Childhood hepatoblastoma: minimal adjuvant chemotherapy yields good outcomes

  • Katzenstein HM & al.
  • Lancet Oncol
  • 08.04.2019

  • von Jim Kling
  • Studien – kurz & knapp
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Takeaway

  • 2 cycles of cisplatin, fluorouracil, and vincristine yielded good disease control in patients with childhood hepatoblastoma who were considered resectable and at low risk during diagnosis.  
  • Some patients had tumor characteristics suggesting they were not at low risk, calling into question whether surgeons are following resection guidelines. This makes it harder to assess when patients can benefit from immediate surgery, according to an accompanying editorial.

Why this matters

  • Standard treatment among these patients is 4-6 cycles. Reducing the exposure in selected patients could reduce acute and long-term toxicities.

Study design

  • Subanalysis of the low-risk group of a phase 3 clinical trial of risk-based therapy in childhood hepatoblastoma (n=51).
  • Funding: NIH.

Key results

  • At 1 year, 47 of 49 evaluable patients (96%) were recurrence-free, exceeding the 45-patient threshold designated to warrant further investigation.
  • 4-year recurrence-free survival, 92% (95% CI, 79%-97%); OS, 95% (95% CI, 82%-99%).
  • 5-year recurrence-free survival, 88% (95% CI, 72%-95%); OS, 91% (95% CI, 75%-97%).
  • The most common grade 3-4 adverse event was febrile neutropenia (14%); 8% experienced grade 3 infection, 8% experienced diarrhea, and 6% had decreased neutrophil counts.
    • Other adverse events included peripheral neuropathy (4%) and grade 3 hearing impairment (2%).

Limitations

  • Small sample size.
  • No control.