TY - JOUR
T1 - Hepatocellular carcinoma in children
T2 - Does modified platinum-and doxorubicin-based chemotherapy increase tumor resectability and change outcome? Lessons learned from the SIOPEL 2 and 3 studies
AU - Murawski, Maciej
AU - Weeda, Víola B.
AU - Maibach, Rudolf
AU - Morland, Bruce
AU - Roebuck, Derek J.
AU - Zimmerman, Arthur
AU - Casanova, Michela
AU - Perilongo, Giorgio
AU - Laithier, Veronique
AU - Kebudi, Rejin
AU - Scopinaro, Marcelo J.
AU - Shun, Albert
AU - Brichard, Benedicte
AU - De Camargo, Beatriz
AU - Childs, Margaret
AU - Aronson, Daniel C.
AU - Czauderna, Piotr
PY - 2016/4/1
Y1 - 2016/4/1
N2 - Introduction: The aim of this article is to present an experience of two prospective studies from the International Childhood Liver Tumor Strategy Group (SIOPEL 2 [S2] and SIOPEL [S3]) trials and to evaluate whether modified platinum-and doxorubicin-based chemotherapy is capable of increasing tumor resectability and changing patient outcomes. Methods: Between 1995 and 2006, 20 patients with hepatocellular carcinoma (HCC) were included in the S2 trial and 70 were included in the S3 trial. Eighty-five patients remained evaluable. Results: Response to preoperative chemotherapy was observed in 29 of 72 patients (40%) who did not have primary surgery, whereas 13 patients underwent upfront surgery. Thirty-three patients had a delayed resection. Thirty-nine tumors never became resectable. Complete tumor resection was achieved in 34 patients (40%), including seven of those treated with liver transplantation (LTX). After a median follow-up period of 75 months, 63 patients (74%) had an event (a progression during treatment, a relapse after treatment, or death from any cause). Sixty patients died. Twenty-three of 46 patients (50%) who underwent tumor resection died. Eighteen of 27 patients (63%) with complete tumor resection (without LTX) and 20 of 34 patients (59%) with LTX survived. Only one of seven patients (14%) with microscopically involved margins survived. Overall survival at 5 years was 22%. Conclusion: Survival in pediatric HCC is more likely when complete tumor resection can be achieved. Intensification of platinum agents in the S2 and S3 trials has not resulted in improved survival. New treatment approaches in pediatric HCC should be postulated.
AB - Introduction: The aim of this article is to present an experience of two prospective studies from the International Childhood Liver Tumor Strategy Group (SIOPEL 2 [S2] and SIOPEL [S3]) trials and to evaluate whether modified platinum-and doxorubicin-based chemotherapy is capable of increasing tumor resectability and changing patient outcomes. Methods: Between 1995 and 2006, 20 patients with hepatocellular carcinoma (HCC) were included in the S2 trial and 70 were included in the S3 trial. Eighty-five patients remained evaluable. Results: Response to preoperative chemotherapy was observed in 29 of 72 patients (40%) who did not have primary surgery, whereas 13 patients underwent upfront surgery. Thirty-three patients had a delayed resection. Thirty-nine tumors never became resectable. Complete tumor resection was achieved in 34 patients (40%), including seven of those treated with liver transplantation (LTX). After a median follow-up period of 75 months, 63 patients (74%) had an event (a progression during treatment, a relapse after treatment, or death from any cause). Sixty patients died. Twenty-three of 46 patients (50%) who underwent tumor resection died. Eighteen of 27 patients (63%) with complete tumor resection (without LTX) and 20 of 34 patients (59%) with LTX survived. Only one of seven patients (14%) with microscopically involved margins survived. Overall survival at 5 years was 22%. Conclusion: Survival in pediatric HCC is more likely when complete tumor resection can be achieved. Intensification of platinum agents in the S2 and S3 trials has not resulted in improved survival. New treatment approaches in pediatric HCC should be postulated.
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U2 - 10.1200/JCO.2014.60.2250
DO - 10.1200/JCO.2014.60.2250
M3 - Article
C2 - 26811523
AN - SCOPUS:84963588750
VL - 34
SP - 1050
EP - 1056
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
SN - 0732-183X
IS - 10
ER -