Retrospective study of effect of neoadjuvant therapy on post-recurrence survival after resection of intrahepatic cholangiocarcinoma

Despite the theoretical advantages of neoadjuvant therapy (NAT) in intrahepatic cholangiocarcinoma (iCCA), its impact of post-recurrence overall survival is poorly defined. A retrospective study at the Mayo Clinic (Rochester, MN) investigated the association between NAT and post-recurrence outcomes in iCCA. The study included 343 patients with iCCA who underwent curative-intent liver resection between 2000 and 2024. NAT recipients were younger, more often treated in the contemporary era, and included a higher proportion of major resections. Targetable molecular alterations (IDH1, FGFR2) were enriched in the NAT group (48.2% versus 13.9%). NAT was associated with significantly improved post-recurrence overall survival (median 31.9 versus 16.4 months; p=0.006) and remained an independent predictor of survival in multivariable Cox regression (HR 0.506; p=0.008) even after accounting for era-dependent molecular profiling. Older age and early recurrence were independently associated with worsen post-recurrence overall survival. After recurrence, NAT recipients more frequently underwent curative-intent local therapies and advanced systemic treatments. The authors conclude that NAT was associated with improved post-recurrence overall survival in resected iCCA-patients, consistent with a potential role in biologic selection.