Achieving optimal response at 12 months is associated with a better health-related quality of life in patients with chronic myeloid leukemia: a prospective, longitudinal, single center study

To assess the relationship between responses within 1 year and health-related quality of life (HRQoL) outcomes by exploring profiles of patients with CML-CP who were treated with front-line imatinib or nilotinib.

A prospective, longitudinal, single-center study was conducted to assess the response to treatment with imatinib or nilotinib and the HRQoL profile of patients who were newly diagnosed with CML in chronic phase enrolled in the ENESTchina study.

Fifty-nine patients were randomized to receive imatinib (n = 31) or nilotinib (n = 28). With a median follow-up of 5 years, there was no difference in HRQoL profile observed between patients receiving imatinib and nilotinib. Achieving optimal response at 12 months was associated with better role limitations due to physical health problems (RP; P = 0.0019) and emotional problems (RE; P = 0.0110) and was the sole factor associated with significantly improving physical component summary over time (PCS; P = 0.0160). Achieving optimal response at 6 months had high probability of better physical functioning (PF; P = 0.0674), better social functioning (SF; P = 0.0571), and reduced role limitations due to emotional problems (RE; P = 0.0916). In addition, factors including age < 40 years, female gender, and higher level of education were also associated with better HRQoL subscale scores. However, optimal response at 3 months had no impact on HRQoL profile. The proportions of patients with failure-free survival and PFS at 5 years were significantly higher among patients who achieved optimal response at 3, 6, or 12 months than among those who did not achieve optimal response (warning or failure), and the OS rate at 5 years was significantly higher among those who achieved optimal response at 12 months. In a multivariate analysis, treatment received (nilotinib vs imatinib) was identified as an independent factor for the achievement of optimal response at both 6 months (OR, 3.9; 95% CI, 1.0–14.9) and 12 months (OR, 5.6; 95% CI, 1.7–17.9).

Achieving optimal response at 12 months was not only associated with longer OS and reduced treatment failure rates and disease progression but also better HRQoL in newly diagnosed patients with CML-CP receiving front-line tyrosine kinase inhibitor treatment.

Chinese Clinical Trial Registry (http://​www.​chictr.​org.​cn): ChiCTR-OCH-11001699.