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Review
. 2025 Apr 30;16(2):699-710.
doi: 10.21037/jgo-2024-923. Epub 2025 Apr 25.

Safety and efficacy of pemigatinib in patients with cholangiocarcinoma: a systematic review

Warda Rasool #  1 , Yassine Alami Idrissi #  2 , Owais Ahmad  3 , Shree Rath  4 , Fatima Saeed  5 , Mohamed Saad Sayed  6 , Bhavya Sharma  7 , Arwa Amer Ibrahim  8 , Ajanta Kumari  9 , Irfan Ullah  10 , Anwaar Saeed  2
Affiliations
Review

Safety and efficacy of pemigatinib in patients with cholangiocarcinoma: a systematic review

Warda Rasool et al. J Gastrointest Oncol. .

Abstract

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 to identify studies investigating pemigatinib in CCA patients. Five studies involving a total of 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, due to differences in patient cohorts. The most common adverse effects (AEs) included hyperphosphatemia (48%), diarrhea (28.6%), fatigue (33%), and dry eyes (20.1%).

Conclusions: This systematic review suggests that pemigatinib has modest therapeutic efficacy in CCA patients, with a considerable proportion achieving disease control. However, 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.

Keywords: Cholangiocarcinoma (CCA); FGFR inhibitors; pemigatinib; targeted therapy.

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Conflict of interest statement

Conflicts of Interest: All authors have completed the ICMJE uniform disclosure form (available at https://jgo.amegroups.com/article/view/10.21037/jgo-2024-923/coif). A.S. reports consulting or advisory board role with AstraZeneca, Bristol-Myers Squibb, Merck, Exelixis, Pfizer, Xilio therapeutics, Taiho, Amgen, Autem therapeutics, KAHR and institutional research funding from AstraZeneca, Bristol-Myers Squibb, Merck, Clovis, Exelixis, Actuate therapeutics, Incyte Corporation, Daiichi Sankyo, Five prime therapeutics, Amgen, Innovent biologics, Dragonfly therapeutics, Oxford Biotherapeutics, Arcus therapeutics, and KAHR medical, Arcus therapeutics and Daiichi Sankyo. The other authors have no conflicts of interest to declare.

Figures

Figure 1
Figure 1
Mechanism of action of pemigatinib; FGFR1–4 inhibitor. This figure has been created by the authors and incorporates images from Servier Medical Art, which is licensed under a Creative Commons Attribution 4.0 Unported License by Servier (https://creativecommons.org/licenses/by/4.0/). RAS, rat sarcoma; RAF, rapidly accelerated fibrosarcoma; MAPK, mitogen-activated protein kinases; JAK, Janus kinase; STAT, signal transducer and activator of transcription; PI3K, phosphoinositide 3 kinase; AKT, protein kinase B; mTOR, mammalian target of rapamycin.
See this image and copyright information in PMC

Comment in

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