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

BACKGROUND Health‐related quality of life (HRQOL) in patients with chronic‐phase chronic myeloid leukemia (CML) is important because of the requirement for long‐term treatment. This study assessed HRQOL in bosutinib‐treated patients with Philadelphia chromosome–positive CML and resistance or intolerance to 1 (chronic‐phase second‐line [CP2L]) or more (chronic‐phase third‐line [CP3L]) tyrosine kinase inhibitors who had 264 weeks or more of follow‐up (ClinicalTrials.gov identifier NCT00261846). METHODS Patient‐reported HRQOL was assessed with the EuroQol 5‐Dimensions Questionnaire (EQ‐5D) and the Functional Assessment of Cancer Therapy–Leukemia (FACT‐Leu). RESULTS In total, 284 and 119 patients composed the CP2L and CP3L cohorts, respectively. At treatment completion, more than 50% of the patients in the CP2L and CP3L cohorts completed the EQ‐5D and FACT‐Leu assessments. The EQ‐5D and EQ‐5D visual analog scale scores were stable in both cohorts throughout treatment. The mean FACT‐Leu scores were generally stable over time but were lower in magnitude in the CP3L cohort versus the CP2L cohort. The FACT‐Leu scale scores of a subset of patients with chronic diarrhea (CP2L, n = 101; CP3L, n = 30) were similar to the scores of the larger cohorts. Minimally important differences (MIDs) from baseline for the FACT‐Leu scale scores were observed for the following: emotional well‐being (EWB), Functional Assessment of Cancer Therapy–General (FACT‐G) Total, FACT‐Leu Total, and Functional Assessment of Cancer Therapy Trial Outcome Index (FACT‐TOI) in the CP2L cohort and FACT‐Leu Total in the CP3L cohort. Among patients with chronic diarrhea, MIDs were observed for EWB, FACT‐G Total, FACT‐Leu Total, and FACT‐TOI in the CP2L cohort and for EWB, FACT‐G Total, and FACT‐Leu Total in the CP3L cohort. CONCLUSIONS HRQOL was maintained with long‐term bosutinib treatment for patients with CP2L and CP3L CML. Cancer 2018;124:587‐95. © 2017 The Authors. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society. This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
Health‐related quality of life (assessed with the Functional Assessment of Cancer Therapy–Leukemia) and the general health status (assessed with the EuroQol 5‐Dimensions Questionnaire) were largely maintained in chronic‐phase chronic myeloid leukemia patients receiving bosutinib for 264 weeks or longer. Health‐related quality of life was maintained by patients who receive bosutinib as a second‐ or third‐line treatment for chronic‐phase chronic myeloid leukemia.