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We performed a retrospective chart review of paediatric and AYA AML patients treated with HMA\u2009+\u2009VEN at Cincinnati Children's Hospital Medical Centre. Twenty\u2010seven patients received 30 HMA\u2009+\u2009VEN treatment courses for relapsed\/refractory (R\/R, <jats:italic>n<\/jats:italic>\u2009=\u200921) or newly diagnosed (<jats:italic>n<\/jats:italic>\u2009=\u20099) AML due to ineligibility for intensive chemotherapy. The R\/R cohort had high\u2010risk cytomolecular genetic alterations and prior extensive treatments, with 50% (<jats:italic>n<\/jats:italic> = 9) of relapse patients (<jats:italic>n<\/jats:italic> = 18) having undergone haematopoietic stem cell transplantation (HSCT). Venetoclax treatment using the 400 mg adult exposure\u2010equivelant dosing (AED) had a median duration of 21\u2009days (range 7\u201330\u2009days). Grade 3\u20134 toxicities included neutropenia (90%), anaemia (64%), thrombocytopenia (64%) and febrile neutropenia (44%). The overall complete remission (CR)\/CR with incomplete blood count recovery (CRi) rate was 73% (77% minimal residual disease [MRD] negativity &lt;0.1%), with 60% undergoing HSCT. Among newly diagnosed patients (<jats:italic>n<\/jats:italic>\u2009=\u20099), 89% achieved CR\/CRi (78% MRD negativity) and 78% proceeded to HSCT. The R\/R cohort (<jats:italic>n<\/jats:italic>\u2009=\u200921) showed a 67% CR\/CRi rate (71% MRD negativity), with 52% undergoing HSCT. These findings support the safety and efficacy of HMA\u2009+\u2009VEN in paediatric\/AYA AML, indicating it as a viable option for patients unfit for intensive chemotherapy. Further studies are necessary to determine optimal venetoclax dosing, chemotherapy combinations and pharmacokinetics in this population.<\/jats:p>","DOI":"10.1111\/bjh.19679","type":"journal-article","created":{"date-parts":[[2024,7,31]],"date-time":"2024-07-31T12:33:04Z","timestamp":1722429184000},"page":"1055-1066","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Clinical outcomes of hypomethylating agents and venetoclax in newly diagnosed unfit and relapsed\/refractory paediatric, adolescent and young adult acute myeloid leukaemia patients"],"prefix":"10.1111","volume":"205","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5979-0479","authenticated-orcid":false,"given":"Francis R.","family":"LeBlanc","sequence":"first","affiliation":[{"name":"Division of Oncology, Cancer and Blood Diseases Institute Cincinnati Children's Hospital Medical Centre  Cincinnati Ohio 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Cancer and Blood Diseases Institute Cincinnati Children's Hospital Medical Centre  Cincinnati Ohio USA"},{"name":"Department of Pediatrics University of Cincinnati College of Medicine  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robin E.","family":"Norris","sequence":"additional","affiliation":[{"name":"Division of Oncology, Cancer and Blood Diseases Institute Cincinnati Children's Hospital Medical Centre  Cincinnati Ohio USA"},{"name":"Department of Pediatrics University of Cincinnati College of Medicine  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maureen M.","family":"O'Brien","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Colorado  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine L.","family":"Phillips","sequence":"additional","affiliation":[{"name":"Division of Oncology, Cancer and Blood Diseases Institute 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