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早期临床试验预后评分的比较:澳大利亚单中心 10 年经验。

Comparison of Prognostic Scores in Early Phase Clinical Trials: A 10-year Single Centre Australian Experience.

机构信息

Department of Medical Oncology, Liverpool Hospital, Liverpool, Australia;

Department of Medical Oncology, Liverpool Hospital, Liverpool, Australia.

出版信息

Anticancer Res. 2024 May;44(5):2095-2102. doi: 10.21873/anticanres.17014.


DOI:10.21873/anticanres.17014
PMID:38677731
Abstract

BACKGROUND/AIM: Early phase clinical trials (EPCTs) assess the tolerability of novel anti-cancer therapeutics in patients with advanced malignancy. Patient selection is important given the modest clinical benefit and time commitments for trials. Prognostic scores have been developed to facilitate identification of high-risk patients. This study aimed to compare five prognostic scores to predict survival for patients on an EPCT. PATIENTS AND METHODS: We performed a retrospective review of patients enrolled in EPCT at Liverpool Hospital, Sydney, from 2013 to 2023. Demographic, biochemical, and survival data were collected from electronic medical records. The score from five prognostic scoring systems (Royal Marsden hospital, MD Anderson Cancer centre, Gustave Roussy Immune, MD Anderson Immune Checkpoint Inhibitor and Princess Margaret Hospital Index) were calculated. Overall survival was measured using the Kaplan-Meier method and predictive discrimination was assessed using Harrell's c-index. RESULTS: A total of 218 patients across 36 EPCTs were included. The median overall survival was 9.8 months with 22% of patients dying in less than 90 days. Seventeen to thirty-four percent of patients were categorised as high-risk. The MDACC score obtained the highest predictability for overall survival for the whole cohort (c-index=0.67, 95%CI=0.62-0.72) and the immunotherapy-based cohort (c-index= 0.65, 95%CI=0.59-0.71). However, all scores performed similarly with a significant overlap in the confidence intervals. CONCLUSION: Our retrospective audit confirms the utility of prognostic scores to predict survival in an Australian EPCT cohort, with similar predictive discrimination across various scoring systems. Integration of these prognostic tools into EPCT screening processes may optimise benefits and reduce risks associated with EPCTs.

摘要

背景/目的:早期临床试验(EPCT)评估新型抗癌疗法在晚期恶性肿瘤患者中的耐受性。鉴于试验的临床获益有限且需要投入大量时间,患者选择非常重要。已经开发了预后评分来帮助识别高危患者。本研究旨在比较五种预后评分,以预测 EPCT 患者的生存情况。 患者和方法:我们对 2013 年至 2023 年在悉尼利物浦医院参加 EPCT 的患者进行了回顾性研究。从电子病历中收集了人口统计学、生化和生存数据。计算了来自五种预后评分系统(皇家马斯登医院、MD 安德森癌症中心、古斯塔夫·鲁西免疫、MD 安德森免疫检查点抑制剂和玛格丽特公主医院指数)的评分。使用 Kaplan-Meier 方法测量总生存率,使用 Harrell's c 指数评估预测区分度。 结果:共纳入 36 项 EPCT 中的 218 名患者。中位总生存率为 9.8 个月,22%的患者在 90 天内死亡。17%至 34%的患者被归类为高危患者。MDACC 评分在整个队列(c 指数=0.67,95%CI=0.62-0.72)和免疫治疗为基础的队列(c 指数=0.65,95%CI=0.59-0.71)中对总生存率的预测性最高。然而,所有评分的表现相似,置信区间有显著重叠。 结论:我们的回顾性审计证实了预后评分在澳大利亚 EPCT 队列中预测生存的实用性,各种评分系统的预测区分度相似。将这些预后工具纳入 EPCT 筛选流程可能会优化 EPCT 的获益并降低风险。

相似文献

[1]
Comparison of Prognostic Scores in Early Phase Clinical Trials: A 10-year Single Centre Australian Experience.

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[2]
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[3]
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[4]
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[6]
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[7]
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[8]
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[9]
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[10]
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