Akkus Erman, Yasar Hatime Arzu, Rimassa Lorenza, Lamarca Angela
Department of Medical Oncology, Faculty of Medicine, Ankara University, Ankara, Turkey.
Ankara University Cancer Research Institute, Ankara, Turkey.
Target Oncol. 2025 May;20(3):389-403. doi: 10.1007/s11523-025-01142-8. Epub 2025 Apr 13.
The efficacy and safety of pemigatinib in advanced cholangiocarcinoma (aCCA) were presented in phase I-II trials and retrospective reports, with small sample sizes and variable results.
A systematic literature search included studies investigating the efficacy/safety of pemigatinib in aCCA harboring FGFR fusions/rearrangements. Primary outcomes were objective response rate (ORR) and treatment-related adverse events (AEs). A pooled proportion meta-analysis was performed.
Three hundred and twenty-seven patients in eight studies were included (three phase-II, one phase-I/II, two phase-I, and two retrospective). In the pooled analyses, the median age was 58.9 years (95% confidence interval (CI): 51.9-65.8); 33.4% (95% CI: 28.1-39.0) were male. Pemigatinib was the second-line treatment in 58.5% (95% CI: 52.7-64.1) and was beyond second-line in the remaining. ORR was 42.2% (95% CI: 35.9-48.7) (I:48.4%) and disease control rate (DCR) was 86.5% (95% CI: 81.6-90.5) (I: 58.8%). Median progression-free survival (PFS) was 7.8 months (95% CI: 6.2-9.4) (I: 11.6%). Two studies reported overall survival (OS) (median 17.5 and 17.1 months). The most common AEs (any grade) were hyperphosphatemia (46%), dysgeusia (33.2%), alopecia (31.4%), fatigue (30.9%), stomatitis (28.5%), and diarrhea (27.5%). Cumulative eye and nail toxicities were observed in 32.5% and 40.9%, and retinal detachment in 5.5%.
This analysis emphasizes the FGFR alteration testing and pemigatinib use in the second-line and beyond treatment of aCCA.
REGISTRATION ID (PROSPERO): CRD42024627459.
培米替尼在晚期胆管癌（aCCA）中的疗效和安全性已在I-II期试验及回顾性报告中有所呈现，但样本量较小且结果各异。
进行系统文献检索，纳入研究培米替尼在携带FGFR融合/重排的aCCA中的疗效/安全性的研究。主要结局为客观缓解率（ORR）和治疗相关不良事件（AE）。进行合并比例荟萃分析。
八项研究中的327例患者被纳入（三项II期、一项I/II期、两项I期和两项回顾性研究）。在合并分析中，中位年龄为58.9岁（95%置信区间（CI）：51.9 - 65.8）；33.4%（95% CI：28.1 - 39.0）为男性。培米替尼在58.5%（95% CI：52.7 - 64.1）的患者中作为二线治疗，其余患者为二线以上治疗。ORR为42.2%（95% CI：35.9 - 48.7）（I²：48.4%），疾病控制率（DCR）为86.5%（95% CI：81.6 - 90.5）（I²：58.8%）。中位无进展生存期（PFS）为7.8个月（95% CI：6.2 - 9.4）（I²：11.6%）。两项研究报告了总生存期（OS）（中位值分别为17.5个月和17.1个月）。最常见的不良事件（任何级别）为高磷血症（46%）、味觉障碍（33.2%）、脱发（31.4%）、疲劳（30.9%）、口腔炎（
