Hepatoblastoma

Section editor
	Wayne H. Liang, MD, MS, FAMIA UAB Birmingham, AL LinkedIn WayneLiangMD

7 regimens on this page 7 variants on this page

All lines of therapy

Cisplatin monotherapy

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Regimen

Study	Years of enrollment	Evidence	Comparator	Comparative Efficacy
Perilongo et al. 2009 (SIOPEL 3)	1998-2006	Phase III (E-de-esc)	Cisplatin & Doxorubicin	Non-inferior complete resection rate

Chemotherapy

• Cisplatin (Platinol)

References

1. SIOPEL 3: Perilongo G, Maibach R, Shafford E, Brugieres L, Brock P, Morland B, de Camargo B, Zsiros J, Roebuck D, Zimmermann A, Aronson D, Childs M, Widing E, Laithier V, Plaschkes J, Pritchard J, Scopinaro M, MacKinlay G, Czauderna P. Cisplatin versus cisplatin plus doxorubicin for standard-risk hepatoblastoma. N Engl J Med. 2009 Oct 22;361(17):1662-70. link to original article PubMed NCT00003912

Cisplatin & Doxorubicin

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PLADO: PLAtinol (Cisplatin) & DOxorubicin

Regimen

Study	Years of enrollment	Evidence	Comparator	Comparative Efficacy	Comparative Toxicity
Ortega et al. 2000	1989-1992	Phase III (E-switch-ic)	Cisplatin, 5-FU, Vincristine		More toxic
Pritchard et al. 2000 (SIOPEL 1)	1990-1994	Non-randomized
Perilongo et al. 2009 (SIOPEL 3)	1998-2006	Phase III (C)	Cisplatin	Non-inferior complete resection rate

Chemotherapy

• Cisplatin (Platinol)
• Doxorubicin (Adriamycin)

References

1. Ortega JA, Douglass EC, Feusner JH, Reynolds M, Quinn JJ, Finegold MJ, Haas JE, King DR, Liu-Mares W, Sensel MG, Krailo MD; Children's Cancer Group; Pediatric Oncology Group. Randomized comparison of cisplatin/vincristine/fluorouracil and cisplatin/continuous infusion doxorubicin for treatment of pediatric hepatoblastoma: a report from the Children's Cancer Group and the Pediatric Oncology Group. J Clin Oncol. 2000 Jul;18(14):2665-75. link to original article PubMed
2. SIOPEL 1: Pritchard J, Brown J, Shafford E, Perilongo G, Brock P, Dicks-Mireaux C, Keeling J, Phillips A, Vos A, Plaschkes J. Cisplatin, doxorubicin, and delayed surgery for childhood hepatoblastoma: a successful approach--results of the first prospective study of the International Society of Pediatric Oncology. J Clin Oncol. 2000 Nov 15;18(22):3819-28. link to original article PubMed
3. SIOPEL 3: Perilongo G, Maibach R, Shafford E, Brugieres L, Brock P, Morland B, de Camargo B, Zsiros J, Roebuck D, Zimmermann A, Aronson D, Childs M, Widing E, Laithier V, Plaschkes J, Pritchard J, Scopinaro M, MacKinlay G, Czauderna P. Cisplatin versus cisplatin plus doxorubicin for standard-risk hepatoblastoma. N Engl J Med. 2009 Oct 22;361(17):1662-70. link to original article PubMed NCT00003912

Cisplatin & Fluorouracil (CF) & Vincristine

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Regimen

Study	Years of enrollment	Evidence	Comparator	Comparative Efficacy
Katzenstein et al. 2019 (AHEP0731)	2010-2014	Phase III (E-esc)	Observation	Seems to have superior EFS

For stage 1 or 2 hepatoblastoma without 100% pure fetal stage I or small-cell undifferentiated histology with complete resection at diagnosis and no history of prior chemotherapy or liver directed therapy. Eligible patients include: patients< 21 years old, elevated serum α-fetoprotein level (>100 ng/mL), at least 50% Karnofsky (patients >16 years) or Lansky (patients ≤16 years) performance status.

Preceding treatment

• Curative surgical resection

Chemotherapy

• Cisplatin as follows:
  • Less than 10 kg: 3.3 mg/kg IV over 6 hours once on day 1
  • 10 kg or more: 100 mg/m² over 6 hours once on day 1
• Fluorouracil as follows:
  • Less than 10 kg: 20 mg/kg IV once on day 2
  • 10 kg or more: 600 mg/m² IV once on day 2
• Vincristine as follows:
  • Less than 10 kg: 0.05 mg/kg IV once per day on days 2, 9, 16
  • 10 kg or more: 1.5 mg/m² (maximum dose of 2 mg) IV once per day on days 2, 9, 16

21-day cycle for 1 cycle (to be given within 42 days of resection)

References

1. AHEP0731: Katzenstein HM, Langham MR, Malogolowkin MH, Krailo MD, Towbin AJ, McCarville MB, Finegold MJ, Ranganathan S, Dunn S, McGahren ED, Tiao GM, O'Neill AF, Qayed M, Furman WL, Xia C, Rodriguez-Galindo C, Meyers RL; Children's Oncology Group. Minimal adjuvant chemotherapy for children with hepatoblastoma resected at diagnosis (AHEP0731): a Children's Oncology Group, multicentre, phase 3 trial. Lancet Oncology. 2019 May 1. 20(5) 719-727. Epub 2019 Apr 8. link to original article PubMed NCT00980460