Outcomes of Patients Treated for Hepatoblastoma with Low Alpha-Fetoprotein and/or Small Cell Undifferentiated Histology: A Report from the Children's Hepatic Tumors International Collaboration (CHIC)
Trobaugh-Lotrario, Angela D; Maibach, Rudolf; Aronson, Daniel C; Rangaswami, Arun; Häberle, Beate; O'Neill, Allison F; Schmid, Irene; Ansari, Marc; Hishiki, Tomoro; Ranganathan, Sarangarajan; Alaggio, Rita; de Krijger, Ronald R; Tanaka, Yukichi; Cho, Soo-Jin; Vokuhl, Christian; Maxwell, Rebecca; Krailo, Mark; Hiyama, Eiso; Czauderna, Piotr; Finegold, Milton; Feusner, James H; Malogolowkin, Marcio H; Meyers, Rebecka L; Lopez-Terrada, Dolores
(2023) Cancers, volume 15, issue 2
(Article)
Abstract
Small cell undifferentiated (SCU) histology and alpha-fetoprotein (AFP) levels below 100 ng/mL have been reported as poor prognostic factors in hepatoblastoma (HB); subsequent studies reported SMARCB1 mutations in some SCU HBs confirming the diagnosis of rhabdoid tumor. The Children's Hepatic tumors International Collaboration (CHIC) database was queried for patients with
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HB who had AFP levels less than 100 ng/mL at diagnosis or were historically diagnosed as SCU HBs. Seventy-three of 1605 patients in the CHIC database were originally identified as SCU HB, HB with SCU component, or HB with low AFP levels. Upon retrospective review, they were re-classified as rhabdoid tumors (n = 11), HB with SCU component (n = 41), and HB with low AFP (n = 14). Seven were excluded for erroneously low AFP levels. Overall survival was 0% for patients with rhabdoid tumors, 76% for patients with HB with SCU component, and 64% for patients with HB with AFP less than 100 ng/mL. Patients with HB with SCU component or low AFP should be assessed for SMARCB1 mutations and, if confirmed, treated as rhabdoid tumors. When rhabdoid tumors are excluded, the presence of SCU component and low AFP at diagnosis were not associated with poor prognosis in patients diagnosed with HB.
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Keywords: AFP, Hepatoblastoma, rhabdoid, SCU (small cell undifferentiated), SMARCB1, Oncology, Cancer Research, Journal Article
ISSN: 2072-6694
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
Note: Funding Information: This study was funded by European Network for Cancer Research in Children and Adolescents, Hepatoblastoma Foundation, Cansearch Foundation, Japan Agency for Medical Research and Development, Japan Society for the Promotion of Science, and Swiss Cancer Research. The funding sources had no role in the study design; collection, analysis, and interpretation of data; writing of the report; nor the decision to submit the report for publication. Framework Program-7 European Commission; European Network for Cancer Research in Children and Adolescents, Grant/Award Number: 261474; COG CureSearch grant contributed by the Hepatoblastoma Foundation; Cansearch Foundation; Japan Agency for Medical Research and Development (AMED), Grant/Award Number: 17ck0106332h0001; Japan Society for the Promotion of Science (JSPS KAKENHI), Grant/Award Number: JP16H02778; Swiss Cancer Research, Grant/Award Number: KFS-3936-08-2016. Publisher Copyright: © 2023 by the authors.
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