Abstract 142P
Background
Advanced cholangiocarcinoma (CCA) cases require genetic testing, with 13% presenting isocitrate dehydrogenase 1 (IDH-1) mutations. The standard treatment, ivosidenib, is expensive and shows limited efficacy in relapsed or advanced cases. Cost-effective alternatives are essential for CCA patients. Previous evidence suggests IDH1 mutations often correlate with hypervascular imaging features, potentially due to mutation-induced angiogenesis pathway activation. Lenvatinib, a multi-kinase inhibitor targeting various growth factor receptors, has shown promise in solid tumors. This research aims to identify a more affordable treatment approach for patients with IDH1 mutations.
Methods
This retrospective study examined metastatic or advanced CCA patients treated with lenvatinib at our institute. We assessed tumor vascularity using the tumor-to-liver contrast enhancement ratio (TLCER) on CT scans. Hypervascularity was defined by a TLCER exceeding 1 and rim enhancement. We compared treatment responses, OS, and PFS between two groups: patients with hypervascular tumors or IDH-1 mutations and those with hypovascular tumors without IDH-1 mutations.
Results
We enrolled 41 patients: 15 with hypervascularity or IDH-1 mutations and 26 with hypovascular tumors without IDH-1 mutations. The hypervascular or IDH-1 mutation group showed significantly longer median OS (10.3 vs. 4.7 months, P = 0.015) and PFS (7.1 vs. 3.5 months, P = 0.004), with better disease control rates (DCR, 85.7% vs. 61.5%, P = 0.055) and objective response rates (ORR, 42.9% vs. 15.4% P = 0.029). Next-generation sequencing in 20 patients revealed IDH-1 mutations in seven cases, with these tumors trending towards hypervascularity (57.1% vs. 30.8%, P = 0.128). IDH-1 mutated tumors also showed trends towards improved treatment response and survival, though not statistically significant.
Conclusions
Lenvatinib could be a safe and cost-effective treatment for metastatic/advanced CCA, particularly in patients with IDH-1 mutations. Among IDH-1 mutated cases, those with tumor hypervascularity on abdominal CT scans demonstrated an even more favorable treatment response to lenvatinib.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.