for 14 days followed by a 7-day break (21-day cycle), or to placebo in combination with ruxolitinib 10 or 15 mg twice daily (BID; 5 mg QD-25 mg BID permitted). Primary endpoint was reduction in spleen volume of ≥35% from baseline at week 24. Key secondary endpoints were absolute change in total symptom score (TSS) and TSS50 response (≥50% reduction in TSS from"],"journal":["Nature medicine"],"pubmed_title":["Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial."],"pmcid":["PMC12092244"],"funding_grant_id":["P30 CA008748"],"pubmed_authors":["Kiladjian JJ","Lavie D","Brown B","Palandri F","Chraniuk D","Mesa R","Lucchesi A","Alvarez-Larran A","Vannucchi AM","Patriarca A","Bose P","Oh ST","Harris M","Grosicki S","Scandura JM","Gupta V","Abruzzese E","Mascarenhas J","Lee SE","Kuykendall AT","Li Q","Boxhammer R","Rampal RK","Jegg AM","Talpaz M","Harrison CN","Gerds AT","Kays SK"],"additional_accession":[]},"is_claimable":false,"name":"Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial.","description":"Janus kinase (JAK) inhibitors provide limited depth and durability of response in myelofibrosis. We evaluated pelabresib-a bromodomain and extraterminal domain (BET) inhibitor-plus ruxolitinib (a JAK inhibitor) compared with placebo plus ruxolitinib as first-line therapy. In this phase 3 study (MANIFEST-2), JAK inhibitor-naive patients with myelofibrosis were randomized 1:1 to pelabresib 125 mg once daily (QD; 50-175 mg QD permitted) for 14 days followed by a 7-day break (21-day cycle), or to placebo in combination with ruxolitinib 10 or 15 mg twice daily (BID; 5 mg QD-25 mg BID permitted). Primary endpoint was reduction in spleen volume of ≥35% from baseline at week 24. Key secondary endpoints were absolute change in total symptom score (TSS) and TSS50 response (≥50% reduction in TSS from","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 May","modification":"2026-06-01T10:24:07.929Z","creation":"2026-04-08T11:27:18.052Z"},"accession":"S-EPMC12092244","cross_references":{"pubmed":["40065169"],"doi":["10.1038/s41591-025-03572-3"]}}