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)

Angela D Trobaugh-Lotrario; Rudolf Maibach; Daniel C Aronson; Arun Rangaswami; Beate Häberle; Allison F O'Neill; Irene Schmid; Marc Ansari; Tomoro Hishiki; Sarangarajan Ranganathan; Rita Alaggio; Ronald R de Krijger; Yukichi Tanaka; Soo-Jin Cho; Christian Vokuhl; Rebecca Maxwell; Mark Krailo; Eiso Hiyama; Piotr Czauderna; Milton Finegold; James H Feusner; Marcio H Malogolowkin; Rebecka L Meyers; Dolores Lopez-Terrada

doi:10.3390/cancers15020467

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)

Cancers (Basel). 2023 Jan 11;15(2):467. doi: 10.3390/cancers15020467.

Authors

Angela D Trobaugh-Lotrario¹, Rudolf Maibach², Daniel C Aronson³, Arun Rangaswami⁴, Beate Häberle⁵, Allison F O'Neill⁶, Irene Schmid⁷, Marc Ansari⁸, Tomoro Hishiki⁹, Sarangarajan Ranganathan¹⁰, Rita Alaggio¹¹, Ronald R de Krijger^{12

13}, Yukichi Tanaka¹⁴, Soo-Jin Cho¹⁵, Christian Vokuhl¹⁶, Rebecca Maxwell⁵, Mark Krailo¹⁷, Eiso Hiyama¹⁸, Piotr Czauderna¹⁹, Milton Finegold²⁰, James H Feusner²¹, Marcio H Malogolowkin²², Rebecka L Meyers²³, Dolores Lopez-Terrada²⁰

Affiliations

¹ Department of Pediatric Hematology/Oncology, Providence Sacred Heart Children's Hospital, Spokane, WA 99204, USA.
² IBCSG Coordinating Center, 3008 Bern, Switzerland.
³ Department of Pediatric Surgery, University Children's Hospital Zurich, 8032 Zurich, Switzerland.
⁴ Division of Pediatric Hematology and Oncology, University of California, San Francisco, CA 94143, USA.
⁵ Division of Pediatric Surgery, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, 80337 Munich, Germany.
⁶ Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston Children's Hospital and Harvard Medical School, Boston, MA 02215, USA.
⁷ Department of Pediatric Hematology and Oncology, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, 80337 Munich, Germany.
⁸ Pediatric Department, Onco-Hematology Unit, University of Geneva Hospitals, 1211 Geneva, Switzerland.
⁹ Department of Pediatric Surgery, Chiba University Graduate School of Medicine, Chiba 260-8677, Japan.
¹⁰ Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
¹¹ Department of Pathology, Bambino Gesu Children's Hospital, 00165 Rome, Italy.
¹² Department of Pathology, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, The Netherlands.
¹³ Laboratory for Childhood Cancer Pathology, Princess Maxima Center for Pediatric Oncology, Heidelberglaan 25, 3584 CS Utrecht, The Netherlands.
¹⁴ Department of Pathology, Kanagawa Children's Medical Center, Yokohama 232-0066, Japan.
¹⁵ Department of Pathology, University of California, San Francisco, CA 94143, USA.
¹⁶ Institute of Pathology, University Hospital Bonn, 53127 Bonn, Germany.
¹⁷ Department of Population and Public Health Sciences, University of Southern California, Los Angeles, CA 90032, USA.
¹⁸ Department of Pediatric Surgery, Hiroshima University, Hiroshima 734-8551, Japan.
¹⁹ Department of Surgery for Children and Adolescents, Medical University of Gdansk, 80-210 Gdansk, Poland.
²⁰ Department of Pathology and Immunology, Baylor College of Medicine Houston, TX 77030, USA.
²¹ Division of Hematology/Oncology, Children's Hospital & Research Center Oakland, Oakland, CA 94618, USA.
²² Division of Pediatric Hematology Oncology, University of California-Davis, Davis, CA 95817, USA.
²³ Division of Pediatric Surgery, Primary Children's Hospital, Salt Lake City, UT 84113, USA.

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 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.

Keywords: AFP; Hepatoblastoma; SCU (small cell undifferentiated); SMARCB1; rhabdoid.

Abstract

Grants and funding