The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors

Abstract

The development of the oral tyrosine kinase inhibitors (TKIs) to treat chronic myeloid leukemia (CML) is one of the great triumphs of cancer research. Although the efficacy of TKIs has dramatically improved the disease-specific overall survival rate, the prevalence of CML is increasing worldwide. Currently, CML patients receive prolonged (even lifelong) treatment, and over the last decade, clinical decision making has become challenging. Therefore, consideration of the effects of TKI therapies on patients' quality of life (QoL) and symptom burden (ie, patient-reported outcomes [PROs]) is now critical to more robustly inform patient care and improve health care quality. Over the last 5 years, a number of studies have generated valuable PRO data, for example, on long-term QoL effects of imatinib therapy or symptom burden of patients switching from imatinib to second-generation TKIs. PRO findings are important, as they provide a unique patient perspective on the burden of the disease and treatments effects. We will review main evidence-based data on the use of PROs in clinical research and highlight the importance of methodological rigor of PRO assessment. Also, we will describe the potential value of using PRO assessment in routine clinical practice, for example, to facilitate timely management of side effects. Areas for future research will also be discussed. Learning Objectives• List main evidence-based data on the impact of tyrosine kinase inhibitors on patients' quality of life and symptom burden • Describe the value of quality of life assessment in CML clinical research • Illustrate the importance of integrating quality of life assessment into routine practice and the relationship between quality of life assessment and adherence to therapy

Dasatinib cessation after deep molecular response exceeding 2 years and natural killer cell transition during dasatinib consolidation

“…Recent data have revealed that an increasing number of patients may gradually achieve a deep molecular response (DMR) if they continue to use second‐generation TKI for several years . However, increasing concern surrounds the long‐term adverse effects and financial burden associated with TKI …”

“…6, 7 However, increasing concern surrounds the long-term adverse effects and financial burden associated with TKI. [8][9][10][11][12] To address these concerns, the possibility of imatinib cessation has been extensively studied. In a prospective study first presented by a French group (STIM study), approximately 39% of CML patients who maintained DMR for ≥2 years while using first-line imatinib could safely stop taking the drug after >6 months, 13 although patients who had received therapy for >5 years or had a low Sokal score were significantly more successful.…”

Long‐term patient‐reported outcomes from an open‐label safety and efficacy study of bosutinib in Philadelphia chromosome–positive chronic myeloid leukemia patients resistant or intolerant to prior therapy

“…Additional assessments that specifically address disease and treatment‐related symptoms, such as the MD Anderson Symptom Inventory for CML, in conjunction with HRQOL instruments could also be used in future studies of bosutinib to provide greater insight into the cumulative impact of bosutinib‐related AEs on HRQOL. For additional information on quality‐of‐life assessment in patients with CML receiving TKIs, the reader is referred to a comprehensive review by Efficace and Cannella …”

“…For additional information on quality-of-life assessment in patients with CML receiving TKIs, the reader is referred to a comprehensive review by Efficace and Cannella. 26 Because CML patients are living longer with TKIs, they may be on treatment for long periods of time. Therefore, it is important to assess the long-term impact of treatment on patient HRQOL.…”

Chronic myeloproliferative disorders: is quality-of-life the new goal?

“…In particular, the improvement in survival seen in CML after the use of targeted drugs, now approaching that of general population 18 , has brought up the need for investigators to explore QoL, because it directly affects patient compliance to lifelong treatment. In this context, even low-grade adverse effects can substantially impair daily life of CML patients and, therefore, negatively affect adherence to therapy and clinical response 19,20 . From this perspective, the role of the patient is becoming increasingly relevant, and the patient's reported outcome assessment (PRO) is rapidly changing clinical and research practice in the setting of hematologic neoplasms.…”