Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy.
Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy; Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France.
Eur J Cancer. 2024 Mar;200:113587. doi: 10.1016/j.ejca.2024.113587. Epub 2024 Feb 6.
Pemigatinib is approved for patients with pretreated, locally advanced or metastatic CCA harboring FGFR2 rearrangements or fusions. We aim to assess the effectiveness and safety of pemigatinib in real-world setting.
A joint analysis of two multicentre observational retrospective cohort studies independently conducted in France and Italy was performed. All consecutive FGFR2-positive patients affected by CCA and treated with pemigatinib as second- or further line of systemic treatment in clinical practice, within or outside the European Expanded Access Program, were included.
Between July 2020 and September 2022, 72 patients were treated with pemigatinib in 14 Italian and 25 French Centres. Patients had a median age of 57 years, 76% were female, 81% had ECOG-PS 0-1, 99% had intrahepatic CCA, 74% had ≥ 2 metastatic sites, 67% had metastatic disease at diagnosis, while 38.8% received ≥ 2 previous lines of systemic treatment. At data cut-off analysis (April 2023), ORR and DCR were 45.8% and 84.7%, respectively. Median DoR was 7 months (IQR: 5.8-9.3). Over a median follow-up time of 19.5 months, median PFS and 1-year PFS rate were 8.7 months and 32.8%. Median OS and 1-year OS rate were 17.1 months and 60.6%. Fatigue (69.4%), ocular toxicity (68%), nail toxicities (61.1%), dermatologic toxicity (41.6%) hyperphosphataemia (55.6%), stomatitis (48.6%), and diarrhea (36.1%) were the most frequent, mainly G1-G2 AEs. Overall incidence of G3 AEs was 22.2%, while no patient experienced G4 AE. Dose reduction and temporary discontinuation were needed in 33.3% and 40.3% of cases, with 1 permanent discontinuation due to AEs.
These results confirm the effectiveness and safety of pemigatinib in a real-world setting.
培米替尼获批用于治疗既往接受过治疗、局部晚期或转移性胆管癌（CCA）且存在 FGFR2 重排或融合的患者。我们旨在评估培米替尼在真实世界环境中的有效性和安全性。
对在法国和意大利独立进行的两项多中心观察性回顾性队列研究进行联合分析。所有 FGFR2 阳性的 CCA 患者均接受培米替尼治疗，这些患者在欧洲扩展准入计划内或外，接受培米替尼作为二线或后线系统治疗。
在 2020 年 7 月至 2022 年 9 月期间，14 家意大利中心和 25 家法国中心共 72 例患者接受培米替尼治疗。患者的中位年龄为 57 岁，76%为女性，81%ECOG-PS 评分为 0-1，99%为肝内 CCA，74%有≥2 个转移部位，67%在诊断时即发生转移，38.8%的患者接受过≥2 线的系统治疗。在数据截止分析（2023 年 4 月）时，ORR 和 DCR 分别为 45.8%和 84.7%。中位缓解持续时间为 7 个月（IQR：5.8-9.3）。在中位随访时间为 19.5 个月时，中位 PFS 和 1 年 PFS 率分别为 8.7 个月和 32.8%。中位 OS 和 1 年 OS 率分别为 17.1 个月和 60.6%。最常见的不良反应（AE）包括乏力（69.4%）、眼部毒性（68%）、指甲毒性（61.1%）、皮肤毒性（41.6%）、高磷血症（55.6%）、口腔炎（48.6%）和腹泻（36.1%），主要为 G1-G2 AE。总体 3 级 AE 发生率为 22.2%，无 4 级 AE 发生。因 AE 需减量和暂时停药的比例分别为 33.3%和 40.3%，因 AE 导致永久性停药 1 例。
这些结果证实了培米替尼在真实世界环境中的有效性和安全性。
