Fibrolamellar hepatocellular carcinoma in children and adolescents

Abstract

BACKGROUND Children with hepatocellular carcinoma (HCC) were treated on a prospective, randomized trial and were then analyzed to determine whether children with the fibrolamellar (FL) histologic variant of HCC have a more favorable presentation, increased surgical resectability, greater response to therapy, and improved outcome compared with children who have typical HCC. METHODS Forty‐six patients were enrolled on Pediatric Intergroup Hepatoma Protocol INT‐0098 (Pediatric Oncology Group Study 8945/Children's Cancer Group Study 8881) between August 1989 and December 1992. After undergoing initial surgery or biopsy, children with Stage I HCC (n = 8 patients), Stage III HCC (n = 25 patients), and Stage IV HCC (n = 13 patients) were assigned randomly, regardless of histology, to receive treatment either with cisplatin, vincristine, and fluorouracil (n = 20 patients) or with cisplatin and continuous‐infusion doxorubicin (n = 26 patients). RESULTS Ten of 46 patients (22%) had the fibrolamellar variant of HCC (FL‐HCC). For the entire cohort, the estimated 5‐year event free survival (EFS) rate (± standard deviation) was 17% ± 6%. There was no difference in outcome among patients who were treated with either regimen. The 5‐year EFS rate for patients with FL‐HCC was no different the rate for patients with typical HCC (30% ± 15% vs. 14% ± 6%, respectively; P = 0.18), although the median survival was longer in patients with FL‐HCC. There was no difference in the number of patients with advanced‐stage disease, the incidence of surgical resectability at diagnosis, or the response to treatment between patients with FL‐HCC and patients with typical HCC. CONCLUSIONS Children with FL‐HCC do not have a favorable prognosis and do not respond any differently to current therapeutic regimens than patients with typical HCC. Children with initially resectable HCC have a good prognosis irrespective of histologic subtype, whereas outcomes are poor uniformly for children with advanced‐stage disease. The use of novel chemotherapeutic agents and the incorporation of other treatment modalities are indicated to improve the dismal survival of pediatric patients with all histologic variants of advanced‐stage HCC. Cancer 2003;97:2006–12. © 2003 American Cancer Society. DOI 10.1002/cncr.11292

Transcriptomic characterization of fibrolamellar hepatocellular carcinoma

“…cancer that usually occurs in adolescents and young adults (1)(2)(3). Effective therapeutic options are limited; FLHCC does not respond well to chemotherapy (4,5), although there have been some encouraging retrospective studies assessing the use of 5-fluororacil and IFN alpha-B2 (6). There have been mixed reports as to whether FLHCC does (7,8) or does not (5) have a better prognosis than hepatocellular carcinoma (HCC).…”

Place of liver transplantation in the treatment of hepatocellular carcinoma in the normal liver

“…Separate Note on FL-HCC [43][44][45][46][47][48][49][50] Because the analysis of the literature shows that NC-HCC and FL-HCC are still perceived differently on account of their different clinical presentations, courses, imaging results, and outcomes, some brief remarks about this tumor variant are made here.…”

“…FL-HCC is said to be associated with a (more) favorable prognosis. [45][46][47] In fact, FL-HCC is an aggressive tumor associated with a less than 50% 5-year OS rate, even in resectable cases. There are several reasons for this discrepancy and the frequently optimistic results reported for FL-HCC: (1) the series are usually small and lack statistical power; (2) the diagnostic pathological triad necessary to unequivocally diagnose this subtype of HCC is frequently missing; (3) FL-HCC often occurs in cirrhotic or abnormal livers; (4) follow-up is frequently lacking; and (5) most importantly, the outcomes of FL-HCC are mostly compared to the outcomes of HCC in general.…”

“…There are several reasons for this discrepancy and the frequently optimistic results reported for FL-HCC: (1) the series are usually small and lack statistical power; (2) the diagnostic pathological triad necessary to unequivocally diagnose this subtype of HCC is frequently missing; (3) FL-HCC often occurs in cirrhotic or abnormal livers; (4) follow-up is frequently lacking; and (5) most importantly, the outcomes of FL-HCC are mostly compared to the outcomes of HCC in general. 46,47 A literature review by Kakar et al 43 clearly showed that the outcomes of FL-HCC and NC-HCC are similar when same-stage diseases are considered; the outcomes of FL-HCC are much better than the outcomes of HCC with cirrhosis. The results of the ELTR study (unpublished data, 2011) confirm these findings.…”

Hepatocarcinoma fibrolamelar (HFL): relato de caso

“…Estudo de Katzenstein et al 7 comparou esta dosagem em pacientes com carcinoma hepatocelular e hepatocarcinoma fibrolamelar, mostrando 89% de normalidade neste último e 82% aumentada no carcinoma hepatocelular.…”