Pemazyre (pemigatinib) is a targeted therapeutic drug indicated for patients with specific types of cholangiocarcinoma. As a FGFR1/2/3 kinase inhibitor, it inhibits tumor growth by blocking abnormally activated signaling pathways.

How Effective is Pemazyre (Pemigatinib) Treatment?

Clinical Benefits

(1) Tumor Response: Some patients may achieve tumor shrinkage or stabilization.

(2) Sustained Response: Patients who respond to the treatment can maintain the therapeutic effect for a relatively long period.

(3) Accelerated Approval: Approved based on objective response rate, with further verification of survival benefits required.

Treatment Characteristics

(1) Intermittent Dosing Regimen: Administered for 14 days followed by a 7-day drug holiday.

(2) Continuous Treatment: Treatment should be continued until disease progression or occurrence of intolerable toxicity.

Suitable Populations for Pemazyre (Pemigatinib)

Genetic Verification

(1) FGFR2 mutations must be confirmed using compliant detection methods (e.g., FDA-approved companion diagnostic reagents).

(2) Common mutation types include FGFR2-BICC1 fusion (accounting for 34%) and other FGFR2 rearrangements.

Excluded Populations

(1) Cholangiocarcinoma patients with no detected FGFR2 fusion/rearrangement.

(2) Patients allergic to the active ingredient of pemigatinib (though no explicit contraindications are listed in the package insert).

Medication Monitoring for Pemazyre (Pemigatinib)

Ophthalmic Monitoring

(1) Examination Frequency: A comprehensive ophthalmic examination before treatment, followed by re-examinations every 2 months for the first 6 months, and then every 3 months thereafter.

(2) Symptom Warning: Seek medical attention immediately if symptoms such as blurred vision or floaters occur.

(3) Dry Eye Management: Routine use of artificial tears is recommended.

Blood Phosphorus Management

(1) Regular Testing: Closely monitor blood phosphorus levels during treatment.

(2) Intervention Measures: Adopt a low-phosphorus diet when blood phosphorus levels rise.

(3) Pharmacological Intervention or Dosage Adjustment: Required when there is a significant increase in blood phosphorus levels.

Adverse Reaction Monitoring

(1) Common Reactions: Including alopecia (hair loss), diarrhea, fatigue, nail changes, etc.

(2) Severe Reactions: Conditions such as hyperphosphatemia and retinopathy require timely management.

Precautions Monitoring

(1) Pregnancy Contraindication: Has embryotoxicity; patients of childbearing age must use strict contraceptive measures.

(2) Drug Interactions: Avoid concurrent use with strong CYP3A inducers/inhibitors.