摘要
目的调查用于治疗不适合强诱导化疗的新诊断急性髓系白血病(AML)患者的靶向B细胞淋巴瘤-2(BCL-2)抑制剂维奈克拉在中国的真实世界使用剂量与疗效,以及相较低强度化疗方案在医疗资源利用上的差异。方法开展两阶段的医生调研,第一阶段招募15名医生进行深度访谈,第二阶段邀请70名医生进行线上调研。结果使用中位值和百分比汇总,并通过非参数中位数检验比较接受维奈克拉与低强度化疗方案患者人群的医疗资源利用情况。结果来自68家三甲医院的85份问卷被纳入分析。爬坡期后,患者使用维奈克拉的稳定剂量为每天400 mg(45.7%)、200 mg(27.6%)、100 mg(19.8%)、70 mg(6.9%),实现复合完全缓解前后患者每疗程用药天数不足28 d的比例分别为50.0%和60.2%。经计算,维奈克拉在爬坡后的中位稳定剂量为191 mg/d。复合完全缓解率中位值为60.0%,中位时间为2.0个月。在达到复合完全缓解的患者中,相对低强度化疗方案,接受维奈克拉联合阿扎胞苷治疗的患者人群输血和抗感染治疗比例以及输血频率降低50.0%,月均住院天数减少30.0%,差异有统计学意义(P<0.05)。结论在真实世界的应用中,维奈克拉大多会参照说明书进行剂量调整,但仍能实现较高的复合完全缓解率。此外,使用维奈克拉联合阿扎胞苷相比低强度化疗,可以明显节约输血等医疗资源利用。
Objective To investigate the dosages and efficacy of targeted BCL-2 inhibitor Vineclavone in real-world use in China for the treatment of newly diagnosed acute myeloid leukemia(AML)patients who are not suitable for strong induction chemotherapy,as well as the differences in medical resource utilization compared to lower intensity chemotherapy regimens.Methods A two-stage nationwide survey was conducted.In stage one,15 hematologists were recruited to participate in in-depth interviews using a semi-structured questionnaire.In stage two,70 hematologists were invited to finish an online survey.Descriptive statistics and non-parametric median tests were conducted to summarize and compare results.Results 85 questionnaires were collected from 68 tertiary hospitals.Based on the collected data,after the ramp-up period,patients received venetoclax in the dosage of 400 mg(45.7%),200 mg(27.6%),100 mg(19.8%),70 mg(6.9%)per day.50.0%and 60.2%of patients took venetoclax less than 28 days per cycle before and after composite complete remission.The venetoclax daily dose was calculated as 191 mg.The median composite complete remission was 60.0%and the median time to first response was 2.0 months.Among patients achieving composite complete remission,compared with low-intensity chemotherapy,the proportion of patients taking blood transfusion and anti-infection and the frequency of blood transfusion were reduced by 50.0%in those taking venetoclax+azacitidine,the days of hospitalization per month was also significantly reduced by 30.0%,the difference were statistically significant(P<0.05).Conclusion Dose modifications aligned with label of venetoclax were common in real-world practice.Despite this,a high response rate was observed.Additionally,venetoclax+azacitidine was associated with less HCRU particularly among patients achieving composite complete remission.
作者
赵馨然
莫璇
赵瑾
刘君
ZHAO Xin-Ran;MO Xuan;ZHAO Jin;LIU Jun(IQVIA Solutions Enterprise Management Consulting(Shanghai)Co.,Ltd.,Shanghai 200041,China)
出处
《中国药物经济学》
2023年第5期29-36,共8页
China Journal of Pharmaceutical Economics
关键词
急性髓系白血病
维奈克拉
真实世界
剂量
疗效
医疗资源利用
Acute myeloid leukemia
Venetoclax
Real-world study
Dosage
Efficacy
Health care resource utilization
