Marked response to preoperative high-dose cis-platinum in children with unresectable hepatoblastoma

Chemotherapy is the only effective method of treating unresectable hepatoblastoma. Most protocols require the administration of multiple highly toxic agents. We evaluated the ability of single-agent high-dose cis-platinum (HD-CDDP) to shrink unresectable primary hepatoblastoma to allow resection. Seven children aged 11 to 72 months had unresectable hepatoblastoma based on size and location. Initial alpha-fetoprotein (AFP) levels were between 4,900 and 1,840,000 ng/mL (mean, 555,900 ng/mL). Chest computed tomography (CT) scans obtained before beginning HD-CDDP therapy showed multiple or bilateral lung masses in 3 patients. Chemotherapy for each of the 7 children consisted of only HD-CDDP, 150 mg/m2, at 3-week intervals. HD-CDDP was stopped and prompt resection performed when AFP levels ceased to decline significantly (n = 4; mean nadir, 18,600); the corrected creatinine clearance decreased below 60 mL/min (n = 2); or, in one case, significant hemorrhage occurred within the tumor. Therefore, the number of HD-CDDP doses given preoperatively varied between 1 and 7 (mean, 3). No children required dialysis. Tumor cells in the bone marrow of one child disappeared completely after one dose of HD-CDDP. Follow-up CT scans showed complete resolution of the pulmonary metastases in 2 children, a partial response in the third, and a marked reduction of primary tumors to resectable sizes. Six children underwent tumor excision with adequate margins; parents of the seventh child refused permission for surgery. There were 2 operative deaths, 3 deaths due to local or distant disease, and 2 patients survived (postoperative follow-up, 22 and 14 months).(ABSTRACT TRUNCATED AT 250 WORDS)