Hepatoblastoma

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Wayne H. Liang, MD, MS, FAMIA
UAB
Birmingham, AL

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3 regimens on this page
2 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

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

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, 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

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