{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T22:04:43Z","timestamp":1790892283123,"version":"4.1.0"},"reference-count":27,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,3,10]],"date-time":"2025-03-10T00:00:00Z","timestamp":1741564800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006483","name":"AbbVie","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006483","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004328","name":"Genentech","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004328","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2025,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Acute lymphoblastic leukemia (ALL) is the most common childhood cancer, and although many patients respond to induction therapy, those who relapse or have refractory disease face a poor prognosis. Venetoclax has promising preclinical and clinical activity in ALL. Here, we report the safety and preliminary efficacy of venetoclax combined with chemotherapy in pediatric and adolescent\/young adult patients with relapsed\/refractory ALL.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>This phase 1, open\u2010label, two\u2010part, multicenter study evaluated venetoclax combined with chemotherapy in pediatric and adolescent\/young adult patients (&lt;25 years of age) with relapsed\/refractory ALL. The study is registered with ClinicalTrials.gov, NCT03236857.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Thirty\u2010one patients were treated and received venetoclax monotherapy (<jats:italic>n<\/jats:italic>\u00a0=\u00a01), venetoclax plus dexamethasone and\/or vincristine and\/or pegasparaginase (VXL; <jats:italic>n<\/jats:italic>\u00a0=\u00a020) or venetoclax plus cytarabine and\/or etoposide and\/or pegasparaginase (<jats:italic>n<\/jats:italic>\u00a0=\u00a010). Patients were heavily pretreated, with a median of 3 prior lines of therapy. The most common grade 3\/4 treatment\u2010emergent adverse event was febrile neutropenia (55%). One fatal adverse event possibly related to venetoclax occurred. The overall response rate of treated patients was 42%, with all responding patients achieving complete remission\/complete remission with incomplete marrow recovery. In biomarker\u2010evaluable patients, responses to venetoclax plus VXL\u2010based or cytarabine\u2010based chemotherapy were observed in patients harboring a range of genetic alterations and heterogeneous BH3 family member dependencies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Venetoclax plus VXL\u2010based or cytarabine\u2010based chemotherapy was overall well tolerated, with promising preliminary efficacy.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.31630","type":"journal-article","created":{"date-parts":[[2025,3,12]],"date-time":"2025-03-12T00:58:54Z","timestamp":1741741134000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Venetoclax Combined With Chemotherapy in Pediatric and Adolescent\/Young Adult Patients With Relapsed\/Refractory Acute Lymphoblastic Leukemia"],"prefix":"10.1002","volume":"72","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9604-4213","authenticated-orcid":false,"given":"Andrew E.","family":"Place","sequence":"first","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8113-8180","authenticated-orcid":false,"given":"Seth E.","family":"Karol","sequence":"additional","affiliation":[{"name":"St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher J.","family":"Forlenza","sequence":"additional","affiliation":[{"name":"Memorial Sloan Kettering Cancer Center  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1203-2371","authenticated-orcid":false,"given":"Todd M.","family":"Cooper","sequence":"additional","affiliation":[{"name":"Seattle Children's Hospital Cancer and Blood Disorders Center  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Fraser","sequence":"additional","affiliation":[{"name":"Queensland Children's Hospital  South Brisbane QLD Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8396-0127","authenticated-orcid":false,"given":"Gunnar","family":"Cario","sequence":"additional","affiliation":[{"name":"Universit\u00e4tsklinikum Schleswig\u2010Holstein  Kiel Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maureen M.","family":"O'Brien","sequence":"additional","affiliation":[{"name":"Cincinnati Children's Hospital Medical Center University of Cincinnati College of Medicine  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas U.","family":"Gerber","sequence":"additional","affiliation":[{"name":"University Children's Hospital Zurich  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6571-6227","authenticated-orcid":false,"given":"Jean\u2010Pierre","family":"Bourquin","sequence":"additional","affiliation":[{"name":"University Children's Hospital Zurich  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dirk","family":"Reinhardt","sequence":"additional","affiliation":[{"name":"University of Duisburg\u2010Essen  Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey E.","family":"Rubnitz","sequence":"additional","affiliation":[{"name":"St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph T.","family":"Opferman","sequence":"additional","affiliation":[{"name":"St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gauri","family":"Sunkersett","sequence":"additional","affiliation":[{"name":"AbbVie Inc  North Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maika","family":"Onishi","sequence":"additional","affiliation":[{"name":"Genentech Inc  South San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana R.","family":"Dunshee","sequence":"additional","affiliation":[{"name":"Genentech Inc  South San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xin","family":"Chen","sequence":"additional","affiliation":[{"name":"AbbVie Inc  North Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristina","family":"Unnebrink","sequence":"additional","affiliation":[{"name":"AbbVie Deutschland GmbH &amp; 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