{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T06:55:06Z","timestamp":1792652106538,"version":"4.5.0"},"reference-count":50,"publisher":"SAGE Publications","issue":"12","license":[{"start":{"date-parts":[[2025,5,28]],"date-time":"2025-05-28T00:00:00Z","timestamp":1748390400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ann Pharmacother"],"published-print":{"date-parts":[[2025,12]]},"abstract":"\n Objective:<\/jats:title>\n \n To review the pharmacology, efficacy, and safety of revumenib (Revuforj) for relapsed or refractory (r\/r) acute leukemia with a lysine methyltransferase 2A (\n KMT2A)<\/jats:italic>\n gene rearrangement or translocation (\n KMT2Ar<\/jats:italic>\n ).\n <\/jats:p>\n <\/jats:sec>\n \n Data Sources:<\/jats:title>\n \n A literature search was conducted using PubMed\/MEDLINE, applicable published abstracts, and ongoing studies from ClinicalTrials.gov between January 1, 1981, and April 23, 2025. Keywords included Revuforj, revumenib, SNDX-5613,\n KMT2A<\/jats:italic>\n ,\n MLL1<\/jats:italic>\n , and menin.\n <\/jats:p>\n <\/jats:sec>\n \n Study Selection and Data Extraction:<\/jats:title>\n \n All English-language studies involving revumenib for r\/r acute leukemia with a\n KMT2Ar<\/jats:italic>\n were included.\n <\/jats:p>\n <\/jats:sec>\n \n Data Synthesis:<\/jats:title>\n \n Revumenib, a protein-protein inhibitor that interrupts the interaction between the\n KMT2A<\/jats:italic>\n protein and the scaffold protein menin, was granted approval by the Food and Drug Administration (FDA) for r\/r acute leukemia with\n KMT2Ar<\/jats:italic>\n based on a phase 2 clinical trial in adult and pediatric patients (n = 57), which reported a complete remission or complete remission with partial hematologic recovery of 22.8%. Common grade 3\/4 adverse reactions reported for revumenib include infectious (febrile neutropenia 33%; infection 29%; bacterial infection 20%) and hematologic events (differentiation syndrome 13%; hemorrhage 9%; thrombosis 5%). Grade 3\/4 QT prolongation, the primary dose-limiting adverse effect, was present in 12% of patients. Differentiation syndrome, related to revumenib\u2019s antileukemic effect, was observed in 29% of patients (grade 3\/4: 13%; grade 5: <1%). We also include long-term follow-up for a total of 104 and 135 patients for efficacy and safety results, respectively.\n <\/jats:p>\n <\/jats:sec>\n \n Relevance to Patient Care and Clinical Practice in Comparison to Existing Drugs:<\/jats:title>\n \n In the high-risk disease of r\/r acute leukemia with\n KMT2Ar<\/jats:italic>\n , given limited treatment options, revumenib appears to be a viable, novel treatment option demonstrating clinical efficacy and a manageable adverse effect profile that can be utilized as a bridge to stem cell transplant. Existing therapy options in this setting may include additional traditional chemotherapy, chimeric antigen receptor T-cell therapy (CAR-T), antibody-drug conjugates (eg, gemtuzumab, inotuzumab), bispecific T-cell engager (BiTE) therapies (eg, blinatumomab), DNA methyltransferase inhibitors (eg, azacitidine, decitabine), histone deacetylase inhibitors (eg, vorinostat, panobinostat), and BCL-2 inhibitors (venetoclax).\n <\/jats:p>\n <\/jats:sec>\n \n Conclusions:<\/jats:title>\n \n Revumenib is an innovative targeted treatment with promising activity in r\/r acute leukemia with\n KMT2Ar<\/jats:italic>\n .\n <\/jats:p>\n <\/jats:sec>","DOI":"10.1177\/10600280251341279","type":"journal-article","created":{"date-parts":[[2025,5,29]],"date-time":"2025-05-29T01:51:11Z","timestamp":1748483471000},"page":"1108-1118","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":7,"title":["Revumenib for Relapsed or Refractory Acute Leukemia With a\n KMT2A<\/i>\n Translocation"],"prefix":"10.1177","volume":"59","author":[{"given":"Emilie J.","family":"Lynch","sequence":"first","affiliation":[{"name":"Department of Pharmacy, University of Florida Health Shands Hospital, Gainesville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah Jane E.","family":"Faro","sequence":"additional","affiliation":[{"name":"Department of Pharmacotherapy and Translational Research, College of Pharmacy, University of Florida, Jacksonville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alec M.","family":"Lindstrom","sequence":"additional","affiliation":[{"name":"Department of Pharmacotherapy and Translational Research, College of Pharmacy, University of Florida, Jacksonville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadia A.","family":"Sethi","sequence":"additional","affiliation":[{"name":"Department of Pharmacotherapy and Translational Research, College of Pharmacy, University of Florida, Jacksonville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy Y.","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Pharmacotherapy and Translational Research, College of Pharmacy, University of Florida, Jacksonville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9670-3792","authenticated-orcid":false,"given":"Nathan D.","family":"Seligson","sequence":"additional","affiliation":[{"name":"Department of Pharmacotherapy and Translational Research, College of Pharmacy, University of Florida, Jacksonville, FL, USA"},{"name":"Precision Medicine, Nemours Children\u2019s Health, Jacksonville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,5,28]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1002\/cncr.29170"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.3322\/caac.21820"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.20.03048"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2024-207450"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V96.8.2691"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.26612"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2015.131680"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.18039"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41408-021-00557-6"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.leu.2402883"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.24.00826"},{"key":"e_1_3_2_13_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023022480"},{"key":"e_1_3_2_14_2","unstructured":"US Food and Drug Administration. FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation. 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