Quality of Life During Early Tyrosine Kinase Inhibitor Treatment As Self-Reported by Chronic Myeloid Leukemia Patients Participating in a Prospective Observational Study (SIMPLICITY)

Over the past decade, oral BCR-ABL inhibitors including imatinib, dasatinib and nilotinib have revolutionized chronic myeloid leukemia (CML) treatment. Clinical trials have provided evidence of improved outcomes with all three agents in the treatment of CML. However, limited information is available regarding the impact of disease and treatment on quality of life (QoL) in CML patients.

SIMPLICITY is an ongoing 5-year USA/European prospective, observational cohort study (ClinicalTrials.gov ID: NCT01244750). Eligibility criteria include: newly diagnosed CML-CP, age ≥18 years and therapy with 1st-line imatinib, dasatinib or nilotinib at participating medical centers. The study aims to capture information on the use of these agents in clinical practice, including their impact on patient QoL. Through self-administered patient-reported outcomes (PRO) questionnaires, QoL and treatment-adherence data are being collected at 6-month intervals throughout study follow-up. Data from available initial PRO assessments of patients enrolled as of August 2011 are presented: Cancer Treatment Satisfaction Questionnaire (CTSQ), MD Anderson Symptom Inventory - CML (MDASI-CML), FACT-G (Functional Assessment of Cancer Therapy-General) and Morisky Medication Adherence Scale - 8 item (MMAS-8).

A total of 74 patients currently enrolled was included in this analysis of which 79.7% (n=59) received imatinib, 10.8% (n=8) received nilotinib and 9.5% (n=7) received dasatinib as 1st-line CML treatment. Median age was 57 years (range 19–94 years); a slightly higher proportion of patients were female (59.5%; n=44); and median time to completion of the PRO questionnaires from initiation of treatment was 383.5 days (range 4-1,225 days). Prior to completion of the PROs, mean treatment exposure was approximately 18 months for imatinib, 5 months for nilotinib and 4 months for dasatinib. Summary scores for the CTSQ, MDASI-CML and FACT-G are presented in Table 1. Due to small sample size, differences in scores between treatments were not statistically tested. At time of enrollment in the study, treatment satisfaction in this cohort was high (SWT score ± standard deviation [SD] =91.2 ±10.3). QoL, as measured by the FACT-G total and sub-scale scores, also indicated positive physical, social, emotional and functional well-being. Mean MDASI scores for Symptom Interference and Symptom Distress suggested the impact of symptoms was mild to moderate. From the MMAS-8, 71.8% (n=51) of patients reported medium/high adherence to their CML medication (scoring between 6–8 on an 8-point scale).

Preliminary QoL evaluation suggests that newly diagnosed CML-CP patients are generally satisfied with treatment and report overall good health, although approximately 30% reported low adherence to their CML treatment. Initial assessment of the enrolled cohort suggests variance in treatment adherence patterns, as well as differences in the impact of symptoms by treatment; however, as these differences were not statistically tested, no firm conclusions can be drawn at this time. As patient enrollment continues, comparative effectiveness of these CML treatments and differences in QoL outcomes will be further observed.

Cortes:Novartis: Research Funding; BMS: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Ariad: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Novartis: Consultancy. Mauro:BMS: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding; Ariad: Research Funding. Goldberg:Novartis Pharmaceutical: Honoraria, Research Funding, Speakers Bureau; Bristol Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Paquette:BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria. Khoury:BMS: Membership on an entity's Board of Directors or advisory committees. Hirji:BMS: Employment. Wagner:BMS: Employment. Joo:BMS: Employment. Davis:BMS: Employment.