Table 2.
Cooperative Group Studies of Childhood Hepatoblastoma
| Study | No. of Patients | Treatment | Outcome | Conclusion/Comments |
|---|---|---|---|---|
| Single-Arm Cooperative Group Studies | ||||
| CCG/SWOG study 221 | 62 | V-CTX-DOX-FU | 12 of 27 patients with measurable disease who completed ≥ one 6-week course had ≥ PR; 5-year survival, approximately 38% | Hepatoblastoma is responsive to chemotherapy |
| CCG-823F15 | 33 | CDDP-DOX | 2-year survival, 67% | Preoperative chemotherapy can facilitate complete resection in substantial number of children; patients with unresectable or metastatic disease remain largely incurable |
| POG 869714 | 60 | CDDP-V-FU | 4-year DFS for stage III disease, 67% (SE, 10.8%) | |
| GPOH/HB 8957 | 72 | IFOS-CDDP-DOX | Long-term EFS, 75% | |
| SIOPEL-121 | 154 | CDDP-DOX | 5-year survival, 75% | |
| JPLT-158 | 134 | CDDP-pirarubicin | 3-year survival, 77.8% | |
| SIOPEL-259 | 67 standard-risk patients (PRETEXT I, II, III) | CDDP only | 3-year survival, 91% (± standard deviation, 7%); CDDP alone with surgery may be sufficient for standard-risk patients | |
| GPOH/HB 9417 | 69 | IFOS-CDDP-DOX | 3-year survival, 77% | |
| SIOPEL-422 | 62 | High-risk patients (PRETEXT IV; P+, V+, E+, metastasis, AFP < 100 ng/mL, or tumor rupture); CDDP-DOX-CBDCA | 3-year EFS, 76%; 3-year OS, 83% | Unique dose-dense CDDP regimen may improve survival in patients with unresectable or metastatic disease |
| Randomized Studies | ||||
| POG/CCG INT009816 | 173 | CDDP-V-FU v CDDP-DOX | 5-year survival, 69% v 72%; P = .88 | Combination chemotherapy without doxorubicin is less toxic but results in similar survival |
| SIOPEL 318 | 126 v 129 | CDDP only v CDDP-DOX | 3-year survival, 95% v 93% | DOX can be safely omitted from treatment of standard-risk patients |
Abbreviations: CBDCA, carboplatin; CCG, Children's Cancer Group; CDDP, cisplatin; COG, Children's Oncology Group; CTX, cyclophosphamide; DOX, doxorubicin; E+, extrahepatic abdominal disease; EFS, event-free survival; FU, fluorouracil; GPOH, German Society for Pediatric Oncology; HB, hepatoblastoma; IFOS, ifosfamide; JPLT, Japanese Study Group for Pediatric Liver Tumors; P+, portal vein involvement; POG, Pediatric Oncology Group; PRETEXT, pretreatment extent of disease; SIOPEL, International Childhood Liver Tumors Strategy Group; V, vincristine; V+, vascular invasion.