Stoeva Vera, Mihaylov Georgi, Mitov Konstantin, Petrova Guenka, Tachkov Konstantin
Specialized Hospital for Active Treatment of Hematological Diseases, 1000 Sofia, Bulgaria.
Faculty of Pharmacy, Medical University of Sofia, 1000 Sofia, Bulgaria.
Cancers (Basel). 2023 Oct 20;15(20):5085. doi: 10.3390/cancers15205085.
The aim of this study was to analyze the therapeutic results and survival of patients with myelofibrosis treated with ruxolitinib in comparison with a group on standard therapy. It is a cross-sectional, retrospective, non-interventional, real-life study that was performed between January 2000 and February 2023. Patients treated between 2000 and 2016, before the introduction of ruxolitinib, constituted the control group ( = 45), while those treated after May 2016, after ruxolitinib inclusion, constituted the active group ( = 66). Demographic characteristics, clinical indicators, the severity of the disease, and survival were explored using Kaplan-Meier survival analyses. Spearman's correlation, linear regression, and other statistical analyses were performed. According to the Kaplan-Meier analysis, there was a 75.33% reduction in the fatality risk in the sample. On a general-population level, the fatality risk in the group treated with ruxolitinib varied between 7.9% and 77.18% compared to that of the risk in the control group. There was a decrease in blood parameters (leukocytes, hemoglobin, and platelets) and spleen size. During the first six months, the spleen size of the patients on ruxolitinib decreased by 6%, and during the second six months, it decreased by another 9%. This study shows that patients in a real-life clinical setting treated with ruxolitinib exhibited improved clinical signs of the disease, had a lower symptom severity, and survived longer than patients on standard therapy before ruxolitinib's entrance into the national market. The improvements correlate with those reported in randomized clinical trials.
本研究的目的是分析与接受标准治疗的一组患者相比，接受鲁索替尼治疗的骨髓纤维化患者的治疗效果和生存率。这是一项横断面、回顾性、非干预性的真实世界研究，于2000年1月至2023年2月进行。2000年至2016年鲁索替尼引入之前接受治疗的患者构成对照组（n = 45），而2016年5月之后鲁索替尼纳入后接受治疗的患者构成治疗组（n = 66）。使用Kaplan-Meier生存分析探讨人口统计学特征、临床指标、疾病严重程度和生存率。进行了Spearman相关性分析、线性回归分析和其他统计分析。根据Kaplan-Meier分析，样本中的死亡风险降低了75.33%。在一般人群水平上，与对照组相比，接受鲁索替尼治疗组的死亡风险在7.9%至77.18%之间变化。血液参数（白细胞、血红蛋白和血小板）和脾脏大小有所下降。在最初的六个月中，接受鲁索替尼治疗的患者脾脏大小下降了6%，在接下来的六个月中，又下降了9%。本研究表明，在现实临床环境中接受鲁索替尼治疗的患者，其疾病的临床体征有所改善，症状严重程度较低，并且比鲁索替尼进入国内市场之前接受标准治疗的患者存活时间更长。这些改善与随机临床试验中报告的结果相关。
