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   Targeted Mechanism of Action of Pemigatinib
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Sep 02, 2025

Pemigatinib is a novel targeted drug used for the treatment of cholangiocarcinoma and myeloid/lymphoid neoplasms. By inhibiting the activation of the FGFR1 receptor and interfering with the abnormal signaling pathways in tumor cells, pemigatinib suppresses tumor growth and metastasis, demonstrating significant clinical efficacy.

Targeted Mechanism of Action of Pemigatinib

Pemigatinib is mainly indicated for the treatment of cholangiocarcinoma and myeloid/lymphoid neoplasms harboring FGFR1 gene rearrangements. FGFR1 (Fibroblast Growth Factor Receptor 1) is a tyrosine kinase receptor belonging to the fibroblast growth factor receptor family, which is involved in processes such as cell growth, differentiation, and angiogenesis. The mechanism of action of pemigatinib primarily involves the inhibition of the FGFR1 signaling pathway.

FGFR1 Inhibition

Pemigatinib is an FGFR inhibitor that exerts its effect primarily by specifically inhibiting the kinase activity of FGFR1. Abnormal enhancement of FGFR1 kinase activity is associated with the development and progression of certain tumors. The inhibitory effect of pemigatinib can reduce the overactivation of the FGFR1 signaling pathway, thereby interfering with the growth and differentiation of tumor cells.

Inhibition of Tumor Angiogenesis

The FGFR signaling pathway also plays a crucial role in the process of angiogenesis. The inhibitory effect of pemigatinib may help suppress tumor angiogenesis, thereby blocking the blood supply to the tumor and restricting tumor growth.

Cell Cycle Regulation

The FGFR signaling pathway is involved in the regulation of the cell cycle, including promoting cells to enter the S phase (DNA synthesis phase) and G2/M phase (pre-mitotic phase). By inhibiting FGFR1, pemigatinib may affect the cell cycle regulation of tumor cells and prevent their excessive proliferation.

It should be noted that the specific therapeutic mechanism of pemigatinib may also be affected by individual differences and tumor types. Professional medical guidance is essential before initiating treatment.

What Precautions Should Patients with Hepatic Impairment Take When Using Pemigatinib?

Before using pemigatinib for the treatment of cholangiocarcinoma and myeloid/lymphoid neoplasms, both patients and physicians should be aware of certain important precautions. The following are key considerations for patients with hepatic impairment when using pemigatinib:

Clinical Monitoring

During treatment with pemigatinib, regular liver function tests are of great importance. These tests include monitoring of serum transaminases (AST and ALT), total bilirubin, alkaline phosphatase, and other indicators. If abnormal liver function is detected, dosage adjustment or treatment discontinuation may be necessary.

Dosage Adjustment

For patients with hepatic impairment, dosage adjustment of pemigatinib may be required. The dosage adjustment should be made by physicians based on the patient's specific condition, and close monitoring and further adjustments should be conducted during the treatment course.

Close Observation of Adverse Reactions

During the use of pemigatinib, patients should closely observe any adverse reactions that may be related to hepatic impairment, such as fatigue, jaundice, and upper abdominal discomfort. If these symptoms occur, patients should promptly inform their physicians.

Important Reminder: Patients must use pemigatinib strictly in accordance with the physician's advice and prescription. Do not adjust the dosage or discontinue the medication on your own, as this may affect the treatment efficacy or lead to adverse reactions. Regular monitoring should be performed, and any abnormal symptoms should be reported in a timely manner. This helps to ensure the effectiveness of the treatment while minimizing potential adverse reactions.

Note: For internal discussion among medical personnel only. For specific medication, please consult the attending physician. Drug information may change over time. For the latest information, we recommend adding a medical consultant or consulting for free online.
pemazyre(pemigatinib)
1. Cholangiocarcinoma 2. Myeloid/lymphoid neoplasms with FGFR1 rearrangement
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