Maciej Murawski1, Víola B Weeda1, Rudolf Maibach1, Bruce Morland1, Derek J Roebuck1, Arthur Zimmerman1, Michela Casanova1, Giorgio Perilongo1, Veronique Laithier1, Rejin Kebudi1, Marcelo J Scopinaro1, Albert Shun1, Benedicte Brichard1, Beatriz de Camargo1, Margaret Childs1, Daniel C Aronson1, Piotr Czauderna2. 1. Maciej Murawski and Piotr Czauderna, Medical University of Gdansk, Gdansk, Poland; Viola B. Weeda and Daniel C. Aronson, Emma Children's Hospital Academic Medical Center, Amsterdam, the Netherlands; Rudolf Maibach, International Breast Cancer Study Group Coordinating Center; Arthur Zimmerman, Institute of Pathology, Berne, Switzerland; Bruce Morland, Birmingham Children's Hospital, Birmingham; Derek J. Roebuck, Great Ormond Street Hospital, London; Margaret Childs, University of Nottingham, Nottingham, United Kingdom; Michela Casanova, National Cancer Institute, Milan; Giorgio Perilongo, University of Padova, Padova, Italy; Veronique Laithier, Centre Hospitalier Régional et Universitaire de Besançon, Besançon, France; Rejin Kebudi, İstanbul University, Oncology Institute, İstanbul, Turkey; Marcelo J. Scopinaro, Hospital de Pediatrı´a SAMIC J.P. Garrahan, Buenos Aires, Argentina; Albert Shun, Children's Hospital at Westmead, Sydney, Australia; Benedicte Brichard, Cliniques Universitaires Saint Luc, University of Louvain, Brussels, Belgium; and Beatriz de Camargo, Cancer Hospital A.C. Camargo, São Paulo, Brazil. 2. Maciej Murawski and Piotr Czauderna, Medical University of Gdansk, Gdansk, Poland; Viola B. Weeda and Daniel C. Aronson, Emma Children's Hospital Academic Medical Center, Amsterdam, the Netherlands; Rudolf Maibach, International Breast Cancer Study Group Coordinating Center; Arthur Zimmerman, Institute of Pathology, Berne, Switzerland; Bruce Morland, Birmingham Children's Hospital, Birmingham; Derek J. Roebuck, Great Ormond Street Hospital, London; Margaret Childs, University of Nottingham, Nottingham, United Kingdom; Michela Casanova, National Cancer Institute, Milan; Giorgio Perilongo, University of Padova, Padova, Italy; Veronique Laithier, Centre Hospitalier Régional et Universitaire de Besançon, Besançon, France; Rejin Kebudi, İstanbul University, Oncology Institute, İstanbul, Turkey; Marcelo J. Scopinaro, Hospital de Pediatrı´a SAMIC J.P. Garrahan, Buenos Aires, Argentina; Albert Shun, Children's Hospital at Westmead, Sydney, Australia; Benedicte Brichard, Cliniques Universitaires Saint Luc, University of Louvain, Brussels, Belgium; and Beatriz de Camargo, Cancer Hospital A.C. Camargo, São Paulo, Brazil. pczaud@gumed.edu.pl.
Abstract
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.
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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