Children and young adults with hepatoblastoma may be able to undergo minimal postoperative chemotherapy and maintain disease control, according to the results of a Children’s Oncology Group study.1

“These results will affect the care of about a third of patients with newly diagnosed hepatoblastoma and are the third dose reduction study reported for hepatoblastoma by the Children’s Oncology Group,” researchers reported in Lancet Oncology.

Patients with hepatoblastoma typically undergo 4 to 6 cycles of postoperative chemotherapy with cisplatin, fluorouracil, and vincristine, putting them at risk for chemotherapy-related complications.

This trial was designed to test if patients aged younger than 21 years could undergo only 2 cycles of postoperative chemotherapy and successfully maintain complete resection of their disease.

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The trial included 49 patients from 32 centers with confirmed stage I or stage II hepatoblastoma with complete resection at diagnosis. Patients received two 21-day cycles of cisplatin, fluorouracil, and vincristine within 42 days of resection. The primary outcome was investigator-assessed event-free survival.

With a median follow-up of 42 months, the 4-year event-free survival was 92%, and the 5-year event-free survival was 88%. One of the 3 patients who relapsed died of their disease. Two patients died in clinical remission after they discontinued therapy.

According to study researchers, “these data show the benefits of upfront tumor resection when feasible, and of diminished chemotherapy with decreased long-term morbidity while maintaining curative intent.”

Common grade 3/4 adverse events included febrile neutropenia (14%), decreased neutrophil count (6%), infections (8%), and diarrhea (8%). Ototoxicity occurred in 1 patient. No treatment-related deaths occurred.

“The development of evidence-based surgical guidelines using internationally constructed and accepted criteria based on PRETEXT group and annotation factors will be crucial for the success of this approach to translate into improved outcomes for patients with low-risk disease,” the researchers concluded.

Reference

  1. Katzenstein HM, Langham MR, Malogolowkin MH, et al. Minimal adjuvant chemotherapy for children with hepatoblastoma resected at diagnosis (AHEP0731): a Children’s Oncology Group, multicentre, phase 3 trial [published online April 8, 2019]. Lancet. doi: 10.1016/S1470-2045(18)30895-7