# SIOPEL-4

Source: https://onco.cc/trials/siopel-4/  
OnCo record `siopel-4` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

SIOPEL-4 showed that giving cisplatin every week in a dense schedule, with doxorubicin, to children with high-risk hepatoblastoma got nearly all of them to respond and three quarters to complete resection, lifting three-year survival to 83 percent in a group that used to do poorly.

## Summary

SIOPEL-4 was a prospective single-arm feasibility study of the SIOPEL group in 62 children with high-risk hepatoblastoma, 39 of them with lung metastases. Pre-operative treatment was dose-dense cisplatin (80 mg/m2 then 70 mg/m2 weekly) with doxorubicin over three cycles, followed by removal of all remaining tumour, including liver transplantation and metastasectomy where needed; patients still unresectable had further doxorubicin and carboplatin, and the rest received post-operative carboplatin and doxorubicin. The primary endpoint was complete remission at the end of treatment.

60 of 61 evaluable patients responded to pre-operative chemotherapy, complete resection was achieved in 74 percent and 79 percent were in complete remission at the end of therapy; three-year event-free survival was 76 percent and overall survival 83 percent. Nearly every patient had grade 3 or 4 haematological toxicity and 71 percent had febrile neutropenia. Dose-dense cisplatin is the high-risk regimen on the corpus's hepatoblastoma page.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-22
- Also known as: SIOPEL 4; SIOPEL4; SIOP-SIOPEL-4
- Tags: soc-trials
- Registry id: NCT00077389
- Phase: 2
- Setting: High-risk hepatoblastoma in children up to 18 (metastases, all liver sectors involved, extrahepatic abdominal disease, major vascular invasion, low alpha-fetoprotein or tumour rupture): dose-dense weekly cisplatin with doxorubicin before surgery, including liver transplantation and metastasectomy where needed, then carboplatin and doxorubicin after surgery
- Sponsor: University of Leicester
- Enrolled: 62
- Result: Complete remission at the end of therapy in 79 percent of 62 high-risk patients; complete resection 74 percent; three-year event-free survival 76 percent and overall survival 83 percent.
- Outcomes: Complete remission at the end of treatment: Dose-dense cisplatin with doxorubicin, surgery, then carboplatin and doxorubicin 79%; Partial response to pre-operative chemotherapy: Dose-dense cisplatin with doxorubicin 98%; Complete resection of all tumour lesions: Dose-dense cisplatin with doxorubicin, surgery 74%; Event-free survival at 3 years: SIOPEL-4 regimen 76%; Overall survival at 3 years: SIOPEL-4 regimen 83%; Febrile neutropenia (at least one episode): SIOPEL-4 regimen 71%

## Sources

- ClinicalTrials.gov NCT00077389: https://clinicaltrials.gov/study/NCT00077389

## Connected records

- cancers: [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Hepatoblastoma](https://onco.cc/cancers/hepatoblastoma/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/)
- drugs: [Carboplatin](https://onco.cc/drugs/carboplatin/), [Cisplatin](https://onco.cc/drugs/cisplatin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/)
- key papers: [SIOPEL-4: dose-dense cisplatin-based chemotherapy and surgery for high-risk hepatoblastoma](https://onco.cc/key-papers/paper-siopel-4-dose-dense-cisplatin-high-risk-hepatoblastoma-zsiros-lancet-oncol-2013/)

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