Prognostic implications of serum alpha-fetoprotein response during treatment of hepatoblastoma†

Background We evaluated the outcomes and prognostic factors, especially serum levels of alpha-fetoprotein (AFP) and their changes during the treatment of hepatoblastoma (HB). Procedure We retrospectively analyzed the medical records of 43 consecutive children with HB treated at a single institution between 1991 and 2010. Results Of 43 patients, 5 (12%) underwent primary tumor resection at diagnosis and 38 (88%) received preoperative chemotherapy. Of those 38 patients, 7 (16%) died of progressive disease during preoperative chemotherapy, and 31 (72%) underwent curative operations, including 5 who underwent liver transplantation, after a median 4 cycles of chemotherapy (range, 3–14 cycles). The 5-year overall survival and disease-free survival rates were 62.1 ± 8.3% and 65.6 ± 7.6%, respectively. AFP >263,000 ng/mL at diagnosis, a decline of <1 log in AFP levels after the first cycle of chemotherapy, preoperative AFP levels in the highest tertile, and postoperative AFP levels in the highest tertiles were significantly associated with treatment failure. Age younger than 1 year at diagnosis, thrombocytosis at diagnosis, and early PRETEXT (pretreatment extent of disease) stage were significantly associated with better survival outcomes, whereas gender and metastasis were not. Multivariate analysis showed that high level of preoperative AFP was an independent predictor of treatment failure. Conclusions Serial monitoring of changes in AFP levels during the treatment, especially perioperative changes, may help identify favorable and poor responders to chemotherapy. Alternative treatment, such as liver transplantation, should be considered for poor responders. Pediatr Blood Cancer 2011; 57: 554–560. © 2011 Wiley-Liss, Inc.