Chew Xin Hui, Sultana Rehena, Mathew Eshani N, Ng David Chee Eng, Lo Richard H G, Toh Han Chong, Tai David, Choo Su Pin, Goh Brian Kim Poh, Yan Sean Xuexian, Loke Kelvin Siu Hoong, Thang Sue Ping, Gogna Apoorva, Venkatanarasimha Nanda Karaddi, Tong Aaron K T, Moe Fiona N N, Chua Jacelyn S S, Ang Reiko W T, Ong Aldwin D, Ng Ashley W Y, Hoang Marjorie T Q, Too Chow Wei, Thng Choon Hua, Chan Wan Ying, Kee Wanyi, Chan Jaclyn H M, Irani Farah, Leong Sum, Lim Kiat Hon, Wang Michael L C, Chow Pierce K H
Department of Hepatopancreatobiliary and Transplantation Surgery, Singapore General Hospital and National Cancer Centre Singapore, Singapore, Singapore.
School of Medicine, University of Glasgow, Glasgow, United Kingdom.
Liver Cancer. 2021 Jun;10(3):224-239. doi: 10.1159/000514400. Epub 2021 Apr 7.
Real-world management of patients with hepatocellular carcinoma (HCC) is crucially challenging in the current rapidly evolving clinical environment which includes the need for respecting patient preferences and autonomy. In this context, regional/national treatment guidelines nuanced to local demographics have increasing importance in guiding disease management. We report here real-world data on clinical outcomes in HCC from a validation of the Consensus Guidelines for HCC at the National Cancer Centre Singapore (NCCS).
We evaluated the NCCS guidelines using prospectively collected real-world data, comparing the efficacy of treatment received using overall survival (OS) and progression-free survival (PFS). Treatment outcomes were also independently evaluated against 2 external sets of guidelines, the Barcelona Clinic Liver Cancer (BCLC) and Hong Kong Liver Cancer (HKLC).
Overall treatment compliance to the NCCS guidelines was 79.2%. Superior median OS was observed in patients receiving treatment compliant with NCCS guidelines for early (nonestimable vs. 23.5 months < 0.0001), locally advanced (28.1 vs. 22.2 months = 0.0216) and locally advanced with macrovascular invasion (10.3 vs. 3.3 months = 0.0013) but not for metastatic HCC (8.1 vs. 6.8 months = 0.6300), but PFS was similar. Better clinical outcomes were seen in BCLC C patients who received treatment compliant with NCCS guidelines than in patients with treatment only allowed by BCLC guidelines (median OS 14.2 vs. 7.4 months = 0.0002; median PFS 6.1 vs. 4.0 months = 0.0286). Clinical outcomes were, however, similar for patients across all HKLC stages receiving NCCS-recommended treatment regardless of whether their treatment was allowed by HKLC.
The high overall compliance rate and satisfactory clinical outcomes of patients managed according to the NCCS guidelines confirm its validity. This validation using real-world data considers patient and treating clinician preferences, thus providing a realistic analysis of the usefulness of the NCCS guidelines when applied in the clinics.
在当前快速发展的临床环境中，肝细胞癌（HCC）患者的实际管理面临着巨大挑战，这其中包括尊重患者偏好和自主权的需求。在此背景下，根据当地人口统计学情况进行细化的区域/国家治疗指南在指导疾病管理方面的重要性日益凸显。我们在此报告新加坡国立癌症中心（NCCS）对肝癌共识指南进行验证后得出的关于肝癌临床结局的真实世界数据。
我们使用前瞻性收集的真实世界数据对NCCS指南进行评估，通过总生存期（OS）和无进展生存期（PFS）比较所接受治疗的疗效。治疗结果还与另外两套外部指南——巴塞罗那临床肝癌（BCLC）指南和香港肝癌（HKLC）指南进行了独立比较。
总体而言，对NCCS指南的治疗依从率为79.2%。在接受符合NCCS指南治疗的早期（不可评估与23.5个月相比，<0.0001）、局部晚期（28.1与22.2个月相比，=0.0216）和伴有大血管侵犯的局部晚期（10.3与3.3个月相比，=0.0013）肝癌患者中观察到了更长的中位总生存期，但转移性肝癌患者未观察到（8.1与6.8个月相比，=0.6300），不过无进展生存期相似。接受符合NCCS指南治疗的BCLC C期患者的临床结局优于仅接受BCLC指南允许治疗的患者（中位总生存期14.2与7.4个月相比，=0.0002；中位无进展生存期6.1与4.0个月相比，=0.0286）。然而，无论其治疗是否被HKLC允许，所有接受NCCS推荐治疗的HKLC各期患者的临床结局相似。
根据NCCS指南管理的患者总体依从率高且临床结局令人满意，证实了该指南的有效性。这种使用真实世界数据进行的验证考虑了患者和治疗医生的偏好，从而对NCCS指南在临床应用中的实用性提供了现实的分析。
