University Hospital Southampton NHS Foundation Trust, Southampton, UK.
OPEN VIE, Marlow, UK.
BMC Cancer. 2020 Feb 3;20(1):91. doi: 10.1186/s12885-020-6577-1.
The standard first- and second- line chemotherapy backbone regimens for metastatic colorectal cancer (mCRC) are 5-fluorouracil (5-FU)/capecitabine-based with addition of irinotecan or oxaliplatin. Until recently, evidence for optimal sequencing post second-line was sparse. Trifluridine/tipiracil (indicated for mCRC and gastric cancer after standard chemotherapies) was made available to UK patients via a named patient programme (NPP) before receiving marketing authorisation in Europe in 2016, allowing characterisation of UK treatment pathways, and evaluation of trifluridine/tipiracil in a UK non-trial population.
Data collected routinely for the NPP were analysed to describe the patient demographics, clinical characteristics and treatment pathways. Patients eligible for the programme were adults (≥18 years) with histologically or cytologically confirmed mCRC who had previously received chemotherapy treatment(s).
Of the 250 eligible patients enrolled in the NPP, 194 patients received ≥1 dose of trifluridine/tipiracil and 56 patients did not receive trifluridine/tipiracil. The following results are reported first for patients who received trifluridine/tipiracil and second for those who did not receive trifluridine/tipiracil: median (IQR) age was 63.0 (54.0-69.0) and 62.0 (54.8-69.0) years; Eastern Cooperative Oncology Group performance status score was 0 for 28 and 14%, 1 for 65 and 70%, 2 for 7 and 16%. In terms of previous systemic treatments 47 and 43% had 2 prior lines of therapy. FOLFOX-, FOLFIRI- and CAPOX-based therapies were the most common first-line regimens in patients receiving trifluridine/tipiracil (37, 35 and 21%, respectively), and in patients not receiving trifluridine/tipiracil (41, 30 and 20%, respectively). Second-line treatment regimens in patients receiving and not receiving trifluridine/tipiracil were most commonly FOLFIRI-based (48 and 41%, respectively) and FOLFOX-based (19 and 21%, respectively). Patients received a median of 2 cycles of trifluridine/tipiracil with a median treatment duration of 1.8 (95% CI: 1.8-2.4) months. In patients who discontinued treatment due to disease progression, the median progression-free duration was 2.8 (95% CI: 2.4-2.9) months.
The results highlight the number of treatment pathways used to treat mCRC in routine UK clinical practice prior to the marketing authorisation and National Institute for Health and Care Excellence approval of trifluridine/tipiracil and highlight the lack of clinical guidelines for mCRC.
转移性结直肠癌（mCRC）的标准一线和二线化疗骨干方案是基于 5-氟尿嘧啶（5-FU）/卡培他滨，加用伊立替康或奥沙利铂。直到最近，二线治疗后最佳治疗顺序的证据还很少。曲氟尿苷/替匹嘧啶（用于标准化疗后 mCRC 和胃癌）于 2016 年在欧洲获得上市许可前通过英国的一项指定患者计划（NPP）提供给英国患者，这使得能够描述英国的治疗途径，并在英国非试验人群中评估曲氟尿苷/替匹嘧啶的疗效。
对 NPP 常规收集的数据进行分析，以描述患者的人口统计学、临床特征和治疗途径。有资格参加该计划的患者为年龄≥18 岁、组织学或细胞学证实的 mCRC 患者，这些患者之前接受过化疗。
在纳入 NPP 的 250 名符合条件的患者中，194 名患者接受了至少 1 个疗程的曲氟尿苷/替匹嘧啶治疗，56 名患者未接受曲氟尿苷/替匹嘧啶治疗。以下结果首先报告接受曲氟尿苷/替匹嘧啶治疗的患者的情况，然后报告未接受曲氟尿苷/替匹嘧啶治疗的患者的情况：中位（IQR）年龄分别为 63.0（54.0-69.0）和 62.0（54.8-69.0）岁；东部肿瘤协作组体能状态评分为 0 的分别为 28%和 14%，1 的为 65%和 70%，2 的为 7%和 16%。就既往系统治疗而言，47%和 43%的患者接受了 2 线治疗。接受曲氟尿苷/替匹嘧啶治疗的患者中，最常见的一线方案为 FOLFOX、FOLFIRI 和 CAPOX（分别为 37%、35%和 21%），未接受曲氟尿苷/替匹嘧啶治疗的患者中，最常见的一线方案也为 FOLFOX、FOLFIRI 和 CAPOX（分别为 41%、30%和 20%）。接受曲氟尿苷/替匹嘧啶治疗和未接受曲氟尿苷/替匹嘧啶治疗的患者中，二线治疗方案最常见的为 FOLFIRI（分别为 48%和 41%）和 FOLFOX（分别为 19%和 21%）。接受曲氟尿苷/替匹嘧啶治疗的患者中位接受 2 个周期治疗，中位治疗时间为 1.8（95%CI：1.8-2.4）个月。由于疾病进展而停止治疗的患者中，中位无进展生存期为 2.8（95%CI：2.4-2.9）个月。
这些结果突出了在曲氟尿苷/替匹嘧啶获得上市许可和英国国家卫生与临床优化研究所批准之前，英国常规临床实践中治疗 mCRC 所使用的治疗途径数量，并强调了 mCRC 缺乏临床指南。
