[Chronic myeloid leukemia – a model disease for targeted therapy]

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• Chronic myeloid leukemia (CML) is a malignancy linked to the Philadelphia chromosome.
• Targeted therapies, including imatinib and other tyrosine kinase inhibitors (TKIs), have revolutionized CML treatment.

Purpose of the Study:

• To review the impact of TKIs on CML outcomes.
• To explore the potential for treatment discontinuation in patients with deep molecular responses.
• To discuss the implications of long-term TKI therapy and cost-effectiveness.

Main Methods:

• Review of existing literature on CML targeted therapy.
• Analysis of TKI efficacy and adverse event profiles.
• Consideration of treatment discontinuation strategies.

Main Results:

• Continuous TKI administration has converted CML into a manageable chronic condition.
• A subset of patients achieving deep molecular responses may be able to discontinue TKI treatment without relapse.
• Long-term TKI therapy can be associated with adverse events impacting quality of life.

Conclusions:

• TKIs have dramatically improved CML patient prognosis.
• Treatment-free remission is a possibility for some CML patients.
• The advent of generic imatinib enhances the cost-effectiveness of CML management.