Kantonsspital Aarau, Aarau, Switzerland.
Novartis Pharma Schweiz, Rotkreuz, Switzerland.
BMC Cancer. 2022 Nov 19;22(1):1192. doi: 10.1186/s12885-022-10241-y.
The real-world experience of Swiss chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) is largely unknown, in particular with regard to achievement of response per European Leukemia Net (ELN) criteria and adherence to ELN recommendations.
This was a retrospective, non-interventional, multicenter chart review of patients with newly diagnosed CML who had received first-line TKI and were solely treated with TKIs between 2010 and 2015, with a minimum follow-up of 18 months, at six Swiss hospitals. Effectiveness was evaluated according to ELN 2013 milestone achievements at 3, 6, 12 and 18 months, and at last follow-up.
Data from 63 patients (56% men; median age at diagnosis 55 years) were collected (first-line imatinib [n = 27], nilotinib [n = 27], dasatinib [n = 8], or ponatinib [n = 1]). TKI switches (49 times) and dosing changes (165 times) due to intolerance or insufficient response were frequent. Compared with patients receiving first-line imatinib, a higher proportion of patients receiving first-line nilotinib or dasatinib achieved optimal response at all timepoints, irrespective of subsequent TKI therapy, and a higher proportion of patients treated with first-line nilotinib and dasatinib reached deep molecular response (BCR-ABL1 ≤ 0.01%) at 18 months (42 and 38%, respectively, versus 27%). Patients who received nilotinib or dasatinib switched therapies less frequently than patients treated with imatinib, irrespective of subsequent TKI therapy.
Although patient numbers were small, this real-world evidence study with patients with CML confirms that ELN guidelines are generally implemented in Swiss clinical practice, with a large proportion of patients achieving ELN 2013 milestones. While TKI use involved all inhibitors approved at the time of the study, an unexpectedly high number of TKI therapy switches suggests a clear difference in TKI use between registration trials and clinical practice.
瑞士慢性髓性白血病（CML）患者接受酪氨酸激酶抑制剂（TKI）治疗的真实世界经验在很大程度上尚不清楚，特别是在达到欧洲白血病网络（ELN）标准的反应和遵循 ELN 建议方面。
这是一项回顾性、非干预性、多中心的图表审查，评估了 2010 年至 2015 年间接受一线 TKI 治疗且仅接受 TKI 治疗的新诊断 CML 患者的疗效，在六家瑞士医院的最低随访时间为 18 个月。根据 ELN 2013 里程碑成就，在 3、6、12 和 18 个月及最后一次随访时评估疗效。
共收集了 63 例患者（56%为男性；中位诊断年龄为 55 岁）的数据（一线伊马替尼 [n=27]、尼洛替尼 [n=27]、达沙替尼 [n=8]或泊那替尼 [n=1]）。由于不耐受或反应不足，频繁进行 TKI 转换（49 次）和剂量调整（165 次）。与接受一线伊马替尼的患者相比，接受一线尼洛替尼或达沙替尼治疗的患者在所有时间点均达到了最佳反应，而不论后续 TKI 治疗如何，且接受一线尼洛替尼和达沙替尼治疗的患者在 18 个月时达到深度分子反应（BCR-ABL1≤0.01%）的比例更高（分别为 42%和 38%，而伊马替尼组为 27%）。无论后续 TKI 治疗如何，接受尼洛替尼或达沙替尼治疗的患者换用药物的频率均低于接受伊马替尼治疗的患者。
尽管患者人数较少，但这项 CML 真实世界证据研究证实，瑞士临床实践中普遍遵循 ELN 指南，大多数患者达到了 ELN 2013 里程碑。虽然 TKI 的使用涵盖了研究时已批准的所有抑制剂，但 TKI 治疗转换的数量之多表明，注册试验和临床实践之间的 TKI 使用存在明显差异。
