Hepatoblastoma (HB) is the most common primary liver
tumor in children and accounts for two-thirds of all malignant
liver neoplasms in the pediatric population. For patients with advanced HB (unresectable or unresponsive to
chemotherapy), combined treatment with
chemotherapy and
liver transplantation is an excellent option. The etiology of HB is mostly obscure because of its extreme rarity although some inherited syndromes and very low birth weight have been associated with it. The prognosis for children with HB has significantly improved in the past three decades thanks to advancements in
chemotherapy, surgical resection and
postoperative care. In 2002 a surgical staging system called pretreatment extent of disease (PRETEXT) was designed to allow a universal, multidisciplinary approach to patients with HB. Between one-third to two-thirds of patients initially present with unresectable
tumors or distant
metastases, but up to 85% of these
tumors become operable after
neoadjuvant chemotherapy. Patients with PRETEXT categories 1, 2, and some 3 are referred for
neoadjuvant chemotherapy followed by surgical resection with the goal of complete
tumor removal. Classic treatments regimens include a combination of
cisplatin,
fluorouracil, and
vincristine or
cisplatin and
doxorubicin.
Liver transplantation is the only treatment option for unresectable HB. In 2010 the pediatric
end-stage liver disease, a pediatric-specific scoring system that determines a patient's ranking on the
liver transplant list, began to award additional "exception" points for patients with HB. We analyzed the Standard Transplant Analysis and Research dataset to assess the impact of changes in exception point criteria for HB on outcomes after
liver transplantation at Texas Children's Hospital in Houston, Texas. We found that patients who were listed for
transplantation with current HB exception criteria experienced a shorter waitlist time but survival was similar between the two eras.