中国慢性髓性白血病慢性期患者报告的酪氨酸激酶抑制相关不良反应及其对日常生活的影响研究

OBJECTIVE: To explore the impact of patient reported outcome of tyrosine kinase inhibitor (TKI) related side effects on daily life in Chinese patients with chronic myeloid leukemia (CML) in the chronic phase (CP). METHODS: From May to November in 2014, anonymous questionnaires were distributed to adult CML patients who were receiving TKI treatment in China. The impact of TKI-related side effects on daily life were assessed by the score of 1 (no impact) to 5 (high impact) from patient self-report. RESULTS: Data from 731 respondents in the CP who reported the score of the impact of TKI-related side effects on daily life were collected. 407 (56%) were male. The median age was 41 years (range, 18 to 88 years). 560 (77%) started TKI treatment within 1 year after diagnosis. With a median treatment duration of 3 years (range, <1 to 13 years), 549 (75%) respondents achieved a complete cytogenetic response (CCyR) and 301 (41%) achieved a complete molecular response (CMR). The most common TKI-related adverse effects were edema (n=323, 44%), fatigue (n=277, 38%), gastrointestinal disorders (n=235, 32%), skin color changes (n=142, 19%), muscle cramps (n=137, 19%), rash (n=105, 14%), hepatic function abnormalities (n=91, 12%), weight gain (n=86, 12%), and cytopenia (n=59, 8%). Multivariate analyses showed that TKI treatment duration <4 years was the factor associated with fatigue; Edema was more observed in female, ≥40 years old and use of 1st generation TKI; Gastrointestinal disorders in use of 1st generation TKI; Hepatic function abnormalities and rash in use of 2nd generation TKI; Weight gain in female; Muscle cramps in long-term interval from diagnosis to therapy and use of 1st generation TKI; Low blood counts in use of generic TKI. There was no impact of TKI-related side effects on daily life in 218 (30%)respondents (1 score). 375 (51%)respondents reported their daily life were slightly or moderately decreased (2 or 3 score), while 138 (19%) significantly decreased (4 or 5 score). Multivariate analyses showed that female, ≥40 years old, use of generic TKI, TKI treatment duration <4 years were factors associated with negative effect on their daily life. When taking TKI related side-effects into considderation, secondary school and below, use of generic TKI, TKI treatment duration <4 years, edema, fatigue, gastrointestinal disorders, skin color changes, rash and hepatic function abnormalities, were factors associated with negative effect on their daily life. CONCLUSION: Edema, fatigue, gastrointestinal disorders, rash, skin color changes and hepatic function abnormalities were common TKI-related side effects and influenced CML patients' daily life in China. In addition, female, older age, lower education level, use of generic TKI and shorter TKI treatment duration were associated with negative impact on quality of life.