{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T03:17:03Z","timestamp":1791515823955,"version":"4.3.4"},"reference-count":38,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T00:00:00Z","timestamp":1773360000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T00:00:00Z","timestamp":1773360000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100004328","name":"Genentech","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004328","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006483","name":"AbbVie","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006483","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp;amp; Cancer"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>B\u2010cell lymphoma 2 (BCL\u20102) is overexpressed in certain solid tumors (including neuroblastoma), representing a promising target. Venetoclax is a first\u2010in\u2010class, oral, highly selective BCL\u20102 inhibitor. We report safety, pharmacokinetics, and efficacy of venetoclax in children and young adults with relapsed\/refractory solid tumors.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>\n                      M13\u2010833 (NCT03236857) was a Phase 1, open\u2010label, global, two\u2010part study. Patients received age\u2010\/weight\u2010adjusted venetoclax (400 or 800\u00a0mg\/day adult equivalent dose [AED]) continuously or intermittently (Days 1\u201310 of 21\u2010day cycles) as monotherapy or with cyclophosphamide and topotecan (Cy\u2013Topo; Cy 250\u00a0mg\/m\n                      <jats:sup>2<\/jats:sup>\n                      \/day + Topo 0.75\u00a0mg\/m\n                      <jats:sup>2<\/jats:sup>\n                      \/day; intravenously Days 1\u20135) and myeloid growth factor support. Primary objectives included safety and pharmacokinetic assessments of venetoclax; secondary objectives included efficacy.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Fifty\u2010nine patients in the neuroblastoma (\n                      <jats:italic>n<\/jats:italic>\n                      = 36; median age: 8\u00a0years [range: 1\u201317]) and solid tumor cohorts (\n                      <jats:italic>n<\/jats:italic>\n                      = 23; median age: 16\u00a0years [range: 3\u201324]) were assessed. Grade \u22653 treatment\u2010emergent adverse events (TEAEs) occurred in 35\/36 (97%) and 21\/23 (91%) neuroblastoma and solid tumor patients, respectively; febrile neutropenia was the most common serious TEAE (neuroblastoma 69%; solid tumors 57%). TEAEs leading to discontinuation of venetoclax occurred in 17% (neuroblastoma) and 9% (solid tumors) of patients. No events of tumor lysis syndrome were observed. Peak venetoclax concentrations (\n                      <jats:italic>T<\/jats:italic>\n                      <jats:sub>max<\/jats:sub>\n                      ) occurred 4\u20136\u00a0h post\u2010dose; exposure was comparable across age\/weight subgroups. Objective response rates were 31% (neuroblastoma) and 22% (solid tumors).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Recommended Phase 2 dose of venetoclax was 800\u00a0mg\/day AED (Days 1\u201310 of 21\u2010day cycles). Venetoclax combined with Cy\u2013Topo chemotherapy had a predictable safety profile and showed modest clinical activity in these cohorts.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/1545-5017.70178","type":"journal-article","created":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T10:52:51Z","timestamp":1773399171000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Venetoclax in Pediatric and Young Adult Patients With Relapsed\/Refractory Solid Tumors: Results of a Phase 1 Study"],"prefix":"10.1002","volume":"73","author":[{"given":"Daniel A.","family":"Morgenstern","sequence":"first","affiliation":[{"name":"Department of Paediatrics Hospital for Sick Children &amp; University of Toronto  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Barone","sequence":"additional","affiliation":[{"name":"Department of Paediatric Oncology Great Ormond Street Hospital for Children NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadege","family":"Corradini","sequence":"additional","affiliation":[{"name":"Hematology and Oncology Pediatric Institute, L\u00e9on B\u00e9rard Center  Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5807-4876","authenticated-orcid":false,"given":"Sara M.","family":"Federico","sequence":"additional","affiliation":[{"name":"Department of Oncology St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Flotho","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine Faculty of Medicine University Medical Center Freiburg University of Freiburg  Freiburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas U.","family":"Gerber","sequence":"additional","affiliation":[{"name":"Department of Oncology University Children's Hospital  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seong Lin","family":"Khaw","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre Royal Children's Hospital and Murdoch Children's Research Institute  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6446-9180","authenticated-orcid":false,"given":"Margaret E.","family":"Macy","sequence":"additional","affiliation":[{"name":"Children's Hospital Colorado  Aurora Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9604-4213","authenticated-orcid":false,"given":"Andrew E.","family":"Place","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kieuhoa T.","family":"Vo","sequence":"additional","affiliation":[{"name":"San Francisco School of Medicine University of California UCSF Benioff Children's Hospital  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamed","family":"Badawi","sequence":"additional","affiliation":[{"name":"AbbVie Inc.  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