Background Currently, nanoliposomal irinotecan (nal-IRI) + 5-fluorouracil/folinic acid (5-FU/LV) is the only approved second-line treatment for patients suffering from metastatic pancreatic ductal adenocarcinoma (mPDAC). However, also other chemotherapeutic regimens are used in this setting and due to the lack of clear real-world data on the efficacy of the different regimens, there is no consensus on the optimal treatment sequence for mPDAC patients. Objectives To provide information on the safe and efficacious use of nal-IRI + 5-FU/LV in clinical practice in Belgium, which is needed for healthcare professionals to estimate the risk-benefit ratio of the intervention. Methods Medical data of adult patients with mPDAC who were treated with nal-IRI + 5-FU/LV in one of the participating Belgian hospitals were retrospectively collected. Kaplan-Meier analysis was performed to obtain survival curves to estimate the median overall survival (OS) and progression-free survival (PFS). All other results were presented descriptively. Results A total of 56 patients [median age at diagnosis: 69 years (range 43 years), 57.1% male] were included. Patients received a median of 5 (range 49 cycles) nal-IRI + 5-FU/LV cycles, extended over 10 weeks (range 130.8 weeks). The median start dose for nal-IRI was 70 mg/m² (range 49.24 mg/m²) and chemotherapy dose reduction and delay occurred in, respectively, 42.8% and 37.5% of the patients. The median OS was 6.8 months (95% CI: 5.6-8.4 months) with a 6-month survival rate of 57.4% and a 1-year survival rate of 27.8% in the overall study population. The median OS for patients treated with nal-IRI as second-line therapy or as later-line treatment was, respectively, 6.8 months (95% CI: 5.9-7.0 months) and 5.6 months (95% CI: 4.2-no upper limit). In the overall study population, a median PFS of 3.1 months (95% CI: 2.4-4.6 months) and a disease control rate of 48.3%, comprising 30.4% stable disease, 16.1% partial and 1.8% complete response, was observed. The median PFS for patients treated with nal-IRI as second-line therapy was 3.9 months (95% CI: 2.8-4.8 months) while this was 2.4 months (95% CI: 1.9-9.1 months) for those that received nal-IRI in a later-line treatment. In terms of safety, gastrointestinal problems occurred most (64.3% of the patients) and from all reported treatment emergent adverse events, 39.2% were grade 3 or 4. Conclusion Nal-IRI + 5-FU/LV is a valuable, effective, and safe sequential treatment option following gemcitabine-based therapy in patients with mPDAC. Trial details Retrospective study on the efficacy and tolerability of liposomal irinotecan (NALIRI); ClinicalTrials.gov Identifier: NCT0509506 (https://clinicaltrials.gov/ct2/show/NCT05095064?term=naliri&draw=2&rank=2).

中文翻译：

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关于脂质体伊立替康联合 5-氟尿嘧啶和亚叶酸治疗比利时转移性胰腺导管腺癌的疗效和耐受性的真实世界分析。

背景 目前，纳米脂质体伊立替康 (nal-IRI) + 5-氟尿嘧啶/亚叶酸 (5-FU/LV) 是唯一批准用于治疗转移性胰腺导管腺癌 (mPDAC) 患者的二线治疗。然而，在这种情况下也使用其他化疗方案，并且由于缺乏关于不同方案疗效的明确的真实数据，因此对于 mPDAC 患者的最佳治疗顺序尚未达成共识。目的 提供有关比利时临床实践中安全有效使用 nal-IRI + 5-FU/LV 的信息，医疗保健专业人员需要这些信息来估计干预措施的风险收益比。方法 回顾性收集在比利时一所参与医院接受 nal-IRI + 5-FU/LV 治疗的成年 mPDAC 患者的医疗数据。进行 Kaplan-Meier 分析以获得生存曲线，以估计中位总生存期 (OS) 和无进展生存期 (PFS)。所有其他结果均以描述性方式呈现。结果 共纳入 56 例患者[诊断时中位年龄：69 岁（范围 43 岁），57.1% 为男性]。患者平均接受 5 个（范围 49 个周期）nal-IRI + 5-FU/LV 周期，延长超过 10 周（范围 130.8 周）。nal-IRI 的中位起始剂量为 70 mg/m2（范围 49.24 mg/m2），化疗剂量减少和延迟发生的患者分别为 42.8% 和 37.5%。总体研究人群的中位 OS 为 6.8 个月（95% CI：5.6-8.4 个月），6 个月生存率为 57.4%，1 年生存率为 27.8%。使用 nal-IRI 作为二线治疗或后期治疗的患者的中位 OS 分别为 6.8 个月（95% CI：5.9-7.0 个月）和 5.6 个月（95% CI：4.2-无上限） ）。在整个研究人群中，观察到中位 PFS 为 3.1 个月（95% CI：2.4-4.6 个月），疾病控制率为 48.3%，其中疾病稳定率为 30.4%，部分缓解率为 16.1%，完全缓解率为 1.8%。使用 nal-IRI 作为二线治疗的患者的中位 PFS 为 3.9 个月（95% CI：2.8-4.8 个月），而接受 nal-IRI 治疗的患者的中位 PFS 为 2.4 个月（95% CI：1.9-9.1 个月）在后期治疗中。就安全性而言，胃肠道问题发生最多（64.3%的患者），在所有报告的治疗中出现的不良事件中，39.2%为3级或4级。 结论 Nal-IRI + 5-FU/LV是一种有价值、有效且安全的治疗方案。 mPDAC 患者接受吉西他滨治疗后的安全序贯治疗选择。试验细节 脂质体伊立替康（NALIRI）疗效和耐受性的回顾性研究；ClinicalTrials.gov 标识符：NCT0509506 (https://clinicaltrials.gov/ct2/show/NCT05095064?term=naliri&draw=2&rank=2)。