Hepatoblastoma
A liver cancer of toddlers that is highly curable with cisplatin chemotherapy and surgery, including liver transplant when the tumour cannot be cut out. A recent success was proving that a simple antidote prevents the permanent hearing loss cisplatin causes.
Hepatoblastoma is an embryonal liver tumour with CTNNB1 (β-catenin) mutations in most cases, associated with prematurity, Beckwith-Wiedemann syndrome and familial adenomatous polyposis. Alpha-fetoprotein (AFP) is elevated in >90% and tracks response; very low AFP (<100 ng/mL) marks an aggressive small-cell-undifferentiated variant. Staging uses PRETEXT (extent within the liver) plus annotation factors (vascular involvement, extrahepatic extension, metastases, rupture) under the international CHIC risk stratification.
Treatment combines cisplatin-based chemotherapy with complete surgical resection; standard-risk disease is cured in ~90% with cisplatin monotherapy (SIOPEL-3), high-risk disease uses cisplatin-doxorubicin (PLADO) or dose-dense cisplatin (SIOPEL-4), and unresectable tumours confined to the liver are transplanted with excellent outcomes. The Paediatric Hepatic International Tumour Trial (PHITT) harmonises COG, SIOPEL and JPLT approaches. Sodium thiosulfate given 6 hours after cisplatin halves permanent hearing loss without compromising survival (SIOPEL-6, NEJM 2018), leading to FDA approval of the first otoprotectant (2022).
State of the art today
- Sodium thiosulfate is a model of supportive-care evidence: a randomised trial in children changed a global standard and produced a drug approval.
- PHITT is the first global paediatric liver tumour trial with harmonised risk groups.
- Low-AFP small-cell undifferentiated tumours and metastatic disease remain the challenging minority.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- Overall survival ~80-90%; cisplatin alone cures most standard-risk children, and transplant rescues unresectable disease.
Show survival figures (1)
Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
- Hepatoblastoma is the most common childhood liver cancer (~1-2 per million children per year, ~100-150 cases per year in the US), mostly under age 3; incidence rising with survival of very-low-birth-weight infants.
Where the cases are
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
Upfront resection for PRETEXT I-II (COG) or cisplatin monotherapy ×4-6 with delayed resection (SIOPEL-3); sodium thiosulfate after each cisplatin dose for otoprotection.
Cisplatin-doxorubicin (PLADO) or dose-dense cisplatin (SIOPEL-4), resection of primary and lung metastases; consider C5VD (COG).
Orthotopic liver transplantation (5-year survival ~80%); early referral to a transplant centre.
Irinotecan-based salvage, surgery for isolated recurrence, transplant if liver-confined; trials.
Subtypes & biomarkers
top- Epithelial : fetal (well-differentiated, favourable), embryonal, macrotrabecular
- Small-cell undifferentiated (low AFP, aggressive; exclude rhabdoid/INI1 loss)
- Mixed epithelial-mesenchymal (with or without teratoid features)
- Hepatocellular neoplasm NOS / transitional liver cell tumour (older children)
- Serum AFP (diagnosis, response, relapse; <100 ng/mL poor)
- PRETEXT / POST-TEXT group and annotation factors (V, P, E, M, R)
- CHIC risk group (age, AFP, PRETEXT, metastases)
- CTNNB1 mutation, NFE2L2, TERT
- Germline APC (FAP) and 11p15 (Beckwith-Wiedemann)
Target prevalence in this cancer
- 1967Ishak and Glunz define hepatoblastoma histology
- 1980Cisplatin and doxorubicin shown active; survival rises from ~30% to ~70%
- 1990SIOPEL-1 introduces PRETEXT staging and pre-operative chemotherapy
- 2009SIOPEL-3: cisplatin alone suffices for standard risk (NEJM)
- 2013SIOPEL-4: dose-dense cisplatin in high-risk disease (Lancet Oncol)
- 2017CHIC international risk stratification (Lancet Oncol)
- 2018SIOPEL-6: sodium thiosulfate halves cisplatin hearing loss (NEJM)
FDA approval of sodium thiosulfate (Pedmark) 2022.
Open problems
- Metastatic and low-AFP disease.
- Long-term effects of cisplatin (hearing, kidney) and doxorubicin (heart).
- Rising incidence with extreme prematurity.
- Transplant organ availability and lifelong immunosuppression in children.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Expert centres
topCentres linked to this cancer in OnCo
- via Liver transplantation for cancer (Milan criteria and beyond)
- via Liver transplantation for cancer (Milan criteria and beyond)
- Children's Oncology Group (COG)Monrovia, CA, USvia this cancer
- Indiana University Melvin and Bren Simon Comprehensive Cancer CenterIndianapolis, IN, USNCI comprehensivevia Cisplatin
Questions to ask
topQuestions to ask your oncologist about Hepatoblastoma
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example Serum AFP, PRETEXT / POST-TEXT group and annotation factors, CHIC risk group, CTNNB1 mutation, NFE2L2, TERT, Germline APCand 11p15), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include Epithelial: fetal, embryonal, macrotrabecular, Small-cell undifferentiated, Mixed epithelial-mesenchymal.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Very low / standard risk (PRETEXT I-III, resectable, AFP >100)
- For my situation (very low / standard risk (pretext i-iii, resectable, afp >100)), which of the standard options do you recommend and why?Why: Guideline options include: Upfront resection for PRETEXT I-II (COG) or cisplatin monotherapy ×4-6 with delayed resection (SIOPEL-3); sodium thiosulfate after each cisplatin dose for otoprotection.
- Am I a candidate for Cisplatin, Sodium thiosulfate (otoprotectant), and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
High risk (metastatic, AFP <100, PRETEXT IV, vascular involvement)
- For my situation (high risk (metastatic, afp <100, pretext iv, vascular involvement)), which of the standard options do you recommend and why?Why: Guideline options include: Cisplatin-doxorubicin (PLADO) or dose-dense cisplatin (SIOPEL-4), resection of primary and lung metastases; consider C5VD (COG).
- Am I a candidate for Cisplatin, Doxorubicin, Vincristine or related drugs, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Unresectable after chemotherapy (POST-TEXT IV, central vascular involvement)
- For my situation (unresectable after chemotherapy (post-text iv, central vascular involvement)), which of the standard options do you recommend and why?Why: Guideline options include: Orthotopic liver transplantation (5-year survival ~80%); early referral to a transplant centre.
Relapsed/refractory
- For my situation (relapsed/refractory), which of the standard options do you recommend and why?Why: Guideline options include: Irinotecan-based salvage, surgery for isolated recurrence, transplant if liver-confined; trials.
- Am I a candidate for Irinotecan (and liposomal irinotecan), and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Sodium thiosulfate (otoprotectant), Liver transplantation for cancer (Milan criteria and beyond)?Why: Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “Metastatic and low-AFP disease”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Long-term effects of cisplatin (hearing, kidney) and doxorubicin (heart)”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
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Direct links plus the targets, companies, and technologies of this cancer's products.
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topQuery for this cancer: (TITLE:"Hepatoblastoma" OR ABSTRACT:"Hepatoblastoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Hepatoblastoma, not a curated reading list.
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