{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T04:08:41Z","timestamp":1791950921839,"version":"4.3.4"},"reference-count":27,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,4,10]],"date-time":"2023-04-10T00:00:00Z","timestamp":1681084800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["T32CA128583"],"award-info":[{"award-number":["T32CA128583"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100001445","name":"Alex's Lemonade Stand Foundation for Childhood Cancer","doi-asserted-by":"publisher","award":["CenterofExcellenceAward"],"award-info":[{"award-number":["CenterofExcellenceAward"]}],"id":[{"id":"10.13039\/100001445","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":[[2023,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Venetoclax is frequently used as salvage treatment in pediatric, adolescent, and young adult (AYA) patients with advanced hematologic malignancies. However, more data are needed from real\u2010world studies to guide the safe and appropriate use of venetoclax in this population.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>We retrospectively reviewed the medical records of all patients diagnosed with hematologic malignancies less than 30 years of age treated with venetoclax outside of clinical trials at the University of California San Francisco Benioff Children's Hospitals from 2016 to 2022.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We identified 13 patients (acute myeloid leukemia<jats:italic>, n<\/jats:italic>\u00a0=\u00a08; B\u2010acute lymphoblastic leukemia, <jats:italic>n<\/jats:italic>\u00a0=\u00a03; myelodysplastic syndrome, <jats:italic>n<\/jats:italic>\u00a0=\u00a02) aged 4 months to 27 years. A median of 3 prior lines of therapy weregiven (range 0\u20135). All patients received venetoclax in combination with either a hypomethylating agent or conventional chemotherapy. Three (23%) patients achieved complete remission (CR); two (15%) achieved partial remission (PR); 3 (23%) had stable disease (SD), and five (42%) had progressive disease. Median survival and time to progression from venetoclax initiation was 9 months (range 2.5\u201352 months) and 3 months (range 2\u00a0weeks to 7.5 months), respectively. Six patients (46%) developed grade 3 or higher infections while receiving venetoclax, including bacteremia due to atypical organisms, invasive pulmonary infections with Aspergillus, cytomegalovirus (CMV) viremia, skin infections, and encephalitis with bacterial brain abscesses.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Venetoclax in combination with hypomethylating agents or cytotoxic chemotherapy was effective in a subset of pediatric\/AYA patients with advanced hematologic malignancies, but multiple severe infections were observed, particularly among patients who received venetoclax in combination with chemotherapy. Prospective studies will be required to determine the optimal dose and duration of venetoclax in this population.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.30335","type":"journal-article","created":{"date-parts":[[2023,4,10]],"date-time":"2023-04-10T13:36:45Z","timestamp":1681133805000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Venetoclax in combination with chemotherapy as treatment for pediatric advanced hematologic malignancies"],"prefix":"10.1002","volume":"70","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8600-6400","authenticated-orcid":false,"given":"Amanda E.","family":"Marinoff","sequence":"first","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Aaronson","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4898-9615","authenticated-orcid":false,"given":"Anurag K.","family":"Agrawal","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6996-0833","authenticated-orcid":false,"given":"Benjamin S.","family":"Braun","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Golden","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin J.","family":"Huang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Michlitsch","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica","family":"Southworth","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's Hospitals, University of California, San Francisco San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allyson","family":"Thrall","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Benioff Children's 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