Marques D, Costa A L, Mansinho A, Quintela A, Pratas E, Brito-da-Silva J, Cruz J, Félix J, Rodrigues J, Mota M, Teixeira A R, Dâmaso S, Pinheiro S, Andreozzi V, Costa L, Barros A G
Department of Medical Oncology, Portuguese Oncology Institute, Porto, Portugal.
Department of Medical Oncology, Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Lisbon, Portugal.
Clin Oncol (R Coll Radiol). 2023 Oct;35(10):665-672. doi: 10.1016/j.clon.2023.07.004. Epub 2023 Jul 14.
In the pivotal RECOURSE trial, trifluridine/tipiracil improved survival outcomes in refractory metastatic colorectal cancer (mCRC), while demonstrating an acceptable toxicity profile. Routine clinical practice evidence is important to support the ongoing value of recently approved medicines. Our objective was to assess the utilisation patterns and real-world effectiveness of trifluridine/tipiracil in previously treated mCRC patients.
This was a retrospective observational study including consecutive patients who started trifluridine/tipiracil between 1 April 2018 and 30 September 2019 in the medical oncology departments of three major public hospitals in Portugal. The primary outcome measure was overall survival. Associations between overall survival and patient and tumour characteristics were assessed using multivariate Cox regression analyses.
In total, 111 patients were included in the study, with a mean age of 64 years. From these, 45.9% received two prior lines of treatment, 47.8% had three or more previous lines of treatment and 83.6% had Eastern Cooperative Oncology Group (ECOG) performance status 0-1 at baseline. The median duration of trifluridine/tipiracil treatment was 3.7 cycles (95% confidence interval 3.4-4.1). Most patients (80.4%) remained on their planned dose throughout the trifluridine/tipiracil treatment period, fulfilling 100% relative dose intensity. The median overall survival in the total study cohort was 7.9 months (95% confidence interval 6.4-9.8) and the median progression-free survival was 3.4 months (95% confidence interval 3.2-3.9). The median overall survival was significantly higher in patients with a normal serum lactate dehydrogenase (LDH) level (median overall survival 11.2 months for [135, 205] IU/l LDH [95% confidence interval 8.2-NR] and 13.6 months for [205, 251] IU/l LDH [95% confidence interval 8.2-NR]) and in better fitted (ECOG = 0-1) patients (median overall survival 8.0 months; 95% confidence interval 6.7-10.0). The median time to worsening performance status was 6.2 months (95% confidence interval 5.0-8.0). Treatment discontinuation due to adverse events was low (3.1%).
Our study confirms the effectiveness of trifluridine/tipiracil in real-life mCRC patients. Overall survival and progression-free survival outcomes are consistent with the efficacy profile reported in the earlier randomised RECOURSE clinical trial. Like other real-world studies, we found no additional safety concerns in the use of trifluridine/tipiracil.
在关键的RECOURSE试验中，曲氟尿苷/替匹嘧啶改善了难治性转移性结直肠癌（mCRC）的生存结局，同时显示出可接受的毒性特征。常规临床实践证据对于支持近期获批药物的持续价值很重要。我们的目的是评估曲氟尿苷/替匹嘧啶在既往接受过治疗的mCRC患者中的使用模式和实际疗效。
这是一项回顾性观察性研究，纳入了2018年4月1日至2019年9月30日期间在葡萄牙三家主要公立医院的肿瘤内科开始使用曲氟尿苷/替匹嘧啶的连续患者。主要结局指标是总生存期。使用多变量Cox回归分析评估总生存期与患者及肿瘤特征之间的关联。
该研究共纳入111例患者，平均年龄64岁。其中，45.9%的患者接受过两线先前治疗，47.8%的患者接受过三线或更多线先前治疗，83.6%的患者在基线时东部肿瘤协作组（ECOG）体能状态为0 - 1。曲氟尿苷/替匹嘧啶治疗的中位疗程为3.7个周期（95%置信区间3.4 - 4.1）。大多数患者（80.4%）在整个曲氟尿苷/替匹嘧啶治疗期间维持计划剂量，实现了100%的相对剂量强度。整个研究队列的中位总生存期为7.9个月（95%置信区间6.4 - 9.8），中位无进展生存期为3.4个月（95%置信区间3.2 - 3.9）。血清乳酸脱氢酶（LDH）水平正常的患者中位总生存期显著更长（LDH水平为[135, 205] IU/L时中位总生存期为11.2个月[95%置信区间8.2 - 未报告（NR）]，LDH水平为[205, 251] IU/L时中位总生存期为13.6个月[95%置信区间8.2 - NR]），以及体能状态较好（ECOG = 0 - 1）的患者（中位总生存期8.0个月；95%置信区间6.7 - 10.0）。体能状态恶化的中位时间为6.2个月（95%置信区间5.0 - 8.0）。因不良事件导致的治疗中断率较低（3.1%）。
我们的研究证实了曲氟尿苷/替匹嘧啶在现实生活中的mCRC患者中的有效性。总生存期和无进展生存期结局与早期随机RECOURSE临床试验报告的疗效特征一致。与其他现实世界研究一样，我们发现在使用曲氟尿苷/替匹嘧啶时没有额外的安全问题。
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