The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually
translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This
knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that
have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability
increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of
these patients now approaches that of the general population. While oral TKIs are now the standard of care
for CML, it should be considered that therapy is lifelong and patients are requested to take medication on
a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to
obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML,
since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously
undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses
to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring
of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom
control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing.
Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to
CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients.
This mismatch might have major clinical implications in disease management, as physicians might not be
able to early identify those patients who might be at heightened risk of poor adherence behavior. Given
these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML
routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This
hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online
platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands
of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this
platform in CML routine practice and evaluate its value in improving symptom management, quality of life,
adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented
information on the value of systematically collecting patient-reported AEs information in CML routine care.
If positive, our results will inform the development of large international randomized controlled trial (RCT)
to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI
therapies in CML patients.
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