Economic and clinical outcomes among patients with cholangiocarcinoma receiving pemigatinib with or without history of cancer of unknown primary.

BACKGROUND: There is limited evidence regarding the economic burden, treatment patterns, and overall survival (OS) of patients with cholangiocarcinoma (CCA) and cancer of unknown primary (CUP) who initiated the FGFR inhibitor pemigatinib. PATIENTS AND METHODS: We used the Komodo Healthcare Map to identify patients with CCA who initiated pemigatinib between 4/17/2020 and 5/31/2023. Follow-up began at initiation and lasted ≥ 1 month. Outcomes included health care resource utilization (HCRU), costs, treatment patterns, and OS. RESULTS: Two hundred twenty-one patients were included: 78 patients (35.3%) with CUP (median follow-up, 5.9 months) and 143 patients (64.7%) without CUP (median follow-up, 7.3 months). Pemigatinib was similarly well-tolerated in CUP vs non-CUP. Discontinuation was observed in 43.6% vs 49.0% (P = .445). Medication possession ratio ≥ 0.80 was achieved by 71.6% vs 67.2% (P = .504). CUP was associated with significantly higher prevalence of metastatic disease (100.0% vs 63.6%), per patient per month (PPPM) ambulatory HCRU (8.2 vs 5.5), and ambulatory costs ($8584 vs $5308). Medical costs averaged $13 444 vs $9881 PPPM for CUP and non-CUP, respectively (P = .066). Median OS was significantly shorter with CUP (10.2 vs 30.7 months). CONCLUSION: Although pemigatinib was similarly well-tolerated regardless of CUP status, patients with CUP incurred greater ambulatory burden and had poorer OS. Patients with CUP were more likely to have evidence of metastatic disease at pemigatinib initiation, which may help explain these results. With the advent of targeted treatments for gene-altered CCA, reflexive genomic testing should be encouraged for all patients with CUP.