OnCo
Learn & contributeTeaching packs

Teaching pack: Hepatoblastoma

Read the page

8 slides generated from the cancer page, with a quiz from the open benchmark and speaker notes that cite the sources. Arrow keys move between slides; Print gives one slide per page.

Slide 1 of 8
  1. Teaching pack · Cancer · paediatric

    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.

    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice1 / 8
  2. What it is

    In two paragraphs

    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).

    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice2 / 8
  3. Standard of care

    What is given today, by setting

    SettingApproachGuideline
    Very low / standard risk (PRETEXT I-III, resectable, AFP >100)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.not mapped
    High risk (metastatic, AFP <100, PRETEXT IV, vascular involvement)Cisplatin-doxorubicin (PLADO) or dose-dense cisplatin (SIOPEL-4), resection of primary and lung metastases; consider C5VD (COG).not mapped
    Unresectable after chemotherapy (POST-TEXT IV, central vascular involvement)Orthotopic liver transplantation (5-year survival ~80%); early referral to a transplant centre.not mapped
    Relapsed/refractoryIrinotecan-based salvage, surgery for isolated recurrence, transplant if liver-confined; trials.not mapped
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice3 / 8
  4. State of the art

    Where the field stands

    • Overall survival ~80-90%; cisplatin alone cures most standard-risk children, and transplant rescues unresectable disease.
    • 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.
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice4 / 8
  5. History

    How we got here

    1. 1967Ishak and Glunz define hepatoblastoma histology
    2. 1980Cisplatin and doxorubicin shown active; survival rises from ~30% to ~70%
    3. 1990SIOPEL-1 introduces PRETEXT staging and pre-operative chemotherapy
    4. 2009SIOPEL-3: cisplatin alone suffices for standard risk (NEJM)
    5. 2013SIOPEL-4: dose-dense cisplatin in high-risk disease (Lancet Oncol)
    6. 2017CHIC international risk stratification (Lancet Oncol)
    7. 2018SIOPEL-6: sodium thiosulfate halves cisplatin hearing loss (NEJM)
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice5 / 8
  6. Pipeline

    What is coming

    • Sodium thiosulfate (otoprotectant) (product)
    • Liver transplantation for cancer (Milan criteria and beyond) (technology)
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice6 / 8
  7. Open problems

    What nobody has solved

    • 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.
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice7 / 8
  8. Sources

    Read the primary sources

    • NCI PDQ: childhood liver cancer: https://www.cancer.gov/types/liver/patient/child-liver-treatment-pdq
    • SIOPEL-6 (NEJM 2018): https://doi.org/10.1056/NEJMoa1801109
    • PHITT trial: https://clinicaltrials.gov/study/NCT03017326
    • Wikipedia: https://en.wikipedia.org/wiki/Hepatoblastoma
    • Guideline: https://doi.org/10.1056/NEJMoa1801109
    Teaching pack: Hepatoblastoma · OnCo, CC BY 4.0 · not medical advice8 / 8