SIOPEL-4
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.
Overview
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.
- 79 out of 100 people had no sign of cancer on scans or tests with Dose-dense cisplatin with doxorubicin, surgery, then carboplatin and doxorubicin.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 98 out of 100 people reached this endpoint with Dose-dense cisplatin with doxorubicin.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- 74 out of 100 people had no sign of cancer on scans or tests with Dose-dense cisplatin with doxorubicin, surgery.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- 76 out of 100 people free of a major event at 3 years with SIOPEL-4 regimen.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 83 out of 100 people alive at 3 years with SIOPEL-4 regimen.
- Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
- 71 out of 100 people reached this endpoint with SIOPEL-4 regimen.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- These results apply to the people the trial enrolled: 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. People in a different situation may not see the same effect.
- Only 62 people took part, so the numbers are less certain than in a large trial.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
62 analysed.
95% CI 67 to 88
Source60 of 61 evaluable; 95% CI 91 to 100
Source95% CI 65 to 87; median follow-up 52 months
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Complete remission at the end of treatmentprimary | Dose-dense cisplatin with doxorubicin, surgery, then carboplatin and doxorubicin | 62 | 79% | - | - | link |
| Partial response to pre-operative chemotherapy | Dose-dense cisplatin with doxorubicin | 61 | 98% | - | - | link |
| Complete resection of all tumour lesions | Dose-dense cisplatin with doxorubicin, surgery | 62 | 74% | - | - | link |
| Event-free survival at 3 years | SIOPEL-4 regimen | 62 | 76% | - | - | link |
| Overall survival at 3 years | SIOPEL-4 regimen | 62 | 83% | - | - | link |
| Febrile neutropenia (at least one episode) | SIOPEL-4 regimen | 62 | 71% | - | - | link |
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