Hepatoblastoma is the most common malignant liver tumor in infants and preschool children, comprising 65% of pediatric liver malignancies in those under 15, with its incidence on the rise in recent years \[1\]. Standard therapy combines surgical resection and chemotherapy: early-stage patients boast a survival rate over 90%, yet high-risk cases only reach around 40%, highlighting unmet treatment needs.
Notably, there is no universal definition for high-risk hepatoblastoma. The U.S. COG (AHEP0731) categorizes it as stage 4 disease, AFP \<100ng/ml at diagnosis (any stage), or small cell undifferentiated histology; conversely, SIOP includes factors like major vascular invasion (inferior vena cava/portal vein), intra-abdominal extrahepatic spread, distant metastasis, AFP \<100ng/ml, or tumor rupture, regardless of PRETEXT stage. To improve outcomes, international teams have tested intensified chemotherapy: Europe's SIOPEL reported that escalated cisplatin-doxorubicin regimens lifted high-risk patients' 3-year overall survival to over 80% \[2\], though with heightened toxicity. Similarly, Germany's IPA (ifosfamide-cisplatin-doxorubicin) and Japan's ITEC (ifosfamide-doxorubicin-carboplatin-VP-16) regimens delivered significant survival benefits but also amplified side effects \[3,4\].
Against this backdrop, the Guangdong Anti-Cancer Association's Pediatric Oncology Committee, led by Sun Yat-sen University Cancer Center and involving 15 hospitals, is launching a multicenter prospective trial to identify optimal chemotherapy regimens for Chinese hepatoblastoma children.
Parallelly, liquid biopsy has become an oncology research priority, offering four core advantages over tissue biopsy: non-invasiveness (peripheral blood sampling avoids tumor seeding), real-time genetic/progression monitoring (eliminating repeated invasive procedures), comprehensive molecular profiling (overcoming intratumoral heterogeneity), and high accuracy (capturing primary tumor-derived data). Given hepatoblastoma's propensity for early distant metastasis and 30-40% advanced-stage survival (with limited late-stage chemo efficacy), the Nano-5hmC-Seal cfDNA epigenetic profiling method holds promise as a novel biomarker for early diagnosis, treatment prediction, recurrence monitoring, and prognosis assessment in this disease.
Patients with primary hepatoblastoma confirmed by pathology.
The age of the subjects was less than 18 years old. ③ Obtain the informed consent from the guardians and sign the informed consent form
Recurrent hepatoblastoma or other malignant tumor.
Age> 18 years old. ③ Patients with other tumors and received chemotherapy and abdominal radiotherapy.
Heart, brain and kidney failure patients.
Parallel
Very low-risk group
DrugVery low-risk group: No chemotherapy after pure fetal type surgery, followed up and observed. Chemotherapy with other types of single-agent DDP regimens for 4 courses.
Low-risk group
DrugLow-risk group: 2 to 4 courses of DDP monotherapy chemotherapy, elective surgery, 2 courses of DDP monotherapy chemotherapy after surgery, totaling 4 to 6 courses.
Intermediate-risk group
DrugIntermediate risk group: randomized treatment: group A SIOPEL - 3 HR solutions can be 2 \~ 4 course of chemotherapy, continue to A total of 6 course of chemotherapy after surgery. Group B C5VD solution after 2 \~ 4 course of chemotherapy, elective surgical procedures continue to a total of 6 course of chemotherapy after surgery.
High-risk group
DrugHigh-risk group：Chemotherapy with the C-CD+ICE+ sorafenib regimen for 3 to 5 courses was followed by elective surgery, with a total of 6 to 7 courses
5hmc dynamic monitoring
Other interventionPeripheral blood was regularly drawn from patients in the very low-risk group, low-risk group, intermediate-risk group and high-risk group for 5hmc dynamic monitoring to evaluate its value in clinical efficacy and recurrence detection
PFS
progression of the disease or the death
OS
Overall survival (OS) is defined as the time from the initiation of treatment with pucotenlimab in combination with theLenvatinib and Irinotecan regimen until the patient's death from any cause. Death is determined based on official medical records or other reliable documentation. The follow - up for OS will continue throughout this estimated assessment period.
Consistency
The consistency of 5hmc dynamic monitoring in the evaluation of therapeutic effect and detection of tumor recurrence in hepatoblastoma in each risk group was analyzed.
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