儿童肝细胞癌：改良铂类和多柔比星为基础的化疗是否能增加肿瘤可切除性并改变结局？来自 SIOPEL 2 和 3 研究的经验教训。

Hepatocellular Carcinoma in Children: Does Modified Platinum- and Doxorubicin-Based Chemotherapy Increase Tumor Resectability and Change Outcome? Lessons Learned From the SIOPEL 2 and 3 Studies.

机构信息

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.

出版信息

J Clin Oncol. 2016 Apr 1;34(10):1050-6. doi: 10.1200/JCO.2014.60.2250. Epub 2016 Jan 25.

DOI:10.1200/JCO.2014.60.2250

PMID:26811523

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.

摘要

简介

本文旨在介绍国际儿童肝脏肿瘤策略组（SIOPEL2[S2]和 SIOPEL[S3]）试验中的两项前瞻性研究经验，并评估改良铂类和阿霉素化疗是否能够提高肿瘤可切除性并改变患者预后。

方法

1995 年至 2006 年，S2 试验纳入 20 例肝细胞癌（HCC）患者，S3 试验纳入 70 例患者。85 例患者可评估。

结果

72 例未行初始手术的患者中，29 例（40%）对术前化疗有反应，而 13 例患者行初始手术。33 例患者行延期切除术。39 例肿瘤始终无法切除。34 例患者（40%）获得完全肿瘤切除，其中 7 例接受肝移植（LTX）治疗。中位随访 75 个月后，63 例患者（74%）发生事件（治疗期间进展、治疗后复发或任何原因死亡）。60 例患者死亡。46 例肿瘤切除患者中，23 例（50%）死亡。18 例完全肿瘤切除（无 LTX）患者和 27 例患者中的 20 例（63%）以及 34 例患者中的 34 例（59%）LTX 存活。仅 7 例患者（14%）有镜下累及边缘的患者存活。5 年总生存率为 22%。