Chronic myeloid leukemia (CML) is characterized by the presence of a constitutively active chimeric Bcr-Abl kinase, a product of the BCR-ABL1 chimeric gene. Clinical outcomes have been greatly improved with the use of tyrosine kinase inhibitors (TKIs) targeting Bcr-Abl kinase, and also associated with periodic and chronic adverse events (AEs) that can substantially impact patients’ quality of life (QoL), adherence to therapy and treatment success. Although TKI therapies have been widely used for more than two decades, there is currently little knowledge on time points, transience and cumulative effects of all available TKIs as the periodic and long-time toxicity of available therapies and their impact on patient QoL have only been assessed superficially. Due to this unmet need, this study proposes to obtain toxicity and QoL data from CML patients treated with TKIs and take into consideration all stages of the disease and variable therapy doses, irrespective of the treatment outcome.
This research aims to describe short-term and long-term toxicity (incidence, transience and cumulation) identified in all CML patients treated with TKIs and its correlation with QoL, regardless of the treatment outcome. This retrospective, non-interventional study will obtain toxicity and QoL data from CML patients treated with TKIs which will consider all disease stages and variable therapy doses. Data will be sourced from academic and commercial CML clinical studies where Common Terminology Criteria for Adverse Events (CTCAE) and patient-reported outcome (PRO) data are available. The data will be analyzed and reported in a descriptive manner. Factors such as side effects and symptoms will be described by frequency or presence/absence and factors such as timepoints will be reported in a quantitative manner.
Access to a larger dataset of CML patients through the HARMONY Alliance Big Data Platform will offer a better insight into when and how often these side effects occur, resolve and accumulate. By understanding the periodic, short-term and long-term toxicity and side effect burden, this research can help to determine the optimum order in which patients are prescribed drugs and optimize the outcome of TKI treatment.