{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T12:09:14Z","timestamp":1790856554895,"version":"4.1.0"},"reference-count":21,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp;amp; Cancer"],"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Relapsed and\/or refractory (R\/R) pediatric acute leukemia carries a dismal prognosis, largely driven by chemoresistance to conventional salvage therapy. The BH3\u2010mimetic venetoclax, combined with chemotherapy or hypomethylating agents, has demonstrated efficacy in small clinical trials. However, data on its role as a bridge to hematopoietic stem cell transplantation (HSCT) and on re\u2010challenge in previously exposed patients remain limited. We report outcomes from 21 children treated with 26 episodes of venetoclax\u2010based regimens as a bridge to HSCT, including five after prior venetoclax exposure. Overall response rate was 75% (15\/20) after initial treatment and 100% (5\/5) after re\u2010treatment. Overall, 62% (16\/26) of all patient\u2010episodes facilitated immediate HSCT, and for another 12% (3\/26), venetoclax\u2010based therapy eradicated disease, and the patient transitioned to surveillance rather than HSCT. As such, venetoclax may represent an effective bridging strategy in R\/R pediatric leukemia.<\/jats:p>","DOI":"10.1002\/1545-5017.70715","type":"journal-article","created":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T11:50:40Z","timestamp":1790855440000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Venetoclax\u2010Based Therapy as a Bridge to Hematopoietic Stem Cell Transplantation in Relapsed or Refractory Pediatric Acute Leukemia"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3062-4506","authenticated-orcid":false,"given":"Katherine S.","family":"Colman","sequence":"first","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Faculty of Medicine Dentistry and Health Sciences The University of Melbourne  Parkville Victoria Australia"},{"name":"Blood Cells and Blood Cancer Division Walter and Eliza Hall Institute of Medical Research  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deborah","family":"Meyran","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"},{"name":"Cancer Immunology Program Peter MacCallum Cancer Centre  Melbourne Victoria Australia"},{"name":"Sir Peter MacCallum Department of Oncology The University of Melbourne  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2718-0171","authenticated-orcid":false,"given":"Vajiranee","family":"Malalasekera","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Faculty of Medicine Dentistry and Health Sciences The University of Melbourne  Parkville Victoria Australia"},{"name":"Blood Cells and Blood Cancer Division Walter and Eliza Hall Institute of Medical Research  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8592-4311","authenticated-orcid":false,"given":"Heather","family":"Weerdenburg","sequence":"additional","affiliation":[{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"},{"name":"Department of Pharmacy Children's Cancer Centre The Royal Children's Hospital  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janine","family":"Campbell","sequence":"additional","affiliation":[{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Department of Laboratory Services The Royal Children's Hospital Melbourne  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2344-1365","authenticated-orcid":false,"given":"Rachel","family":"Conyers","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5476-9323","authenticated-orcid":false,"given":"Stacie","family":"Wang","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Blood Cells and Blood Cancer Division Walter and Eliza Hall Institute of Medical Research  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3851-7502","authenticated-orcid":false,"given":"Hannah","family":"Walker","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9084-8974","authenticated-orcid":false,"given":"Seong Lin","family":"Khaw","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diane","family":"Hanna","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre The Royal Children's Hospital Melbourne  Parkville Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Melbourne Victoria Australia"},{"name":"Stem Cell Medicine Murdoch Children's Research Institute  Parkville Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,10,1]]},"reference":[{"key":"e_1_2_13_2_1","unstructured":"Australian Institute of Health and Welfare Australia's Children(AIHW 2022) https:\/\/www.aihw.gov.au\/reports\/children\u2010youth\/australias\u2010children."},{"issue":"11","key":"e_1_2_13_3_1","doi-asserted-by":"crossref","first-page":"2382","DOI":"10.1038\/s41375-024-02395-4","article-title":"Determinants of Survival After First Relapse of Acute Lymphoblastic Leukemia: A Children's Oncology Group Study","volume":"38","author":"Rheingold S. R.","year":"2024","journal-title":"Leukemia"},{"issue":"10","key":"e_1_2_13_4_1","doi-asserted-by":"crossref","first-page":"2336","DOI":"10.3390\/cancers13102336","article-title":"Survival Following Relapse in Children With Acute Myeloid Leukemia: A Report From AML\u2010BFM and COG","volume":"13","author":"Rasche M.","year":"2021","journal-title":"Cancers (Basel)"},{"issue":"1","key":"e_1_2_13_5_1","doi-asserted-by":"crossref","first-page":"1590","DOI":"10.1182\/blood-2023-189080","article-title":"Pre\u2010Transplant Levels of Multiparametric Flow\u2010Cytometry Measurable Residual Disease (MFC\u2010MRD) Predict Outcomes in Children With Acute Myeloid Leukemia Given Allogeneic Hematopoietic Stem Cell Transplantation","volume":"142","author":"Buldini B.","year":"2023","journal-title":"Blood"},{"issue":"5","key":"e_1_2_13_6_1","doi-asserted-by":"crossref","first-page":"680","DOI":"10.1111\/bjh.15086","article-title":"Pre\u2010 and Post\u2010Transplant Minimal Residual Disease Predicts Relapse Occurrence in Children With Acute Lymphoblastic Leukaemia","volume":"180","author":"Lovisa F.","year":"2018","journal-title":"British Journal of Haematology"},{"issue":"6","key":"e_1_2_13_7_1","doi-asserted-by":"crossref","first-page":"1440","DOI":"10.1158\/2159-8290.CD-20-1465","article-title":"Venetoclax and Navitoclax in Combination With Chemotherapy in Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma","volume":"11","author":"Pullarkat V. A.","year":"2021","journal-title":"Cancer Discovery"},{"issue":"6","key":"e_1_2_13_8_1","doi-asserted-by":"crossref","DOI":"10.1002\/pbc.31630","article-title":"Venetoclax Combined With Chemotherapy in Pediatric and Adolescent\/Young Adult Patients With Relapsed\/Refractory Acute Lymphoblastic Leukemia","volume":"72","author":"Place A. E.","year":"2025","journal-title":"Pediatric Blood and Cancer"},{"issue":"4","key":"e_1_2_13_9_1","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1016\/S1470-2045(20)30060-7","article-title":"Venetoclax in Combination With Cytarabine With or Without Idarubicin in Children With Relapsed or Refractory Acute Myeloid Leukaemia: A Phase 1, Dose\u2010Escalation Study","volume":"21","author":"Karol S. E.","year":"2020","journal-title":"Lancet Oncology"},{"key":"e_1_2_13_10_1","doi-asserted-by":"crossref","DOI":"10.1002\/pbc.31714","article-title":"Venetoclax Alone or in Combination With Chemotherapy in Paediatric and Adolescent\/Young Adult Patients With Relapsed\/Refractory Acute Myeloid Leukaemia","volume":"72","author":"Karol S. E.","year":"2025","journal-title":"Pediatric Blood and Cancer"},{"issue":"13","key":"e_1_2_13_11_1","doi-asserted-by":"crossref","first-page":"3583","DOI":"10.1182\/bloodadvances.2023012041","article-title":"Venetoclax: A New Player in the Treatment of Children With High\u2010Risk Myeloid Malignancies?","volume":"8","author":"Masetti R.","year":"2024","journal-title":"Blood Advances"},{"issue":"10","key":"e_1_2_13_12_1","doi-asserted-by":"crossref","DOI":"10.1002\/pbc.28398","article-title":"Single\u2010Center Pediatric Experience With Venetoclax and Azacitidine as Treatment for Myelodysplastic Syndrome and Acute Myeloid Leukemia","volume":"67","author":"Winters A. C.","year":"2020","journal-title":"Pediatric Blood and Cancer"},{"issue":"1","key":"e_1_2_13_13_1","doi-asserted-by":"crossref","first-page":"150","DOI":"10.3390\/cancers14010150","article-title":"Venetoclax for Children and Adolescents With Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma","volume":"14","author":"Gibson A.","year":"2022","journal-title":"Cancers"},{"issue":"3","key":"e_1_2_13_14_1","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1038\/s41409-022-01877-2","article-title":"Venetoclax\u2010Based Therapy as a Bridge to Allogeneic Hematopoietic Cell Transplantation in Children With Relapsed\/Refractory AML","volume":"58","author":"Pfeiffer T.","year":"2023","journal-title":"Bone Marrow Transplant"},{"issue":"7","key":"e_1_2_13_15_1","doi-asserted-by":"crossref","first-page":"1983","DOI":"10.3390\/cancers15071983","article-title":"Venetoclax for Acute Myeloid Leukemia in Pediatric Patients: A Texas Medical Center Experience","volume":"15","author":"Trabal A.","year":"2023","journal-title":"Cancers"},{"issue":"11","key":"e_1_2_13_16_1","doi-asserted-by":"crossref","first-page":"3142","DOI":"10.3324\/haematol.2022.282637","article-title":"Clinical Responses in Pediatric Patients With Relapsed\/Refractory Leukemia Treated With Azacitidine and Venetoclax","volume":"108","author":"Niswander L.","year":"2023","journal-title":"Haematologica"},{"issue":"6","key":"e_1_2_13_17_1","doi-asserted-by":"crossref","DOI":"10.1002\/pbc.30335","article-title":"Venetoclax in Combination With Chemotherapy as Treatment for Pediatric Advanced Hematologic Malignancies","volume":"70","author":"Marinoff A. 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M.","year":"2011","journal-title":"PLoS ONE"},{"issue":"10","key":"e_1_2_13_21_1","doi-asserted-by":"crossref","first-page":"1382","DOI":"10.1182\/blood-2016-03-707414","article-title":"Venetoclax Responses of Pediatric All Xenografts Reveal Sensitivity of MLL\u2010Rearranged Leukemia","volume":"128","author":"Khaw S. L.","year":"2016","journal-title":"Blood"},{"issue":"20","key":"e_1_2_13_22_1","doi-asserted-by":"crossref","first-page":"2127","DOI":"10.1182\/blood.2022016040","article-title":"Single\u2010Cell Multiomics Reveal the Scale of Multilayered Adaptations Enabling CLL Relapse During Venetoclax Therapy","volume":"140","author":"Thijssen R.","year":"2022","journal-title":"Blood"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/1545-5017.70715","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/1545-5017.70715","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/1545-5017.70715","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T11:50:44Z","timestamp":1790855444000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/1545-5017.70715"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10,1]]},"references-count":21,"alternative-id":["10.1002\/1545-5017.70715"],"URL":"https:\/\/doi.org\/10.1002\/1545-5017.70715","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,10,1]]},"assertion":[{"value":"2026-01-06","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-09-09","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-10-01","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70715"}}