Objective: To investigate the current status of treatment choice and responses in patients with chronic myeloid leukemia (CML) in China. Methods: From the end of April to mid-May in 2020, a cross-sectional survey, by filling out a survey questionnaire, was conducted to explore the first-line choice of tyrosine kinase inhibitors (TKI) , current medications, drug switch and major molecular responses (MMR) as well as the variables associated with them in patients in China. Results: Data of 2933 respondents with CML from 31 provinces, municipalities, and autonomous regions across the country were included in this study. 1683 respondents (57.4%) were males. Median age was 38 (16-87) years old. 2481 respondents (84.6%) received imatinib as first-line TKI; 1803 (61.5%) , the original new drug (branded drug) . When completing the questionnaire, 1765 respondents (60.2%) were receiving imatinib; 1791 (61.1%) , branded drug. 1185 respondents (40.4%) had experienced TKI switch. With a median follow-up of 45 (3-227) months, 1417 of 1944 (72.9%) respondents with newly diagnosed CML in the chronic phase achieved MMR. Multivariate analysis showed that the respondents with urban household registration (OR=0.6, 95%CI 0.5-0.8, P<0.001) , ≥ bachelor degree (OR=0.5, 95%CI 0.4-0.7, P<0.001) , and in the advanced phase at diagnosis (OR=0.5, 95%CI 0.3-0.8, P=0.001) less preferred Chinese generic TKI, while the respondents from the central region in China more preferred Chinese generic TKI more than those from the eastern region (OR=1.7, 95%CI 1.4-2.0, P<0.001) . Moreover, the respondents in the advanced phase at diagnosis more preferred second-generation TKI (OR=5.4, 95%CI 3.6-8.2, P<0.001) ; those ≥60 years old, less preferred second-generation TKI (OR=0.4, 95%CI 0.2-0.7, P=0.002) . Being in the advanced phase at diagnosis (OR=2.2, 95%CI 1.6-3.2, P<0.001) , first-line choice of imatinib (OR=2.0, 95%CI 1.6-2.6, P<0.001) or Chinese generic drugs (OR=1.3, 95%CI 1.1-1.6, P=0.002) , longer interval from diagnose of CML to starting TKI treatment (OR=1.2, 95%CI 1.1-1.2, P<0.001) and longer duration of TKI therapy (OR=1.1, 95%CI 1.0-1.1, P<0.001) were significantly associated with TKI switch; urban household registration (OR=0.7, 95%CI 0.6-0.8, P<0.001) , ≥MMR (OR=0.6, 95%CI 0.5-0.8, P<0.001) and unknown response (OR=0.7, 95%CI 0.6-0.9, P=0.003) , no TKI switch. Female sex (OR=1.4, 95%CI 1.1-1.7, P=0.003) , urban household registration (OR=1.6, 95%CI 1.3-2.0, P<0.001) , front-line imatinib therapy (OR=1.4, 95%CI 1.1-1.9, P=0.016) and longer duration of TKI treatment (OR=1.2, 95%CI 1.2-1.3, P<0.001) were significantly associated with achieving a MMR or better response; age ≥ 60 years old (OR=0.7, 95%CI 0.4-1.0, P=0.047) and TKI switch (OR=0.6, 95%CI 0.5-0.7, P<0.001) , achieving no MMR. Conclusions: By 2020, the majority of Chinese CML patients received imatinib as the fist-line TKI therapy and continue to take it. More than half of TKIs were branded drugs. Socio-demographic characteristics and clinical variables affect their TKI choice, drug switch, and treatment response.
目的： 调查中国慢性髓性白血病（CML）患者的治疗现状。 方法： 横断面研究，2020年4月末至5月中旬，以填写调研问卷的形式在全国范围内调研CML患者，分析酪氨酸激酶抑制剂（TKI）一线选择、目前用药、药物转换和获得主要分子学反应（MMR）的比例及其影响因素。 结果： 2933份来自全国31个省市自治区CML受访者的问卷可供分析，男性1683例（57.4%），中位年龄38（16~87）岁。一线选择：伊马替尼2481例（84.6%），原创性新药（原研药）1803例（61.5%）。填写问卷时用药：伊马替尼1765例（60.2%），原研药1791例（61.1%）。共1185例（40.4%）受访者曾经历TKI药物转换。1944例初发慢性期受访者TKI中位治疗45（3~227）个月，1417例（72.9%）获得≥MMR的疗效。多因素分析显示，城镇户籍（OR=0.6，95%CI 0.5~0.8，P<0.001）、≥大学学历（OR=0.5，95%CI 0.4~0.7，P<0.001）和进展期（OR=0.5，95%CI 0.3~0.8，P=0.001）受访者更少首选仿制TKI，而来自中部地区受访者比东部地区更多首选国产仿制TKI（OR=1.7，95%CI 1.4~2.0，P<0.001）。进展期受访者更多首选二代TKI（OR=5.4，95%CI 3.6~8.2，P<0.001），≥60岁受访者更少首选二代TKI（OR=0.4，95%CI 0.2~0.7，P=0.002）。诊断时处于进展期（OR=2.2，95%CI 1.6~3.2，P<0.001）、首选伊马替尼（OR=2.0，95%CI 1.6~2.6，P<0.001）、首选国产仿制药（OR=1.3，95%CI 1.1~1.6，P=0.002）、诊断距开始TKI治疗的时间更长（OR=1.2，95%CI 1.1~1.2，P<0.001）和服用TKI的时间更长（OR=1.1，95%CI 1.0~1.1，P<0.001）与药物转换比例增高显著相关。城镇户籍（OR=0.7，95%CI 0.6~0.8，P<0.001）、获≥MMR（OR=0.6，95%CI 0.5~0.8，P<0.001）和疗效未知（OR=0.7，95%CI 0.6~0.9，P=0.003）与药物转换比例低显著相关。女性（OR=1.4，95%CI 1.1~1.7，P=0.003）、城镇户籍（OR=1.6，95%CI 1.3~2.0，P<0.001）、初始服用伊马替尼（OR=1.4，95%CI 1.1~1.9，P=0.016）和TKI治疗时间更长（OR=1.2，95%CI 1.2~1.3，P<0.001）与获得≥MMR显著相关，而年龄≥60岁（OR=0.7，95%CI 0.4~1.0，P=0.047）和药物转换（OR=0.6，95%CI 0.5~0.7，P<0.001）与未获得MMR显著相关。 结论： 截至2020年，中国CML患者中大多数首选并持续服用伊马替尼，半数以上服用原研药。社会人口学特征和疾病分期影响了患者的TKI选择、药物转换和治疗反应。.
Keywords: Drug switch; Leukemia, myeloid, chronic; Questionnaires; Socio-demographic characteristics; Treatment response; Tyrosine kinase inhibitor.