Hepatoblastoma is the most common liver
malignancy in children, typically diagnosed before age 2. The survival rate for
hepatoblastoma has increased dramatically in the last 30 years, but the typical chemotherapeutic agents used for treatment are associated with significant toxicity. In this report, the authors present two cases of
hepatoblastoma treated with surgical resection and a novel biotherapeutic regimen that included
opioid growth factor (OGF). Case #1 is an infant diagnosed with a large mass on prenatal ultrasound. After subsequent diagnosis of
hepatoblastoma, she was treated with one course of
neoadjuvant chemotherapy at approximately 1 week of age. Following significant complications from the
chemotherapy (neutropenic
fever,
pneumonia and
sepsis), the patient's parents declined further
chemotherapy, and the infant was treated with surgical resection and
opioid growth factor (OGF)/low dose
naltrexone (LDN). She is currently at close to 10 years disease-free survival. Case #2 is a child diagnosed with a liver mass on ultrasound at 20 months of age, later biopsy-proven to represent
hepatoblastoma. Due to existing co-morbidities including
autosomal recessive polycystic kidney disease and
hypertension, and indications from the biopsy that the
tumor might be insensitive to
chemotherapy, the parents elected not to proceed with
neoadjuvant chemotherapy. The patient was treated with surgical resection and OGF/LDN, and is currently at more than 5 years disease-free survival. This case series highlights the need for less toxic treatment options than conventional
chemotherapy. Modulation of the OGF-
OGF receptor axis represents a promising safe and therapeutic avenue for effective treatment of
hepatoblastoma.