[Key points in selecting first-line therapy for chronic myeloid leukemia]

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• Chronic myeloid leukemia (CML) is driven by the BCR::ABL1 fusion gene.
• Tyrosine kinase inhibitors (TKIs) have revolutionized CML treatment, significantly improving survival.
• Five TKIs are approved for first-line therapy in Japan, each with unique profiles.

Purpose of the Study:

• To review current evidence and practical considerations for selecting first-line TKI therapy in chronic-phase CML.
• To discuss optimizing long-term outcomes and advancing towards individualized treatment strategies.
• To explore emerging goals such as treatment-free remission (TFR) and potential curative approaches.

Main Methods:

• Review of current clinical trial data and evidence for first-line TKIs in CML.
• Analysis of distinct efficacy and toxicity profiles of approved agents.
• Consideration of patient-specific factors influencing treatment decisions.

Main Results:

• Individualized treatment selection is crucial, considering age, comorbidities, cardiovascular risk, fertility, adherence, and financial factors.
• Treatment goals are evolving from disease control to achieving treatment-free remission (TFR).
• Low-dose and step-up TKI regimens aim to balance efficacy with tolerability and quality of life.

Conclusions:

• Optimizing first-line TKI selection is key for long-term CML management.
• Future directions include exploring immunologic determinants of TFR and novel stem cell-targeting therapies for potential cures.
• Personalized medicine approaches are advancing CML treatment towards improved outcomes and remission.