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Reuters Health Information (2011-07-22): Complete resection cures stage 1 pediatric hepatoblastoma


Complete resection cures stage 1 pediatric hepatoblastoma

Last Updated: 2011-07-22 17:13:02 -0400 (Reuters Health)

NEW YORK (Reuters Health) - A subgroup of children with hepatoblastoma can be treated with surgery alone, according to results of a small study.

Only patients with the most favorable type of hepatoblastoma -- stage I disease with pure fetal histology, were included in the study.

Although the group represents only the "rarest of the rare" pediatric cancers, the finding is significant because it could help these youngsters avoid the long-term side effects of chemotherapy, said lead author Dr. Marcio Malogolowkin at Children's Hospital Los Angeles.

For example, he said, doxorubicin is one of the most common therapies for pediatric hepatoblastoma, but it's highly cardiotoxic, and cisplatin can significantly impair hearing.

"If you are talking about one and two year olds, obviously you have major concerns about their development," Dr. Malogolowkin said.

Dr. Daniel Aronson, chief of Pediatric Surgery at Radboud University Nijmegen Medical Center in The Netherlands, agrees, although he wasn't involved in the research. "This is an important finding," he told Reuters Health in an email. Although it's a very small study, it's the first to show that this small group of patients "can be cured with surgery only," he said.

As reported online July 18 in the Journal of Clinical Oncology, the researchers drew their subjects from two pediatric hepatoblastoma trials. The first group, treated between August 1989 and December 1992, included nine children with stage I pure fetal histology (PFH) disease who received single-agent bolus doxorubicin for three consecutive days after surgical resection. The second group, treated from March 1999 to November 2006, included 16 children with stage I PFH who were treated with surgical resection alone.

With a median follow-up of five years, event-free survival and overall survival were both 100% for patients with stage I PFH in each group.

Because histology can usually only be confirmed after resection, the study also assessed whether staging at diagnosis could be used to predict which patients can avoid chemotherapy. By retrospectively applying the current international standard for classifying liver disease -- the PRETEXT scoring system, which divides the liver into segments -- the investigators found that PRETEXT staging could be used to accurately predict surgical resectability.

"We are coming up with some very specific criteria of who should be submitted to upfront surgery at diagnosis," said Dr. Malogolowkin. He said that currently all patients that have a more limited involvement of the liver, typically PRETEXT stage I or II, should have upfront surgery without chemotherapy.

The larger question now is whether chemotherapy regimens for other hepatoblastoma subtypes can be stratified based on histology.

"What we don't know yet, is are there are other subtypes of hepatoblastoma for whom we would be able also to decrease therapy," Dr. Malogolowkin added.


J Clin Oncol 2011.

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