Safety and efficacy of pemigatinib in patients with cholangiocarcinoma: a systematic review
Pemigatinib demonstrated an overall objective response rate (ORR) of 43.2% in cholangiocarcinoma patients with FGFR2 fusions or rearrangements.
Background: Cholangiocarcinoma (CCA) is an aggressive bile duct cancer with limited therapeutic options and poor prognosis. Pemigatinib, a selective FGFR inhibitor, has emerged as a promising targeted therapy for CCA patients harboring FGFR2 fusions or rearrangements. This systematic review evaluated the safety and efficacy of pemigatinib in this patient population. Methods: A comprehensive systematic review was conducted across PubMed, Scopus, Embase, Cochrane Library, and Web of Science. Five studies involving 459 patients met the inclusion criteria. Results: Pemigatinib demonstrated an overall objective response rate (ORR) of 43.2%, with a complete response (CR) achieved in 3% of patients. Stable disease was observed in 36.9% of patients, while 14.9% experienced disease progression. Median progression‑free survival (PFS) varied across studies. The most common adverse effects (AEs) included hyperphosphatemia (48%), diarrhea (28.6%), fatigue (33%), and dry eyes (20.1%). Conclusions: This systematic review suggests pemigatinib has modest therapeutic efficacy in CCA patients. The ORR of less than 50% highlights the potential need for combination or sequential therapies to improve outcomes. Close monitoring and management of AEs, particularly hyperphosphatemia, are crucial for optimizing treatment. Further large‑scale randomized trials and research are warranted to identify predictive biomarkers and optimize pemigatinib‑based treatment strategies for CCA patients with FGFR2 alterations.
The modest efficacy of pemigatinib underscores the need for combination strategies and emphasizes careful adverse‑event management. These findings highlight gaps that future clinical trials must address to improve outcomes for cholangiocarcinoma patients with FGFR2 alterations.
Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.
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