Summary

This collection contains data from the National Cancer Institute Clinical Trial NCT00980460, "Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer." It was sponsored by NCI's Children’s Oncology Group (COG) under study number AHEP0731. This phase III trial studies the side effects and how well risk-based therapy works in treating younger patients with newly diagnosed liver cancer. Select individual patient-level data from this trial can be requested from the NCTN/NCORP Data Archive.

Trial Description

Surgery, chemotherapy drugs (cancer fighting medicines), and when necessary, liver transplant, are the main current treatments for hepatoblastoma. The stage of the cancer is one factor used to decide the best treatment. Treating patients according to the risk group they are in may help get rid of the cancer, keep it from coming back, and decrease the side effects of chemotherapy.

Hepatoblastoma treatment with curative intent requires surgical resection, but only about a third of newly diagnosed patients with hepatoblastoma have resectable disease at diagnosis. Patients who have upfront resection typically receive a total of 4–6 cycles of adjuvant chemotherapy post-surgery, with the combination of cisplatin, fluorouracil, and vincristine. The aim is to investigate whether event-free survival in children with hepatoblastoma who had complete resection at diagnosis could be maintained with two cycles of adjuvant chemotherapy.  This multicentre, phase 3 trial was designed to test a risk-based treatment approach for children with hepatoblastoma, to diminish toxicity in low-risk patients, improve survival in intermediate-risk patients, and identify new agents that may be used in high-risk and recurrent patients.  Patients were staged for risk classification using the Children’s Oncology Group staging guidelines before the initiation of chemotherapy, with stage IV indicating metastatic disease.  Pretreatment extent of disease (PRETEXT) grouping also was performed at the time of diagnosis and with any subsequent abdominal computed tomography or magnetic resonance imaging and was used to guide the surgical management but was not used for risk classification.  The response rate and outcome to the combination of vincristine and irinotecan administered in an upfront window to children newly diagnosed with high-risk hepatoblastoma was determined.

For Low-Risk patients CT chest was used for metastatic tumor response assessment.  Abdominal Ultrasound was obtained at baseline.  For Intermediate- and High-Risk patients abdominal ultrasound, CT and/or MRI was used for primary tumor response assessment and CT chest for metastatic tumor response assessment.

Trial Outcomes

Results of the trial for Low-Risk patients have been reported in the following publication:

Katzenstein, H. M., Langham, M. R., Malogolowkin, M. H., Krailo, M. D., Towbin, A. J., McCarville, M. B., Finegold, M. J., Ranganathan, S., Dunn, S., McGahren, E. D., Tiao, G. M., O’Neill, A. F., Qayed, M., Furman, W. L., Xia, C., Rodriguez-Galindo, C., & Meyers, R. L. (2019). Minimal adjuvant chemotherapy for children with hepatoblastoma resected at diagnosis (AHEP0731): a Children’s Oncology Group, multicentre, phase 3 trial. The Lancet Oncology, 20(5), 719–727. DOI: https://doi.org/10.1016/s1470-2045(18)30895-7. Epub 2019 Apr 8. Erratum in: Lancet Oncol. 2019 May;20(5):e243. PMID: 30975630; PMCID: PMC6499702. Epub 2019 Apr 8. Erratum in: Lancet Oncol. 2019 May;20(5):e243. PMID: 30975630; PMCID: PMC6499702.