OBJECTIVES: Clinical outcomes of patients with chronic myeloid leukemia (CML) have drastically improved with tyrosine kinase inhibitors (TKIs). However, one of the main challenges is the management of intolerance experienced by patients with CML treated with TKI, resulting in poorer disease control. This targeted literature review examined the association of intolerance to TKIs in CML and clinical, economic and humanistic outcomes in a real-world setting.

METHODS: This targeted literature review identified articles between 01/2010-10/2022. Searches were conducted in PubMed using a combination of terms to identify association of TKI intolerance on difference scopes. A total of 225 unique articles were identified and screened based on title, abstract and full text; 45 articles were included in this review.

RESULTS: There was heterogeneity in treatment intolerance definition across studies, if available; most of the studies did not provide intolerance definition. The literature consistently reported that intolerance has negative impacts on clinical, economic, and humanistic outcomes. CML patients with intolerance to TKI therapies were observed with lower treatment adherence mainly due to intentional non-adherence, more dose modifications/treatment discontinuations/switches, worse clinical outcomes, higher healthcare resource utilization and costs, lower health-related quality of life, and greater work and activity impairment. Most of intolerance burdens were reported from a patient’s perspective.

CONCLUSIONS: This targeted literature review suggests that intolerance to TKIs in CML is associated with unmet needs; intolerance was an important factor contributing to the clinical, economic, and humanistic burden of the disease. Focus on prevention and management of intolerance in CML care considering patient’s perspective is critical for making more informed treatment decisions and to evaluate clinical benefits of CML treatment strategies. The heterogeneity of treatment intolerance definitions reflects the challenges in classifying intolerance in clinical practice and points to the need for consistency in definitions and clear guidelines in the US to help inform treatment decisions.