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.
目的: 评估中国慢性髓性白血病（CML）慢性期（CP）患者报告的酪氨酸激酶抑制剂（TKI）相关不良反应及其对日常生活的影响。
方法: 2014年5月至11月在全国范围内向正在接受TKI治疗的成年CML患者发放无记名调查问卷。TKI不良反应对患者日常生活的影响程度采用自我报告的形式，以1分（没有影响）至5分（严重影响）进行评估。
结果: 731例CML-CP受访者报告了TKI相关不良反应影响其日常生活评分，中位年龄41 (18~88）岁，男性407例（56%），560例（77%）确诊至TKI治疗时间< 1年。中位TKI治疗时间为3（<1~13）年，549例（75%）获得完全细胞遗传学反应，301例（41%）获得完全分子学反应。TKI最多见的不良反应依次为水肿（44%）、乏力（38%）、胃肠道不适（32%）、面部颜色改变（19%）、肌肉痉挛（19%）、皮疹（14%）、肝功能异常（12%）、体重增加（12%）和血细胞计数降低（8%）。多因素分析显示，TKI服药时间<4年是乏力的独立影响因素；女性、年龄≥40岁、服用一代TKI者容易发生水肿；服用一代TKI容易发生胃肠道不适；服用二代TKI容易出现皮疹及肝功能异常；女性容易出现体重增加；确诊至TKI治疗时间≥1年、服用一代TKI者较易出现肌肉痉挛；服用仿制品是血细胞计数降低的独立影响因素。患者自我报告TKI相关不良反应影响日常生活的评分显示218例（30%）没有影响（1分），375例（51%）受轻中度影响（2~3分），138例（19%）受严重影响（4~5分）。多因素分析显示，仅将人口学特征纳入分析时，女性、年龄≥40岁、服用仿制品、TKI服药时间<4年与受访者较高的日常生活受影响程度显著相关；将不良反应也纳入多因素分析后，未接受高等教育、TKI服药时间<4年、服用仿制品以及以下不良反应：水肿、乏力、胃肠道不适、面部颜色改变、皮疹和肝功能异常与较高的日常生活受影响程度相关。
结论: 服用TKI的中国CML-CP患者中，水肿、乏力、胃肠道不适、皮疹、面部颜色改变和肝功能异常是最常见的、影响其日常生活的药物不良反应。女性、年龄≥40岁、未接受高等教育、服用仿制品和TKI治疗时间较短与患者日常生活受影响显著相关。