Abstract

Clinical trials in chronic myeloid leukemia (CML) are usually carried out in specialized centers whereas primary care for patients (pts) with CML is mainly provided by local oncology practices. The aim of this study was to assess treatment practices in pts with CML in the setting of private oncology practices in Germany. We collected data of 819 pts with a confirmed diagnosis (dx) of CML in 2013 or later from 43 practices. At dx, 84.2% (n=690) and 9.4% (n=77) of pts were in chronic or accelerated phase, 0.7% (n=6) had a blast crisis. Molecular monitoring was provided by EUTOS certified laboratories in 87.7% of pts. Typical BCR::ABL1 transcripts were detected in 86.6% (n=709). Molecular response was assessed after 2.8, 6.0, 9.4 and 12.9 m (mean) after start of treatment. Of the pts with available data, 11.1% did not achieve early molecular response and at 18 m, 83.7% had at least a major molecular response. 288 (35.2%) of pts switched to 2^nd line (2L) treatment after a mean of 21.0 months. Reasons for 2L treatment were side effects in 43.4% and suboptimal response or failure in 31.4% of pts. 106 pts went on to third line (3L) treatment. 36.8 % of pts switched to and 92.8 % of pts still on 3L treatment achieved BCR::ABL1^IS ≤1% at 12 m. In conclusion, in Germany pts with CML are routinely monitored by qPCR and good responses are achieved in the majority. Treatment changes are mainly due to adverse events rather than suboptimal responses.

中文翻译：

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聚焦德国 CML 患者的现实治疗：私人肿瘤实践的回顾性调查

摘要

慢性粒细胞白血病 (CML) 的临床试验通常在专门中心进行，而 CML 患者 (pt) 的初级护理主要由当地肿瘤诊所提供。本研究的目的是评估德国私人肿瘤诊所中 CML 患者的治疗实践。我们从 43 个诊所收集了 2013 年或以后确诊为 CML 的 819 名患者的数据。在 dx 时，84.2% ( n = 690) 和 9.4% ( n = 77) 的患者处于慢性或加速期，0.7% ( n = 6) 患有急变期。EUTOS 认证的实验室对 87.7% 的患者进行了分子监测。典型的BCR::ABL1转录本的检测率为 86.6% ( n =709)。治疗开始后 2.8、6.0、9.4 和 12.9 m（平均）后评估分子反应。在有可用数据的患者中，11.1% 没有达到早期分子反应，而在 18 m 时，83.7% 至少有主要分子反应。平均 21.0 个月后，288 名 (35.2%) 患者转为二线^{( 2L) 治疗。}2L 治疗的原因是 43.4% 的患者出现副作用，31.4% 的患者反应欠佳或失败。106 名患者继续接受三线 (3L) 治疗。36.8% 的患者转用 3L 治疗，92.8% 仍在接受 3L 治疗的患者在 12 m 处达到BCR::ABL1 ^IS ≤1%。总之，在德国，CML 患者定期通过 qPCR 进行监测，并且大多数患者都取得了良好的反应。治疗变化主要是由于不良事件而不是次优反应。