Supplementary Material for: Multicenter retrospective analysis of original versus modified FOLFIRINOX in metastatic pancreatic cancer: Results of the NAPOLEON study

Introduction: Original FOLFIRINOX (oFFX) is more toxic than other regimens for patients with metastatic pancreatic cancer (mPC); therefore, a modified FFX (mFFX) regimen with a reduced dosage has been used in Japanese clinical practice. However, very few studies have compared these two regimens. Methods: This study was conducted as part of a multicenter retrospective study of 318 patients with mPC across 14 centers in Japan (NAPOLEON study). To control for potential bias and confounders, we conducted a propensity score-adjusted analysis of patient characteristics and clinical outcomes. Results: oFFX and mFFX were administered to 48 and 54 patients. More patients with younger age and poorer performance status were included in the oFFX group. The overall survival (OS; median, 11.6 vs. 11.3 months; hazard ratio (HR), 0.91; 95% confidence interval (CI), 0.60-1.40; p=0.67), progression-free survival (PFS) (median, 6.3 vs. 5.7 months; HR, 0.85; 95% CI, 0.56-1.28; p=0.44), and overall response rate (29 vs. 26%, p=0.71) were not significantly different for the oFFX and mFFX groups. Thrombopenia and liver dysfunction were significantly more frequent with oFFX than with mFFX. The median received dose intensity of CPT-11 was higher with oFFX than with mFFX (299 vs. 270 mg/m2/week, p <0.01). The propensity score-adjusted analysis revealed no statistically significant differences in OS and PFS between the two groups. Conclusion: In our data, there was no significant difference in efficacy between mFFX and oFFX, and mFFX has fewer adverse events.

引言：相较于其他治疗方案，原始FOLFIRINOX方案(Original FOLFIRINOX, oFFX)对转移性胰腺癌(metastatic pancreatic cancer, mPC)患者的毒性更强，因此日本临床实践中已采用剂量下调的改良型FOLFIRINOX方案(mFFX)。然而，直接对比这两种方案的临床研究极为匮乏。
方法：本研究作为日本14家中心共318例转移性胰腺癌患者的多中心回顾性研究(NAPOLEON研究)的一部分开展。为控制潜在偏倚与混杂因素，我们针对患者基线特征及临床结局进行了倾向得分校正分析。
结果：最终48例患者接受oFFX方案治疗，54例患者接受mFFX方案治疗。oFFX组中，年轻患者及体能状态较差的患者占比更高。两组患者的总生存期(overall survival, OS；中位生存期：11.6 vs 11.3个月；风险比(hazard ratio, HR)=0.91；95%置信区间(confidence interval, CI)：0.60~1.40；p=0.67)、无进展生存期(progression-free survival, PFS；中位生存期：6.3 vs 5.7个月；HR=0.85；95%CI：0.56~1.28；p=0.44)及总体缓解率(29% vs 26%，p=0.71)均无显著统计学差异。与mFFX组相比，oFFX组的血小板减少症与肝功能异常发生率显著更高。伊立替康(CPT-11)的中位给药剂量强度在oFFX组中更高(299 vs 270 mg/m²/周，p<0.01)。倾向得分校正分析显示，两组患者的OS与PFS仍无统计学显著差异。
结论：本研究数据表明，mFFX方案与oFFX方案的疗效无显著差异，且mFFX方案的不良事件发生率更低。