{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T23:40:15Z","timestamp":1792453215217,"version":"4.5.0"},"reference-count":71,"publisher":"American Society of Hematology","issue":"14","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2026,10,1]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>Ziftomenib, a potent, selective, oral menin inhibitor, is approved as monotherapy for adults with relapsed\/refractory (R\/R) NPM1-mutated acute myeloid leukemia (NPM1-m AML). The KOMET-007 phase 1 trial investigated clinical activity and tolerability of ziftomenib in combination with standard therapies for R\/R and newly diagnosed AML. Here, we report the outcomes of adults with R\/R NPM1-m AML treated with ziftomenib plus venetoclax\/azacitidine. In phase 1a, patients received ziftomenib 200, 400, or 600 mg once daily with standard doses of venetoclax\/azacitidine. In phase 1b, ziftomenib 600 mg was selected for expansion. A total of 67 patients were treated (27 phase 1a; 40 phase 1b). Median age was 66 years; 55% were male. Median number of previous therapies was 1 (range, 1-8); 55% received previous venetoclax, and 22% had received previous transplantation. Most common (\u226520%) grade \u22653 treatment-emergent adverse events were leukopenia (34%), thrombocytopenia (28%), febrile neutropenia and neutropenia (25% each). Six patients developed corrected QT prolongation (1 ziftomenib-related; grade 1), and 2 experienced differentiation syndrome (grade 3); all events were successfully managed. In patients receiving ziftomenib 600 mg, composite complete remission (CRc) rate was 46% (22\/48), with 67% (12\/18) achieving central measurable residual disease (MRD) negativity (&amp;lt;0.01% threshold). In venetoclax-na\u00efve and -exposed patients, CRc rates were 70% (16\/23) and 24% (6\/25), with MRD negativity rates of 75% (9\/12) and 50% (3\/6), respectively. Median duration of response was 8.6 months, and median overall survival was not reached. The combination of ziftomenib 600 mg with venetoclax\/azacitidine was well tolerated with deep and durable clinical activity in R\/R NPM1-m AML. This trial was registered at www.clinicaltrials.gov as NCT05735184.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/blood.2026034043","type":"journal-article","created":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T12:31:31Z","timestamp":1780403491000},"page":"1775-1788","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":6,"title":["Ziftomenib with venetoclax and azacitidine for relapsed\/refractory\n                    <i>NPM1<\/i>\n                    -mutated acute myeloid leukemia"],"prefix":"10.1182","volume":"148","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8644-0968","authenticated-orcid":false,"given":"Eunice S.","family":"Wang","sequence":"first","affiliation":[{"name":"1Leukemia Service, Department of Medicine, Roswell Park Comprehensive Cancer Center, Buffalo, NY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1093-2189","authenticated-orcid":false,"given":"Harry","family":"Erba","sequence":"additional","affiliation":[{"name":"2Department of Medicine, Duke Cancer Institute, Durham, NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amer M.","family":"Zeidan","sequence":"additional","affiliation":[{"name":"3Division of Hematologic Malignancies, Yale University and Yale Comprehensive Cancer Center, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0384-3658","authenticated-orcid":false,"given":"Gail J.","family":"Roboz","sequence":"additional","affiliation":[{"name":"4Weill Cornell Medicine and New York\u2013Presbyterian Hospital, New York, NY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica K.","family":"Altman","sequence":"additional","affiliation":[{"name":"5Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, IL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0015-5902","authenticated-orcid":false,"given":"Anjali S.","family":"Advani","sequence":"additional","affiliation":[{"name":"6Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0242-6449","authenticated-orcid":false,"given":"Tara L.","family":"Lin","sequence":"additional","affiliation":[{"name":"7Department of Internal Medicine, The University of Kansas Medical Center, Kansas City, KS"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6861-2041","authenticated-orcid":false,"given":"Stephen A.","family":"Strickland","sequence":"additional","affiliation":[{"name":"8Sarah Cannon Research Institute at TriStar Centennial, Nashville, TN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2074-5678","authenticated-orcid":false,"given":"Mark B.","family":"Juckett","sequence":"additional","affiliation":[{"name":"9Department of Hematology, University of Minnesota, Minneapolis, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keith W.","family":"Pratz","sequence":"additional","affiliation":[{"name":"10Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James K.","family":"Mangan","sequence":"additional","affiliation":[{"name":"11Division of Blood and Marrow Transplantation, University of California San Diego Moores Cancer Center, La Jolla, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9745-9595","authenticated-orcid":false,"given":"Christine M.","family":"McMahon","sequence":"additional","affiliation":[{"name":"12Division of Hematology, Anschutz Medical Campus, University of Colorado, Aurora, CO"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leonard C.","family":"Alsfeld","sequence":"additional","affiliation":[{"name":"13Hematology and Oncology, Ochsner MD Anderson Cancer Center, New Orleans, LA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suresh Kumar","family":"Balasubramanian","sequence":"additional","affiliation":[{"name":"14Karmanos Cancer Institute, Wayne State University, Detroit, MI"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guru Subramanian","family":"Guru Murthy","sequence":"additional","affiliation":[{"name":"15Froedtert & Medical College of Wisconsin, Milwaukee, WI"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcello","family":"Rotta","sequence":"additional","affiliation":[{"name":"16Colorado Blood Cancer Institute, Denver, CO"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neil D.","family":"Palmisiano","sequence":"additional","affiliation":[{"name":"17Rutgers Cancer Institute of New Jersey, New Brunswick, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"McCloskey","sequence":"additional","affiliation":[{"name":"18John Theurer Cancer Center, Hackensack University Medical Center, Hackensack, NJ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5134-7336","authenticated-orcid":false,"given":"Antoine N.","family":"Saliba","sequence":"additional","affiliation":[{"name":"19Division of Hematology\/Oncology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamad","family":"Khawandanah","sequence":"additional","affiliation":[{"name":"20University of Oklahoma Health Sciences Center, Stephenson Cancer Center, Oklahoma City, OK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9573-2690","authenticated-orcid":false,"given":"Yazan F.","family":"Madanat","sequence":"additional","affiliation":[{"name":"21Division of Hematology and Medical Oncology, The University of Texas Southwestern Medical Center, Dallas, TX"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kiran","family":"Naqvi","sequence":"additional","affiliation":[{"name":"22Chao Family Comprehensive Cancer Center, University of California Irvine Health, Orange, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ayman H.","family":"Qasrawi","sequence":"additional","affiliation":[{"name":"23Department of Internal Medicine, University of Kentucky, Lexington, KY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary J.","family":"Schiller","sequence":"additional","affiliation":[{"name":"24David Geffen School of Medicine at University of California Los Angeles, Los Angeles, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1548-918X","authenticated-orcid":false,"given":"Talha","family":"Badar","sequence":"additional","affiliation":[{"name":"25Department of Hematology, Mayo Clinic, Jacksonville, FL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0312-3412","authenticated-orcid":false,"given":"Ivana","family":"Gojo","sequence":"additional","affiliation":[{"name":"26Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"George","family":"Yaghmour","sequence":"additional","affiliation":[{"name":"27Division of Hematology, University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diaa","family":"Osman","sequence":"additional","affiliation":[{"name":"28Texas Oncology, Lakeway, TX"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongling","family":"Zhang","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ying","family":"Tian","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harris S.","family":"Soifer","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcie","family":"Riches","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Corum","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mollie","family":"Leoni","sequence":"additional","affiliation":[{"name":"29Kura Oncology, Inc, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5848-6221","authenticated-orcid":false,"given":"Amir T.","family":"Fathi","sequence":"additional","affiliation":[{"name":"30Massachusetts General Hospital, Harvard Medical School, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4339-8683","authenticated-orcid":false,"given":"Ghayas C.","family":"Issa","sequence":"additional","affiliation":[{"name":"31Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"1","key":"2026100111262654800_bib1","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1182\/asheducation-2016.1.348","article-title":"Mutations in AML: prognostic and therapeutic implications","volume":"2016","author":"DiNardo","year":"2016","journal-title":"Hematol Am Soc Hematol Educ Program"},{"key":"2026100111262654800_bib2","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.gde.2016.03.011","article-title":"Genetic and epigenetic heterogeneity in acute myeloid leukemia","volume":"36","author":"Li","year":"2016","journal-title":"Curr Opin Genet Dev"},{"issue":"4","key":"2026100111262654800_bib3","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1182\/blood-2016-08-733196","article-title":"Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel","volume":"129","author":"Dohner","year":"2017","journal-title":"Blood"},{"issue":"10","key":"2026100111262654800_bib4","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1002\/ajh.25214","article-title":"Acute myeloid leukemia: 2019 update on risk-stratification and management","volume":"93","author":"Estey","year":"2018","journal-title":"Am J Hematol"},{"issue":"10147","key":"2026100111262654800_bib5","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1016\/S0140-6736(18)31041-9","article-title":"Acute myeloid leukaemia","volume":"392","author":"Short","year":"2018","journal-title":"Lancet"},{"issue":"5","key":"2026100111262654800_bib6","doi-asserted-by":"crossref","first-page":"454","DOI":"10.1056\/NEJMoa1614359","article-title":"Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation","volume":"377","author":"Stone","year":"2017","journal-title":"N Engl J Med"},{"issue":"18","key":"2026100111262654800_bib7","doi-asserted-by":"crossref","first-page":"1728","DOI":"10.1056\/NEJMoa1902688","article-title":"Gilteritinib or chemotherapy for relapsed or refractory FLT3-mutated AML","volume":"381","author":"Perl","year":"2019","journal-title":"N Engl J Med"},{"issue":"10388","key":"2026100111262654800_bib8","doi-asserted-by":"crossref","first-page":"1571","DOI":"10.1016\/S0140-6736(23)00464-6","article-title":"Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial","volume":"401","author":"Erba","year":"2023","journal-title":"Lancet"},{"issue":"25","key":"2026100111262654800_bib9","doi-asserted-by":"crossref","first-page":"2386","DOI":"10.1056\/NEJMoa1716984","article-title":"Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML","volume":"378","author":"DiNardo","year":"2018","journal-title":"N Engl J Med"},{"issue":"6","key":"2026100111262654800_bib10","doi-asserted-by":"crossref","first-page":"722","DOI":"10.1182\/blood-2017-04-779405","article-title":"Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia","volume":"130","author":"Stein","year":"2017","journal-title":"Blood"},{"issue":"7954","key":"2026100111262654800_bib11","doi-asserted-by":"crossref","first-page":"920","DOI":"10.1038\/s41586-023-05812-3","article-title":"The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia","volume":"615","author":"Issa","year":"2023","journal-title":"Nature"},{"issue":"6","key":"2026100111262654800_bib12","doi-asserted-by":"crossref","first-page":"e572","DOI":"10.1097\/HS9.0000000000000572","article-title":"Treatment for relapsed\/refractory acute myeloid leukemia","volume":"5","author":"Thol","year":"2021","journal-title":"Hemasphere"},{"issue":"1","key":"2026100111262654800_bib13","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1182\/blood.2023022481","article-title":"How I treat refractory and relapsed acute myeloid leukemia","volume":"143","author":"Thol","year":"2024","journal-title":"Blood"},{"issue":"10","key":"2026100111262654800_bib14","doi-asserted-by":"crossref","first-page":"1293","DOI":"10.1200\/JCO.2011.40.5977","article-title":"Curability of patients with acute myeloid leukemia who did not undergo transplantation in first remission","volume":"31","author":"Burnett","year":"2013","journal-title":"J Clin Oncol"},{"issue":"5","key":"2026100111262654800_bib15","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1200\/JCO.2013.54.4973","article-title":"Allogeneic stem-cell transplantation in patients with NPM1-mutated acute myeloid leukemia: results from a prospective donor versus no-donor analysis of patients after upfront HLA typing within the SAL-AML 2003 trial","volume":"33","author":"Rollig","year":"2015","journal-title":"J Clin Oncol"},{"issue":"23","key":"2026100111262654800_bib16","doi-asserted-by":"crossref","first-page":"2209","DOI":"10.1056\/NEJMoa1516192","article-title":"Genomic classification and prognosis in acute myeloid leukemia","volume":"374","author":"Papaemmanuil","year":"2016","journal-title":"N Engl J Med"},{"issue":"5","key":"2026100111262654800_bib17","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1038\/ng.796","article-title":"Mutant nucleophosmin and cooperating pathways drive leukemia initiation and progression in mice","volume":"43","author":"Vassiliou","year":"2011","journal-title":"Nat Genet"},{"issue":"3","key":"2026100111262654800_bib18","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1016\/j.ccell.2018.08.005","article-title":"Mutant NPM1 maintains the leukemic state through HOX expression","volume":"34","author":"Brunetti","year":"2018","journal-title":"Cancer Cell"},{"issue":"3","key":"2026100111262654800_bib19","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1007\/s11864-017-0456-2","article-title":"Treatment of relapsed\/refractory acute myeloid leukemia","volume":"18","author":"Bose","year":"2017","journal-title":"Curr Treat Options Oncol"},{"issue":"7","key":"2026100111262654800_bib20","doi-asserted-by":"crossref","first-page":"1115","DOI":"10.1007\/s00277-018-3304-y","article-title":"Salvage regimens using conventional chemotherapy agents for relapsed\/refractory adult AML patients: a systematic literature review","volume":"97","author":"Megias-Vericat","year":"2018","journal-title":"Ann Hematol"},{"issue":"6","key":"2026100111262654800_bib21","doi-asserted-by":"crossref","first-page":"933","DOI":"10.1182\/bloodadvances.2022008316","article-title":"Clinical outcomes associated with NPM1 mutations in patients with relapsed or refractory AML","volume":"7","author":"Issa","year":"2023","journal-title":"Blood Adv"},{"issue":"suppl 1","key":"2026100111262654800_bib22","doi-asserted-by":"crossref","first-page":"2802","DOI":"10.1182\/blood-2018-99-113109","article-title":"More than 10% of NPM1-mutated AML relapses occur after 5 years from complete remission [abstract]","volume":"132","author":"Bertoli","year":"2018","journal-title":"Blood"},{"issue":"8","key":"2026100111262654800_bib23","doi-asserted-by":"crossref","first-page":"1317","DOI":"10.3324\/haematol.2014.104133","article-title":"Molecular response assessment by quantitative real-time polymerase chain reaction after induction therapy in NPM1-mutated patients identifies those at high risk of relapse","volume":"99","author":"Hubmann","year":"2014","journal-title":"Haematologica"},{"issue":"5","key":"2026100111262654800_bib24","doi-asserted-by":"crossref","first-page":"1552","DOI":"10.1182\/bloodadvances.2020003734","article-title":"Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed\/refractory AML","volume":"5","author":"Stahl","year":"2021","journal-title":"Blood Adv"},{"issue":"3","key":"2026100111262654800_bib25","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1007\/s00277-020-03911-z","article-title":"Cytogenetics and mutations could predict outcome in relapsed and refractory acute myeloid leukemia patients receiving BCL-2 inhibitor venetoclax","volume":"99","author":"Wang","year":"2020","journal-title":"Ann Hematol"},{"issue":"1","key":"2026100111262654800_bib26","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1182\/blood-2018-08-868752","article-title":"Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia","volume":"133","author":"DiNardo","year":"2019","journal-title":"Blood"},{"issue":"7","key":"2026100111262654800_bib27","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1056\/NEJMoa2012971","article-title":"Azacitidine and venetoclax in previously untreated acute myeloid leukemia","volume":"383","author":"DiNardo","year":"2020","journal-title":"N Engl J Med"},{"issue":"2","key":"2026100111262654800_bib28","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1002\/ajh.26039","article-title":"Venetoclax with azacitidine or decitabine in patients with newly diagnosed acute myeloid leukemia: long term follow-up from a phase 1b study","volume":"96","author":"Pollyea","year":"2021","journal-title":"Am J Hematol"},{"issue":"15","key":"2026100111262654800_bib29","doi-asserted-by":"crossref","first-page":"1277","DOI":"10.1200\/JCO.18.01600","article-title":"Venetoclax combined with low-dose cytarabine for previously untreated patients with acute myeloid leukemia: results from a phase Ib\/II study","volume":"37","author":"Wei","year":"2019","journal-title":"J Clin Oncol"},{"issue":"13","key":"2026100111262654800_bib30","doi-asserted-by":"crossref","first-page":"1499","DOI":"10.1200\/JCO.23.01911","article-title":"Azacitidine, venetoclax, and gilteritinib in newly diagnosed and relapsed or refractory FLT3-mutated AML","volume":"42","author":"Short","year":"2024","journal-title":"J Clin Oncol"},{"issue":"1","key":"2026100111262654800_bib31","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1038\/s41408-025-01353-2","article-title":"Who truly benefits from gilteritinib combinations in FLT3-mutated relapsed-refractory(R\/R) AML: a Canadian single center analysis","volume":"15","author":"Rajendra","year":"2025","journal-title":"Blood Cancer J"},{"key":"2026100111262654800_bib32","author":"Komzifti (ziftomenib)","year":"2025"},{"issue":"3","key":"2026100111262654800_bib33","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1158\/2159-8290.CD-22-0366","article-title":"Mutant NPM1 directly regulates oncogenic transcription in acute myeloid leukemia","volume":"13","author":"Uckelmann","year":"2023","journal-title":"Cancer Discov"},{"issue":"9","key":"2026100111262654800_bib34","doi-asserted-by":"crossref","first-page":"1452","DOI":"10.1002\/ajh.26989","article-title":"NPM1-mutated acute myeloid leukemia: New pathogenetic and therapeutic insights and open questions","volume":"98","author":"Falini","year":"2023","journal-title":"Am J Hematol"},{"issue":"4","key":"2026100111262654800_bib35","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1097\/MOH.0000000000000245","article-title":"Deregulation of the HOXA9\/MEIS1 axis in acute leukemia","volume":"23","author":"Collins","year":"2016","journal-title":"Curr Opin Hematol"},{"issue":"1","key":"2026100111262654800_bib36","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.ccell.2016.05.008","article-title":"Epigenetic perturbations by Arg882-mutated DNMT3A potentiate aberrant stem cell gene-expression program and acute leukemia development","volume":"30","author":"Lu","year":"2016","journal-title":"Cancer Cell"},{"issue":"23","key":"2026100111262654800_bib37","doi-asserted-by":"crossref","first-page":"3079","DOI":"10.1038\/onc.2015.359","article-title":"DNMT3A mutations mediate the epigenetic reactivation of the leukemogenic factor MEIS1 in acute myeloid leukemia","volume":"35","author":"Ferreira","year":"2016","journal-title":"Oncogene"},{"issue":"1","key":"2026100111262654800_bib38","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1038\/ng.2836","article-title":"Large conserved domains of low DNA methylation maintained by Dnmt3a","volume":"46","author":"Jeong","year":"2014","journal-title":"Nat Genet"},{"issue":"1","key":"2026100111262654800_bib39","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1182\/blood-2004-12-4664","article-title":"Meis1 programs transcription of FLT3 and cancer stem cell character, using a mechanism that requires interaction with Pbx and a novel function of the Meis1 C-terminus","volume":"106","author":"Wang","year":"2005","journal-title":"Blood"},{"issue":"5","key":"2026100111262654800_bib40","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1038\/embor.2011.43","article-title":"The oncometabolite 2-hydroxyglutarate inhibits histone lysine demethylases","volume":"12","author":"Chowdhury","year":"2011","journal-title":"EMBO Rep"},{"issue":"7","key":"2026100111262654800_bib41","doi-asserted-by":"crossref","first-page":"1608","DOI":"10.1038\/s41375-019-0382-3","article-title":"CEBPA-mutated leukemia is sensitive to genetic and pharmacological targeting of the MLL1 complex","volume":"33","author":"Schmidt","year":"2019","journal-title":"Leukemia"},{"issue":"6","key":"2026100111262654800_bib42","doi-asserted-by":"crossref","first-page":"863","DOI":"10.1016\/j.ccell.2016.10.019","article-title":"NUP98 fusion proteins interact with the NSL and MLL1 complexes to drive leukemogenesis","volume":"30","author":"Xu","year":"2016","journal-title":"Cancer Cell"},{"issue":"3","key":"2026100111262654800_bib43","doi-asserted-by":"crossref","first-page":"724","DOI":"10.1158\/2159-8290.CD-22-0424","article-title":"Mutant NPM1 hijacks transcriptional hubs to maintain pathogenic gene programs in acute myeloid leukemia","volume":"13","author":"Wang Xonce-daily","year":"2023","journal-title":"Cancer Discov"},{"issue":"15","key":"2026100111262654800_bib44","doi-asserted-by":"crossref","first-page":"1707","DOI":"10.1182\/blood.2019004226","article-title":"NPM1-mutated acute myeloid leukemia: from bench to bedside","volume":"136","author":"Falini","year":"2020","journal-title":"Blood"},{"issue":"suppl 1","key":"2026100111262654800_bib45","doi-asserted-by":"crossref","first-page":"6226","DOI":"10.1182\/blood-2022-167566","article-title":"The menin inhibitor ziftomenib (KO-539) synergizes with agents targeting chromatin regulation or apoptosis and sensitizes AML with MLL rearrangement or NPM1 mutation to venetoclax [abstract]","volume":"140","author":"Rausch","year":"2022","journal-title":"Blood"},{"issue":"suppl 1","key":"2026100111262654800_bib46","doi-asserted-by":"crossref","first-page":"3357","DOI":"10.1182\/blood-2021-149831","article-title":"Clinical-stage menin inhibitor KO-539 is synergistically active with multiple classes of targeted agents in KMT2A-r and NPM1-mutant AML models [abstract]","volume":"138","author":"Davis","year":"2021","journal-title":"Blood"},{"issue":"suppl 1","key":"2026100111262654800_bib47","doi-asserted-by":"crossref","first-page":"3491","DOI":"10.1182\/blood-2022-157225","article-title":"Preclinical in vivo activity of the menin inhibitor ziftomenib (KO-539) in pediatric KMT2A-rearranged acute lymphoblastic leukemia [abstract]","volume":"140","author":"Falkenstein","year":"2022","journal-title":"Blood"},{"issue":"7","key":"2026100111262654800_bib48","doi-asserted-by":"crossref","first-page":"1734","DOI":"10.3390\/cancers14071734","article-title":"Use of venetoclax in patients with relapsed or refractory acute myeloid leukemia: the PETHEMA Registry Experience","volume":"14","author":"Labrador","year":"2022","journal-title":"Cancers (Basel)"},{"issue":"10","key":"2026100111262654800_bib49","doi-asserted-by":"crossref","first-page":"e724","DOI":"10.1016\/S2352-3026(20)30210-6","article-title":"10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial","volume":"7","author":"DiNardo","year":"2020","journal-title":"Lancet Haematol"},{"issue":"6","key":"2026100111262654800_bib50","doi-asserted-by":"crossref","first-page":"1402","DOI":"10.3324\/haematol.2024.286589","article-title":"Real-life outcome after failure to venetoclax and hypomethylating-based therapy for acute myeloid leukemia","volume":"110","author":"Jimenez-Vicente","year":"2025","journal-title":"haematol"},{"issue":"13","key":"2026100111262654800_bib51","doi-asserted-by":"crossref","first-page":"3245","DOI":"10.1080\/10428194.2022.2113530","article-title":"Dismal survival outcomes of patients with acute myeloid leukemia after failure of venetoclax with hypomethylating agents","volume":"63","author":"Zainaldin","year":"2022","journal-title":"Leuk Lymphoma"},{"issue":"3","key":"2026100111262654800_bib52","doi-asserted-by":"crossref","first-page":"894","DOI":"10.3324\/haematol.2020.252569","article-title":"Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens","volume":"106","author":"Maiti","year":"2021","journal-title":"Haematologica"},{"issue":"1","key":"2026100111262654800_bib53","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1111\/ejh.13527","article-title":"Outcomes of relapsed or refractory acute myeloid leukemia patients failing venetoclax-based salvage therapies","volume":"106","author":"Zucenka","year":"2021","journal-title":"Eur J Haematol"},{"key":"2026100111262654800_bib54","doi-asserted-by":"crossref","first-page":"106942","DOI":"10.1016\/j.leukres.2022.106942","article-title":"Efficacy of FLT3 and IDH1\/2 inhibitors in patients with acute myeloid leukemia previously treated with venetoclax","volume":"122","author":"Bewersdorf","year":"2022","journal-title":"Leuk Res"},{"issue":"11","key":"2026100111262654800_bib55","doi-asserted-by":"crossref","first-page":"3170","DOI":"10.3324\/haematol.2022.282677","article-title":"Outcome of patients with acute myeloid leukemia following failure of frontline venetoclax plus hypomethylating agent therapy","volume":"108","author":"Gangat","year":"2023","journal-title":"Haematologica"},{"issue":"4","key":"2026100111262654800_bib56","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1002\/ajh.27246","article-title":"Long-term follow-up of VIALE-A: venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemia","volume":"99","author":"Pratz","year":"2024","journal-title":"Am J Hematol"},{"issue":"31","key":"2026100111262654800_bib57","doi-asserted-by":"crossref","first-page":"3381","DOI":"10.1200\/JCO-25-01694","article-title":"Ziftomenib in relapsed or refractory NPM1-mutated AML","volume":"43","author":"Wang","year":"2025","journal-title":"J Clin Oncol"},{"issue":"1","key":"2026100111262654800_bib58","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1038\/s41408-025-01344-3","article-title":"Venetoclax and azacitidine for molecular relapse after intensive chemotherapy in NPM1 or CBF AML: a FILO study","volume":"15","author":"Higue","year":"2025","journal-title":"Blood Cancer J"},{"issue":"18","key":"2026100111262654800_bib59","doi-asserted-by":"crossref","first-page":"2161","DOI":"10.1200\/JCO.23.01599","article-title":"Targeting molecular measurable residual disease and low-blast relapse in AML with venetoclax and low-dose cytarabine: a prospective phase II study (VALDAC)","volume":"42","author":"Tiong","year":"2024","journal-title":"J Clin Oncol"},{"issue":"10","key":"2026100111262654800_bib60","doi-asserted-by":"crossref","first-page":"1310","DOI":"10.1016\/S1470-2045(24)00386-3","article-title":"Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial","volume":"25","author":"Wang","year":"2024","journal-title":"Lancet Oncol"},{"issue":"suppl 1","key":"2026100111262654800_bib61","doi-asserted-by":"crossref","first-page":"1359","DOI":"10.1182\/blood-2019-125377","article-title":"Hematologic recovery from venetoclax-containing regimens in relapsed\/refractory acute myeloid leukemia patients depending on prior allogeneic hematopoietic cell transplantation [abstract]","volume":"134","author":"Shahswar","year":"2019","journal-title":"Blood"},{"issue":"9","key":"2026100111262654800_bib62","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.1182\/blood.2025028357","article-title":"Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study","volume":"146","author":"Arellano","year":"2025","journal-title":"Blood"},{"issue":"23","key":"2026100111262654800_bib63","doi-asserted-by":"crossref","first-page":"2606","DOI":"10.1200\/JCO-25-00914","article-title":"Azacitidine, venetoclax, and revumenib for newly diagnosed NPM1-mutated or KMT2A-rearranged AML","volume":"43","author":"Zeidner","year":"2025","journal-title":"J Clin Oncol"},{"issue":"1","key":"2026100111262654800_bib64","doi-asserted-by":"crossref","first-page":"142","DOI":"10.3390\/cancers17010142","article-title":"Menin inhibitors: new targeted therapies for specific genetic subtypes of difficult-to-treat acute leukemias","volume":"17","author":"Niscola","year":"2025","journal-title":"Cancers (Basel)"},{"issue":"1","key":"2026100111262654800_bib65","doi-asserted-by":"crossref","first-page":"75","DOI":"10.1200\/JCO.24.00826","article-title":"Menin inhibition with revumenib for KMT2A-rearranged relapsed or refractory acute leukemia (AUGMENT-101)","volume":"43","author":"Issa","year":"2025","journal-title":"J Clin Oncol"},{"issue":"22","key":"2026100111262654800_bib66","doi-asserted-by":"crossref","first-page":"3743","DOI":"10.3390\/cancers16223743","article-title":"Targeting menin in acute myeloid leukemia: therapeutic advances and future directions","volume":"16","author":"Dhiman","year":"2024","journal-title":"Cancers (Basel)"},{"issue":"1","key":"2026100111262654800_bib67","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1186\/s13045-025-01718-x","article-title":"Menin inhibitors from monotherapies to combination therapies: clinical trial updates from 2024 ASH annual meeting","volume":"18","author":"Cao","year":"2025","journal-title":"J Hematol Oncol"},{"issue":"3","key":"2026100111262654800_bib68","doi-asserted-by":"crossref","first-page":"599","DOI":"10.1111\/bjh.18887","article-title":"A venetoclax and azacitidine bridge-to-transplant strategy for NPM1-mutated acute myeloid leukaemia in molecular failure","volume":"202","author":"Sartor","year":"2023","journal-title":"Br J Haematol"},{"key":"2026100111262654800_bib69","doi-asserted-by":"crossref","first-page":"20406207251372770","DOI":"10.1177\/20406207251372770","article-title":"Real-world outcomes of azacitidine plus venetoclax in acute myeloid leukemia: a multicenter retrospective cohort study from Thailand","volume":"16","author":"Rattanathammethee","year":"2025","journal-title":"Ther Adv Hematol"},{"issue":"2","key":"2026100111262654800_bib70","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1182\/bloodadvances.2023011106","article-title":"Venetoclax-based low intensity therapy in molecular failure of NPM1-mutated AML","volume":"8","author":"Jimenez-Chillon","year":"2024","journal-title":"Blood Adv"},{"issue":"8","key":"2026100111262654800_bib71","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.3390\/cancers14082025","article-title":"Azacitidine plus venetoclax for the treatment of relapsed and newly diagnosed acute myeloid leukemia patients","volume":"14","author":"Garciaz","year":"2022","journal-title":"Cancers (Basel)"}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/148\/14\/1775\/2593349\/blood_bld-2026-034043-main.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/148\/14\/1775\/2593349\/blood_bld-2026-034043-main.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T15:26:44Z","timestamp":1790868404000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/148\/14\/1775\/568820\/Ziftomenib-with-venetoclax-and-azacitidine-for"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10,1]]},"references-count":71,"journal-issue":{"issue":"14","published-print":{"date-parts":[[2026,10,1]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2026034043","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2026,10,1]]},"published":{"date-parts":[[2026,10,1]]}}}