OBJECTIVES : In the single-arm trial FIGHT-202, patients with advanced FGFR2 translocation-positive cholangiocarcinoma treated with pemigatinib have shown a sustained response. As this trial has no comparator, unanchored indirect treatment comparisons are required to assess the overall survival (OS) and progression-free survival (PFS) gains seen for patients treated with pemigatinib compared to those treated with standard of care (SoC).

METHODS : A systematic literature review identified suitable sources of comparator data. Eight SoC treatment arms, from five different studies, were considered eligible for comparison with FIGHT-202. Endpoints of interest were OS and PFS. Covariates used for matching, based on clinical expert opinion and availability in published studies, were: age, sex, Eastern Cooperative Oncology Group performance status, and albumin levels. FGFR2 translocation status, which retrospective studies have suggested could be associated with positive prognosis in the patient population of interest was not reported in any of the identified comparator studies. Matching-adjusted indirect comparisons (MAICs) were performed. Patients in FIGHT-202 were assigned statistical weights that adjust for their over- or underrepresentation relative to that observed in each comparative evidence source.

RESULTS : Weighted Kaplan–Meier plots, median survival and Cox proportional hazard ratios (HRs) with bootstrapped confidence intervals (CI) were generated to show the relative treatment effect for OS and PFS. The weighted HRs ranged from 0.163 (95% CI: 0.099, 0.249) to 0.475 (95% CI: 0.328, 0.657) for OS and from 0.195 (95% CI: 0.117, 0.302) to 0.436 (95% CI: 0.319, 0.599) for PFS.

CONCLUSIONS : Based on the available evidence and on the MAIC, patients treated with pemigatinib are expected to experience longer time in remission and survival than cholangiocarcinoma patients treated according to SoC. The robustness and interpretation of the MAIC results is subject to known methodological limitations, including the unknown FGFR2 status of and impact on patients recruited in the available comparator studies.