This retrospective cohort study evaluated the efficacy of chemotherapy regimens of differing intensities in children with high‑risk hepatoblastoma (HB) treated at a single tertiary center. The analysis included 99 patients treated at Sun Yat‑sen University Cancer Center between December 2004 and July 2024. Short‑term tumor responses, adverse effects, and long‑term survival were compared across treatment groups.
Ninety‑nine patients younger than 18 years with high‑risk HB were included. Patients were assigned to one of three regimen groups based on the treating team's choice over the study period: PLADO (n = 27), C5VD/IIV (n = 29), and modified SIOPEL‑4 (n = 43). The study report provides aggregate numbers per group; detailed baseline characteristics by group and selection criteria for each regimen were not reported in the abstract.
Objective response rates (ORR) among evaluable patients were reported as follows: PLADO 73.9% (17/23), C5VD/IIV 70.8% (17/24), and modified SIOPEL‑4 88.6% (31/35). The difference in ORR among the three regimens was not statistically significant (P = 0.190).
Disease control rates (DCR), however, differed: PLADO 82.6% (19/23), C5VD/IIV 83.3% (20/24), and modified SIOPEL‑4 100% (35/35). The modified SIOPEL‑4 group achieved a significantly higher DCR compared with the other two regimens (P < 0.05).
Three‑year progression‑free survival (PFS) and overall survival (OS) rates reported by regimen were:
The modified SIOPEL‑4 regimen demonstrated significantly better PFS compared with the PLADO and C5VD/IIV regimens (P < 0.05). When the PLADO and C5VD/IIV groups were combined and compared to modified SIOPEL‑4, the modified SIOPEL‑4 group showed significantly superior PFS and OS (both P < 0.05).
The abstract indicates that adverse effects were compared among the three groups, but it does not report detailed rates or severity grades for specific toxicities in the summary. The modified SIOPEL‑4 regimen omitted the day‑15 high‑dose cisplatin, a protocol modification likely intended to adjust toxicity, but full toxicity data and comparative tolerability metrics were not provided in the abstract.
The investigators conclude that the modified SIOPEL‑4 regimen achieved superior disease control and longer PFS compared with PLADO and C5VD/IIV in children with high‑risk HB, and that overall survival was also better when comparing modified SIOPEL‑4 to the combined alternative regimens. They note that despite these improvements, further enhancement of outcomes is needed. The authors recommend intensifying supportive care measures and pursuing aggressive local therapies; specifically, they advocate consideration of upfront liver transplantation for patients whose primary tumor remains unresectable after preoperative chemotherapy.
As presented in the abstract, several methodological and reporting details are limited or not included: granular baseline patient characteristics by regimen, criteria guiding regimen selection, complete adverse event tables with grades and frequencies, and specific statistical methods used for comparisons. The summary does not provide subgroup analyses or multivariable adjustment for potential confounders. These details were not reported in the abstract and would require consultation of the full text for comprehensive assessment.
Conflict of interest statement: all authors declared no conflicts of interest.
Overall, in this single‑center retrospective cohort, the modified SIOPEL‑4 regimen was associated with higher disease control and improved short‑ and mid‑term survival endpoints compared with PLADO and C5VD/IIV for high‑risk pediatric hepatoblastoma, while recognizing the need for further therapeutic optimization and enhanced supportive and local treatment strategies.