Evaluation of real-world versus clinical trial outcomes of tyrosine kinase inhibitor therapy for chronic myeloid leukemia.

Tyrosine kinase inhibitors (TKIs) are the mainstay of treatment for chronic myeloid leukemia (CML). Patients enrolled in clinical trials investigating the safety and efficacy of TKIs in CML are generally younger, have fewer comorbidities, and are monitored differently than patients treated in the real world. This narrative literature review summarizes efficacy outcomes (complete cytogenetic response, major molecular response, and disease progression) and safety outcomes (duration of TKI therapy, TKI discontinuation rates, dosage changes, and frequently reported adverse events) from landmark clinical trials as well as real-world studies.