Outcomes for patients with congenital hepatoblastoma: Have they changed since 2013?

Deborah Stephanie Gho; Sara Claire Horton; James Henry Feusner

doi:10.1200/JCO.2025.43.16_suppl.e22019

e22019 Background: Congenital hepatoblastoma (cHB), hepatoblastoma diagnosed in the first month of life, is rare. Some previously reported data in 2013 were quite discrepant. To update the knowledge surrounding cHB and address the discrepancies, we performed an extensive literature review of characteristics and outcomes for cHB cases since that report. Methods: Retrospective literature review was performed using PubMed and EMBASE for reports of cHB from 2013 to present. Data on age, prematurity, gender, duration of follow up, survival, presence of metastatic disease and serum alpha-fetoprotein (AFP) levels were extracted by manual review and compared to the prior report, which included a literature review and a collection of patients known to be congenital from review of major clinical hepatoblastoma trials (Trial data review). Results: A total of 57 cases of cHB were collected in the contemporary review. Age at diagnosis ranged from 0-30 days (median 9 days). Similar to the previous Trial data review, 25% of patients with cHB were born prematurely. The survival in our case series was 88% (follow-up 0.5-108 months, median 24 months), which was equivalent to the survival in the Trial data review series but significantly superior to the 50% survival in the previous literature review. In our cohort of patients, serum AFP was elevated for age in 31% of patients with median AFP of 120,000 ng/mL (1,940-2,900,000 ng/mL). Finally, we found that 13% of patients had metastatic disease at diagnosis. Conclusions: Our review confirms the excellent survival rates of cHB patients, consistent with the prior Trial data review. The presence of metastatic disease remains unchanged from the prior report and is not higher than patients with non-congenital HB. Contrary to what has been thought, our data does not show an increased proportion of preterm infants in this disease group. Here, we provide unique insight into the serum AFP levels at the time of diagnosis and how often they are elevated for age and gestational age. Ref: Trobaugh-Lotrario AD, Chaiyachati BH, Meyers RL, Haberle B, Tomlinson GE, Katzenstein HM, Malogolowkin MH, von Schweinitz D, Krailo M, Feusner JH. Outcomes for patients with congenital hepatoblastoma. Pediatr Blood Cancer. 2013 Nov;60(11):1817-25. doi: 10.1002/pbc.24655. Epub 2013 Jun 24. Age at diagnosis (days) median (range) Preterm (<37 weeks) Gender Survival Duration of follow up (mo) median (range) Serum AFP (ng/mL) median (range) Elevated for age Presence of metastasis Trial data review (N=27) 7 (1-28)n=27 29%n=14 M: 17F: 6 88% n=25 82 (15-230) n=22 335,000 (1,540-1,150,000) n=10 80% n=10 0% n=23 Previous lit review (N=52) 1 (1-28) n=52 ND ND 50% n=44 22.5 (9wks-10.2yrs) n=20 189,000 (9,800-3,200,000) n=27 14.8% n=27 5% n=40 Current Data (N=57) 9 (0-30) n=41 25% n=29 M=27F=30 88% n=35 24 (0.5-108) n=27 120,000 (1,940-2,900,000) n=36 31% n=32 13% n=44 n=evaluable patients. ND=no data.

Outcomes for patients with congenital hepatoblastoma: Have they changed since 2013?

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