Cancer · 2026
The landscape of pediatric hepatocellular carcinoma and fibrolamellar carcinoma: A report from the Children's Hepatic tumors International Collaboration (CHIC).
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O'Neill Allison F, Maibach Rudolf, Krailo Mark D, Piao Jin, Lopez-Terrada Dolores, Alaggio Rita, Tanaka Yukichi, Häberle Beate, Aronson Daniel C, Hiyama Eiso, Vokuhl Christian, Schmid Irene, Malogolowkin Marcio H, Meyers Rebecka L, Czauderna Piotr, Rangaswami Arun
Аннотация
BACKGROUND: Pediatric hepatocellular carcinoma (HCC) and fibrolamellar carcinoma (FLC) have historically been studied on prospective trials in aggregate with hepatoblastoma (HB). It is now recognized that both HCC and FLC have unique histologies, characteristic genomics, and a more aggressive clinical course than HB. It is anticipated that data analyzed from patients treated on previously conducted international trials will inform current and future approaches to the study of these rare diseases.
METHODS: The Children's Hepatic tumors International Collaboration houses data from clinical trials conducted by three global consortia as well as international registries. Data from patients with HCC or FLC who were enrolled on historic trials designed for patients with HB were analyzed for patient demographics, disease characteristics, treatment, and outcomes.
RESULTS: The authors identified 236 patients with pediatric HCC or FLC who were enrolled on trials conducted in the United States, Europe, and Japan. As anticipated, HCC and FLC predominantly affected adolescent patients, and outcomes were generally related to extent of disease and resectability. Independent of histology, upfront resection (p = .004) predicted superior survival, likely reflecting the inadequacies of systemic agents to control disease. Outcomes were comparable across studies, with a 5-year survival rate of 36%, independent of chemotherapy received. Survival was equivalent for patients with HCC and FLC after 7-year follow-up.
CONCLUSIONS: Treatment of pediatric HCC and FLC remains challenging. The current results support risk-stratification methods currently under study prospectively and the independent study of these tumors in the future.
METHODS: The Children's Hepatic tumors International Collaboration houses data from clinical trials conducted by three global consortia as well as international registries. Data from patients with HCC or FLC who were enrolled on historic trials designed for patients with HB were analyzed for patient demographics, disease characteristics, treatment, and outcomes.
RESULTS: The authors identified 236 patients with pediatric HCC or FLC who were enrolled on trials conducted in the United States, Europe, and Japan. As anticipated, HCC and FLC predominantly affected adolescent patients, and outcomes were generally related to extent of disease and resectability. Independent of histology, upfront resection (p = .004) predicted superior survival, likely reflecting the inadequacies of systemic agents to control disease. Outcomes were comparable across studies, with a 5-year survival rate of 36%, independent of chemotherapy received. Survival was equivalent for patients with HCC and FLC after 7-year follow-up.
CONCLUSIONS: Treatment of pediatric HCC and FLC remains challenging. The current results support risk-stratification methods currently under study prospectively and the independent study of these tumors in the future.