中国慢性髓性白血病患者酪氨酸激酶抑制剂治疗现状的调研：从患者的角度

OBJECTIVE: To assess tyrosine kinase inhibitor (TKI) treatment status in patients with chronic myeloid leukemia (CML) in China and analyze the response-associated factors. METHODS: From May to November in 2014, anonymous questionnaires were distributed to adult CML patients who were receiving TKI treatment all over China. RESULTS: 1 038 questionnaires were collected, 949 questionnaires were evaluable. Of the 949 evaluable respondents, 549 (58%) were male with the median age of 41 years (range, 18 to 88 years). 623 (66%) respondents lived in an urban area and 449 (47%) had an education level ≥ a bachelor degree. 888 (94%) respondents were in the chronic phase at diagnosis, and 690 (78%) of them started TKI treatment within one year after diagnosis. 794 (84%) respondents were on imatinib, 768 (81%) on the branded. With a median TKI treatment duration of 3 years (range, <1 to 13 years), 708 of 834 (85%) evaluable respondents achieved Ph-negative (i.e. complete cytogenetic response, CCyR), and 497 of 859 (46%) BCR-ABL negative (i.e. complete molecular response, CMR). Multivariate analyses showed that female (OR=1.8, 95% CI 1.1–2.8,P=0.019 andOR=1.5, 95% CI 1.1–2.0,P=0.015), TKI treatment duration >3 years (OR=4.1, 95% CI 2.6–6.5,P<0.001 andOR=3.7, 95% CI 2.7–5.1,P< 0.001) and imatinib taken (OR=2.1, 95% CI 1.2–3.7,P=0.007 andOR=3.3, 95% CI 2.1–5.1,P<0.001) were factors affecting achieving both CCyR and CMR. In addition, higher education level (OR=2.0, 95% CI 1.3–3.1,P=0.003), starting TKI treatment <1 year (OR=2.4, 95% CI 1.5–3.8,P<0.001) and branded drugs received (OR=2.4, 95% CI 1.4–4.0,P=0.001) were factors affecting achieving a CCyR. In 884 respondents, 534 (62%) reported “heavy financial burden” as the biggest treatment impediment, only 152 (17%) reported “poor quality of life related to adverse effects of TKI”. CONCLUSION: The survey showed that majority of the Chinese CML patients received imatinib as a TKI therapy, and most of the patients achieved satisfied responses by TKI. Financial burden became the major obstacle during TKI treatment.