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Treatment of Hepatoblastoma
Source: Childhood Liver Cancer Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: January 6, 2025 · Captured 2026-09-09.
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Treatment options for newly diagnosed hepatoblastoma depend on the following:
Whether the cancer is resectable at diagnosis.
The tumor histology.
How the cancer responds to chemotherapy.
Whether the cancer has metastasized.
Cisplatin-based chemotherapy has resulted in a survival rate of more than 90% for children with PRETEXT and POST-Treatment EXTent (POSTTEXT) group I and II resectable disease before or after chemotherapy.[54,56,67]
Chemotherapy regimens used in the treatment of hepatoblastoma and their respective outcomes are described in Table 6. For information describing each stage, see the Tumor Stratification by Imaging section.
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| Study | Chemotherapy Regimen | Number of Patients | Outcomes |
|---|---|---|---|
| AFP = alpha-fetoprotein; C5V = cisplatin, fluorouracil (5-FU), and vincristine; CARBO = carboplatin; CCG = Children’s Cancer Group; CDDP = cisplatin; CITA = pirarubicin-cisplatin; COG = Children's Oncology Group; DOXO = doxorubicin; EFS = event-free survival; GPOH = Gesellschaft für Pädiatrische Onkologie und Hämatologie (Society for Paediatric Oncology and Haematology); H+ = rupture or intraperitoneal hemorrhage; HR = high risk; IFOS = ifosfamide; IPA = ifosfamide, cisplatin, and doxorubicin; ITEC = ifosfamide, pirarubicin, etoposide, and carboplatin; JPLT = Japanese Study Group for Pediatric Liver Tumor; LR = low risk; NR = not reported; OS = overall survival; PLADO = cisplatin and doxorubicin; POG = Pediatric Oncology Group; PRETEXT = PRE-Treatment EXTent of disease; SIOPEL = International Childhood Liver Tumors Strategy Group; SR = standard risk; SUPERPLADO = cisplatin, doxorubicin, and carboplatin; THP = tetrahydropyranyl-adriamycin (pirarubicin); VP = vinorelbine and cisplatin; VPE+ = venous, portal, and extrahepatic involvement; VP16 = etoposide. | |||
| aAdapted from Czauderna et al.,[79] Meyers et al.,[91] and Malogolowkin et al.[92] | |||
| bStudy closed early because of inferior results in the CDDP/CARBO arm. | |||
| INT0098 (CCG/POG) 1989–1992 | C5V vs. CDDP/DOXO | Stage I/II: 50 | 4-Year EFS/OS: |
| I/II = 88%/100% vs. 96%/96% | |||
| Stage III: 83 | III = 60%/68% vs. 68%/71% | ||
| Stage IV: 40 | IV = 14%/33% vs. 37%/42% | ||
| P9645 (COG)b 1999–2002 | C5V vs. CDDP/CARBO | Stage III: 38 | 3-year EFS/OS: |
| III/IV: C5V = 60%/74%; CDDP/CARBO = 38%/54% | |||
| Stage IV: 50 | |||
| AHEP0731 (COG) 2010–2014 [93][Level of evidence C1] | LR: C5V (2 cycles) | LR (stage I/II): 49 | 5-year EFS: 88%; 5-year OS: 91% |
| HB 94 (GPOH) 1994–1997 | I/II: IFOS/CDDP/DOXO | Stage I: 27 | 4-Year EFS/OS: |
| I = 89%/96% | |||
| Stage II: 3 | II = 100%/100% | ||
| III/IV: IFOS/CDDP/DOXO + VP/CARBO | Stage III: 25 | III = 68%/76% | |
| Stage IV: 14 | IV = 21%/36% | ||
| HB 99 (GPOH) 1999–2004 | SR: IPA | SR: 58 | 3-Year EFS/OS: |
| SR = 90%/88% | |||
| HR: CARBO/VP16 | HR: 42 | HR = 52%/55% | |
| SIOPEL-2 1994–1998 | SR: PLADO | PRETEXT I: 6 | 3-Year EFS/OS: |
| SR: 73%/91% | |||
| PRETEXT II: 36 | |||
| PRETEXT III: 25 | |||
| HR: CDDP/CARBO/DOXO | PRETEXT IV: 21 | HR: IV = 48%/61% | |
| Metastases: 25 | HR: metastases = 36%/44% | ||
| SIOPEL-3 1998–2006 | SR: CDDP vs. PLADO | SR: PRETEXT I: 18 | 3-Year EFS/OS: |
| SR: CDDP = 83%/95%; PLADO = 85%/93% | |||
| PRETEXT II: 133 | |||
| PRETEXT III: 104 | |||
| HR: SUPERPLADO | HR: PRETEXT IV: 74 | HR: Overall = 65%/69% | |
| VPE+: 70 | |||
| Metastases: 70 | Metastases = 57%/63% | ||
| AFP <100 ng/mL: 12 | |||
| SIOPEL-4 2005–2009 | HR: Block A: Weekly; CDDP/3 weekly DOXO; Block B: CARBO/DOXO | PRETEXT I: 2 | 3-Year EFS/OS: |
| All HR = 76%/83% | |||
| PRETEXT II: 17 | |||
| PRETEXT III: 27 | |||
| PRETEXT IV: 16 | HR: IV = 75%/88% | ||
| Metastases: 39 | HR: Metastases = 77%/79% | ||
| JPLT-1 1991–1999 | I/II: CDDP(30)/THP-DOXO | Stage I: 9 | 5-Year EFS/OS: |
| I = NR/100% | |||
| Stage II: 32 | II = NR/76% | ||
| III/IV: CDDP(60)/THP-DOXO | Stage IIIa: 48 | IIIa = NR/50% | |
| Stage IIIb: 25 | IIIb = NR/64% | ||
| Stage IV: 20 | IV = NR/77% | ||
| JPLT-2 1999–2010 [94][Level of evidence C1] | Initial surgery and 2 cycles of CITA | Stratum 1: PRETEXT I/II, 0 annotation factors except H+ (n = 40) | 5-Year EFS/OS: |
| 74.2%/89.9% | |||
| 2 cycles of CITA followed by surgery and 2–4 cycles of CITA | Stratum 2: PRETEXT II with multifocality (n = 80) | 84.8%/90.8% | |
| 2 cycles of CITA followed by 2 cycles of CITA (responders); attempted surgery including transplant | Stratum 3: PRETEXT I/II (annotation factors present) and III/IV (n = 176) responders | 71.6%/85.9% | |
| 2 cycles of CITA followed by 2 cycles of ITEC (nonresponders); attempted surgery including transplant | Stratum 4: PRETEXT I/II (annotation factors present) and III/IV (n = 59) nonresponders | 59.1%/67.3% | |
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