Araki Tomonori, Kawahira Machiko, Shimokawa Mototsugu, Otsuka Taiga, Hayashi Kohei, Sonoda Yuki, Honda Takuya, Nakao Kazuhiko, Shibuki Taro, Nakazawa Junichi, Arima Shiho, Fukahori Masaru, Miwa Keisuke, Koga Futa, Ueda Yujiro, Kubotsu Yoshihito, Makiyama Akitaka, Shimokawa Hozumi, Takeshita Shigeyuki, Nishikawa Kazuo, Komori Azusa, Otsu Satoshi, Hosokawa Ayumu, Sakai Tatsunori, Oda Hisanobu, Arita Shuji, Taguchi Hiroki, Tsuneyoshi Kengo, Kawaguchi Yasunori, Fujita Toshihiro, Sakae Takahiro, Nio Kenta, Ide Yasushi, Ureshino Norio, Shirakawa Tsuyoshi, Mizuta Toshihiko, Mitsugi Kenji
Department of Gastroenterology and Hepatology, Graduate School of Biomedical Sciences, Nagasaki University, Nagasaki, Japan,
Department of Gastroenterology, Kagoshima Kouseiren Hospital, Kagoshima, Japan.
Oncology. 2025;103(7):569-579. doi: 10.1159/000542137. Epub 2024 Oct 19.
Fluorouracil, leucovorin, irinotecan, and oxaliplatin (FOLFIRINOX) improve overall survival (OS) and progression-free survival (PFS) in patients with pancreatic cancer, compared with gemcitabine (GEM). However, whether PFS is a surrogate marker of OS in pancreatic cancer chemotherapy focusing on FOLFIRINOX or GEM plus nab-paclitaxel remains unknown. We aimed to verify whether PFS can be a surrogate marker of OS in prognosis prediction.
This was an integrated analysis of the NAPOLEON study and retrospective cohort of the NAPOLEON-2 study - a multicenter observational study conducted in Japan, using real-world data. The primary and secondary endpoints were OS and PFS, respectively. The correlation between OS and PFS in first- and second-line treatments was assessed using Method of Moments estimation. An analysis was performed in patients with confirmed OS at the end of follow-up. The NAPOLEON-2 cohort included only patients who received 5-fluorouracil, leucovorin, and nanoliposomal irinotecan (NFF) as second-line treatment.
Among 479 patients, the correlation between PFS and OS from first- and second-line chemotherapies was calculated in 310 and 225 patients, respectively. The R-squared values for the correlation between PFS and OS from first- and second-line chemotherapies were 0.74 and 0.76, respectively. There was no statistically significant difference in first-line treatment between the FOLFIRINOX and GEM plus nab-paclitaxel groups (p = 0.92). Therefore, the FOLFIRINOX group may not have shown a stronger correlation than the NFF group.
PFS can be a surrogate marker of OS in first- and second-line therapies. Appropriate prognostic estimation might contribute to proper treatment selection.
与吉西他滨（GEM）相比，氟尿嘧啶、亚叶酸钙、伊立替康和奥沙利铂（FOLFIRINOX）可改善胰腺癌患者的总生存期（OS）和无进展生存期（PFS）。然而，在以FOLFIRINOX或GEM联合纳米白蛋白结合型紫杉醇为主的胰腺癌化疗中，PFS是否为OS的替代标志物仍不清楚。我们旨在验证PFS在预后预测中是否可作为OS的替代标志物。
这是一项对NAPOLEON研究和NAPOLEON - 2研究回顾性队列的综合分析——一项在日本进行的多中心观察性研究，使用真实世界数据。主要和次要终点分别为OS和PFS。采用矩估计法评估一线和二线治疗中OS与PFS之间的相关性。对随访结束时确认有OS的患者进行分析。NAPOLEON - 2队列仅包括接受5 - 氟尿嘧啶、亚叶酸钙和纳米脂质体伊立替康（NFF）作为二线治疗的患者。
在479例患者中，分别在310例和225例患者中计算了一线和二线化疗的PFS与OS之间的相关性。一线和二线化疗中PFS与OS相关性的R平方值分别为0.74和0.76。FOLFIRINOX组和GEM联合纳米白蛋白结合型紫杉醇组在一线治疗中无统计学显著差异（p = 0.92）。因此，FOLFIRINOX组可能未显示出比NFF组更强的相关性。
PFS可作为一线和二线治疗中OS的替代标志物。适当的预后估计可能有助于正确的治疗选择。
