Objective: To observe the curative efficacy of tyrosine kinase inhibitors (TKIs) in the treatment of e19a2 transcript (P230) CML chronic phase (CML-CP) patients.
Methods: The clinical data of 11 P230 CML-CP patients were collected from July 2008 to December 2019. Blood routine examination, bone marrow cytology, chromosome, and BCR-ABL qualitative and quantitative tests were performed at initial diagnosis. After TKIs treatment, BCR-ABL (P230)/ABL in peripheral blood was regularly detected to evaluate molecular response by real-time quantitative PCR.
Results: There were 11 patients (7 males and 4 females) in chronic phase from 6 domestic hospitals enrolled, their median age was 46 years old (range from 19 to 56 years old). Among 4 patients treated with imatinib (400 mg, qd) firstly, 3 cases switched to nilotinib (400 mg, bid) and 1 case switched to dasatinib (100 mg, qd) due to failure to achieve best molecular response at the landmark time or mutation of ABL kinase. Then major molecular response (MMR) was obtained within 1 year. In addition, 5 patients were treated with nilotinib (300 mg, bid) and 2 patients with dasatinib (100 mg, qd) as first-line treatment, all of them got MMR within 6 months.
Conclusion: For intolerance or resistance to imatinib, second-generation TKIs can enable P230 CML patients to achieve deeper molecular response, and MMR in a short time.
题目: 酪氨酸激酶抑制剂治疗P230慢性髓系白血病临床疗效观察.
目的: 观察酪氨酸激酶抑制剂（TKI）治疗e19a2转录本（P230）慢性髓系白血病（CML）慢性期患者的疗效。.
方法: 收集2008年7月至2019年12月11例P230 CML慢性期患者临床资料，初诊时进行血常规、骨髓细胞学、染色体和BCR-ABL定性和定量检测，接受TKI治疗后，定期通过实时定量PCR方法检测外周血BCR-ABL（P230）/ABL评估分子学反应。.
结果: 来自国内6家医院11例患者中男性7例，女性4例，中位年龄为46（19-56）岁。11例患者均处于慢性期，4例先接受伊马替尼（400 mg，qd）治疗的患者，由于在规定的时间界点未达到最佳分子学反应或出现ABL激酶区突变，其中3例改为尼洛替尼（400 mg，bid）治疗，1例改为达沙替尼（100 mg，qd）治疗，之后均在1年内获得主要分子学反应。另外5例使用尼洛替尼（300 mg，bid）、2例使用达沙替尼（100 mg，qd）作为一线治疗的患者在6个月内获得了主要分子学反应。.
结论: 对伊马替尼不耐受或疗效不佳的P230 CML患者，二代TKI可使其达到更深层次分子学反应，且能在更短时间达到主要分子学反应。.