Ding Philip Q, Tam Vincent C, Ramjeesingh Ravi, Asselah Jamil, Sheffield Brandon S, Mitchell Taylor, Gaudreau Anne-Julie, Knox Jennifer J, Cheung Winson Y
Oncology Outcomes Research Program, University of Calgary, Calgary, AB T2N 4N1, Canada.
Faculty of Medicine & Dentistry, University of Alberta, Edmonton, AB T6G 2R3, Canada.
Curr Oncol. 2025 Jul 16;32(7):405. doi: 10.3390/curroncol32070405.
In September 2021, pemigatinib received Health Canada approval for previously treated locally advanced/metastatic cholangiocarcinoma (CCA) with rearrangements/fusions. This retrospective study aimed to characterize the real-world management and outcomes of patients with CCA receiving pemigatinib through a Canadian patient support program (PSP).
We evaluated a multi-centre case series of Canadian patients who were prescribed pemigatinib between September 2021 and January 2023 for eligible CCA diagnoses and enrolled in the PSP. The retrospective study data included demographic and disease-, treatment-, and outcome-related information, and these were collected using a survey of prescribing physicians.
Of the 26 patients who initiated pemigatinib in the PSP, we received survey responses for 18 (69%). Their median age was 57 years, 67% were female, 61% had stage IV disease, and 83% had intrahepatic CCA. Prior to pemigatinib, a partial hepatectomy was performed in 44% of the patients, and 66% of the patients received 2-4 prior lines of systemic therapy. All patients were treated with platinum-based regimens as the first-line treatment for unresectable/metastatic disease. The median follow-up time on pemigatinib was 12.6 (range: 2.3-28.4) months, and their median real-world progression-free survival (rwPFS) was 12.1 months (95% CI 7.2-NR). The physician-assessed objective response and disease control rates were 56% and 89%, respectively. For the nine patients who discontinued pemigatinib, the median treatment duration was 10.6 months (range: 0.8-21.7). Disease progression was the most common reason for discontinuation (89%). None discontinued due to adverse events.
Objective response rates, disease control rates, and a PFS comparable to that in the phase 2 FIGHT-202 trial was reported with pemigatinib use in this Canadian PSP cohort.
2021年9月，培米替尼获得加拿大卫生部批准，用于治疗先前接受过治疗的局部晚期/转移性胆管癌（CCA）且伴有重排/融合的患者。这项回顾性研究旨在通过加拿大患者支持项目（PSP）描述接受培米替尼治疗的CCA患者的真实世界管理情况和治疗结果。
我们评估了一个多中心病例系列，这些加拿大患者在2021年9月至2023年1月期间因符合条件的CCA诊断而被处方培米替尼，并参加了PSP。回顾性研究数据包括人口统计学信息以及与疾病、治疗和结果相关的信息，这些信息是通过对开处方医生的调查收集的。
在PSP中开始使用培米替尼的26例患者中，我们收到了18例（69%）的调查回复。他们的中位年龄为５７岁；67%为女性；61%患有IV期疾病；83%患有肝内CCA。在使用培米替尼之前，44%的患者接受了部分肝切除术，66%的患者接受了2-4线先前的全身治疗。所有患者均接受铂类方案作为不可切除/转移性疾病的一线治疗。培米替尼的中位随访时间为12.6（范围：2.3-28.4）个月，他们的中位真实世界无进展生存期（rwPFS）为12.1个月（95%CI 7.2-未达到）。医生评估的客观缓解率和疾病控制率分别为56%和89%。对于9例停用培米替尼的患者，中位治疗持续时间为10.6个月（范围：0.8-21.7）。疾病进展是停药的最常见原因（89%）。没有患者因不良事件而停药。
在这个加拿大PSP队列中，使用培米替尼报告的客观缓解率、疾病控制率和无进展生存期与2期FIGHT-202试验中的情况相当。
