{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T16:28:45Z","timestamp":1791822525665,"version":"4.3.4"},"reference-count":32,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2024,10,22]],"date-time":"2024-10-22T00:00:00Z","timestamp":1729555200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100017655","name":"Incyte","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100017655","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:label\/><jats:p>Autoimmune hemolytic anemia (AIHA) is a group of acquired autoimmune disorders characterized by red blood cell hemolysis. In a phase 2, open\u2010label, multicenter study, adults with warm AIHA, cold agglutinin disease, or mixed\u2010type AIHA were administered once\u2010daily 1.0 or 2.5\u2009mg parsaclisib (selective phosphoinositide 3\u2010kinase \u03b4 inhibitor) orally for 12\u2009weeks, followed by an extension period. Dose increases (for AIHA worsening) or decreases (for tolerability) were permitted. Primary efficacy endpoint was the proportion of patients with complete (\u226512\u2009g\/dL hemoglobin [Hgb]) or partial (10\u201312\u2009g\/dL Hgb or \u22652\u2009g\/dL increase from baseline) response at any visit during weeks 6\u201312 not attributable to transfusion. Among 25 enrolled patients (median age, 63 y), 16 (64%) achieved a partial or complete Hgb response during weeks 6\u201312. Responses were observed by week 1 in 52.0% of patients with incremental improvements during weeks 6\u201312 and sustained responses during the extension period. Responses were higher among patients with warm AIHA versus other types (75.0% vs. 44.4%). Clinically meaningful improvements in Functional Assessment of Chronic Illness Therapy\u2010Fatigue scores were observed at weeks 6 and 12. All patients had treatment\u2010emergent adverse events (TEAEs), most commonly diarrhea (32.0%) and pyrexia (28.0%). Grade \u22653 TEAEs occurred in 13 patients (52.0%). TEAEs considered possibly related to treatment occurred in 11 patients (44.0%). No dose reductions were required; six patients (24%) discontinued for a TEAE. In summary, parsaclisib was well tolerated and resulted in substantial improvements in Hgb response at week 1, with durable responses through the extension period.<\/jats:p><\/jats:sec><jats:sec><jats:title>Clinical trial registration<\/jats:title><jats:p>This trial was registered at <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/clinicaltrials.gov\">ClinicalTrials.gov<\/jats:ext-link> (NCT03538041).<\/jats:p><\/jats:sec>","DOI":"10.1002\/ajh.27493","type":"journal-article","created":{"date-parts":[[2024,10,22]],"date-time":"2024-10-22T11:55:10Z","timestamp":1729598110000},"page":"2313-2320","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open\u2010label study"],"prefix":"10.1002","volume":"99","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1428-9944","authenticated-orcid":false,"given":"Wilma","family":"Barcellini","sequence":"first","affiliation":[{"name":"Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabrizio","family":"Pane","sequence":"additional","affiliation":[{"name":"Department of Medicine and Surgery University Federico II of Naples  Naples Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Patriarca","sequence":"additional","affiliation":[{"name":"University of Eastern Piedmont and AOU \u201cMaggiore della Carit\u00e0\u201d  Novara Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Murakhovskaya","sequence":"additional","affiliation":[{"name":"Albert Einstein College of Medicine\/Montefiore Medical Center  Bronx New York 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