{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T00:03:02Z","timestamp":1790467382075,"version":"4.1.0"},"reference-count":32,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2025,5,22]],"date-time":"2025-05-22T00:00:00Z","timestamp":1747872000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100007313","name":"University of Texas MD Anderson Cancer Center","doi-asserted-by":"publisher","award":["CA100632"],"award-info":[{"award-number":["CA100632"]}],"id":[{"id":"10.13039\/100007313","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011096","name":"Jazz Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011096","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":[[2025,8]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:p>Outcomes in patients with relapsed\/refractory (RR) AML are poor. We sought to investigate if CPX\u2010531 in combination with venetoclax (CPX\u2009+\u2009VEN) was tolerable and effective in RR AML. This was a single institution phase 1b\/2 trial of CPX\u2009+\u2009VEN. Patients aged \u2265\u200918\u2009years with RR AML who were fit for intensive chemotherapy were eligible. Prior venetoclax exposure was allowed. The phase 1b portion followed a 3\u2009+\u20093 design to identify the recommended phase 2 dose (RP2D) for the expansion cohort. At the starting dose level of \u22121, prolonged myelosuppression was observed, leading to dose level \u22122 (CPX\u2010351 dosed at daunorubicin 44\u2009mg\/m<jats:sup>2<\/jats:sup> on days 1,3, and 5 and venetoclax 300\u2009mg\u2009days 2\u20138) being chosen as the RP2D. Thirty three patients with a median age of 58\u2009years (range, 26\u201372) were treated. Patients were heavily pretreated, with 58% with prior venetoclax exposure, 44% in the second salvage or later, and 30% with prior stem cell transplant (SCT). Adverse cytogenetics were present in 51% of patients, with myelodysplasia\u2010related mutations in 64%, and <jats:italic>TP53<\/jats:italic><jats:sup>mut<\/jats:sup> in 21%. The overall response rate (ORR) was 46% (95% CI, 30\u201362), with a composite CR rate (CRc) of 39% (95% CI, 25\u201356). Patients in first salvage with wildtype <jats:italic>TP53<\/jats:italic> had a CRc rate of 70% (95% CI, 40\u201389), with undetectable MRD in 71% (95% CI, 36\u201392) and a 2\u2010year OS of 49% (95% CI, 23\u2013100). Eleven (73%) responding patients underwent SCT. The 30\u2010day mortality was 9%, with a 60\u2010day mortality of 21%. The most common adverse events were related to myelosuppression. CPX\u2009+\u2009VEN has activity in RR AML, particularly when used in first salvage and in patients who do not harbor <jats:italic>TP53<\/jats:italic> mutations.<\/jats:p><jats:p><jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/clinicaltrials.gov\">ClinicalTrials.gov<\/jats:ext-link> Identifier: <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT03629171\">NCT03629171<\/jats:ext-link><\/jats:p>","DOI":"10.1002\/ajh.27723","type":"journal-article","created":{"date-parts":[[2025,5,22]],"date-time":"2025-05-22T07:24:06Z","timestamp":1747898646000},"page":"1365-1373","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["A Phase 2 Trial of <scp>CPX<\/scp>\u2010351 Combined With Venetoclax in Relapsed or Refractory Acute Myeloid Leukemia"],"prefix":"10.1002","volume":"100","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9892-9832","authenticated-orcid":false,"given":"Tapan M.","family":"Kadia","sequence":"first","affiliation":[{"name":"Department of Leukemia The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9339-3362","authenticated-orcid":false,"given":"Wei\u2010Ying","family":"Jen","sequence":"additional","affiliation":[{"name":"Department of Leukemia The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6085-2745","authenticated-orcid":false,"given":"Alex","family":"Bataller","sequence":"additional","affiliation":[{"name":"Department of Leukemia The University of Texas MD Anderson Cancer Center  Houston Texas 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