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 (n= 45), while those treated after May 2016, after ruxolitinib inclusion, constituted the active group (n= 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.
本研究旨在分析接受芦可替尼治疗的骨髓纤维化患者的治疗效果及生存情况，并与标准治疗组进行对比。这是一项横断面、回顾性、非干预性的真实世界研究，研究时间跨度为2000年1月至2023年2月。2000年至2016年间（芦可替尼上市前）接受治疗的患者构成对照组（n=45），而2016年5月后（芦可替尼纳入治疗方案后）接受治疗的患者构成活性药物组（n=66）。研究通过Kaplan-Meier生存分析探讨人口学特征、临床指标、疾病严重程度及生存情况，并采用Spearman相关性分析、线性回归及其他统计方法进行分析。Kaplan-Meier分析显示，样本总体死亡风险降低75.33%。在总体人群层面，芦可替尼治疗组的死亡风险较对照组降低幅度在7.9%至77.18%之间。血液学参数（白细胞、血红蛋白、血小板）及脾脏体积均呈现下降趋势：芦可替尼治疗组患者脾脏体积在治疗前六个月缩小6%，随后六个月再缩小9%。本研究表明，在真实临床环境中，接受芦可替尼治疗的患者较该药物进入国内市场前接受标准治疗的患者，其疾病临床症状改善更显著，症状严重程度更低，生存期更长。这些改善趋势与随机临床试验报告的结果具有一致性。
肿瘤(癌症)患者之家