Study on the influencing factors of venetoclax trough concentration and its association with efficacy in patients with acute myeloid leukemia

HE Weiwei; LIU Zhirui; QIN Shiwei; GONG Qiang; CHENG Lin

doi:10.6039/j.issn.1001-0408.2026.09.16

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Study on the influencing factors of venetoclax trough concentration and its association with efficacy in patients with acute myeloid leukemia

更新时间：2026-05-14

- Study on the influencing factors of venetoclax trough concentration and its association with efficacy in patients with acute myeloid leukemia
- ZHONGGUO YAOFANG Vol. 37, Issue 9, Pages: 1200-1205(2026)
- 作者机构：
  
  1.陆军军医大学第一附属医院药剂科，重庆　400038
  2.陆军军医大学第一附属医院感染病科，重庆　400038
  3.陆军军医大学第一附属医院血液科，重庆　400038
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2026.09.16
  CLC： R733;R969
- Received：14 January 2026，
  
  Revised：2026-03-17，
  
  Accepted：17 April 2026，
  
  Published：15 May 2026
- 稿件说明：
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何韦韦,刘职瑞,秦士玮,等.急性髓系白血病患者维奈托克血浆谷浓度的影响因素及其与疗效的关系研究 [J].中国药房,2026,37(09):1200-1205.

HE Weiwei,LIU Zhirui,QIN Shiwei,et al.Study on the influencing factors of venetoclax trough concentration and its association with efficacy in patients with acute myeloid leukemia[J].ZHONGGUO YAOFANG,2026,37(09):1200-1205.
何韦韦,刘职瑞,秦士玮,等.急性髓系白血病患者维奈托克血浆谷浓度的影响因素及其与疗效的关系研究 [J].中国药房,2026,37(09):1200-1205. DOI： 10.6039/j.issn.1001-0408.2026.09.16.

HE Weiwei,LIU Zhirui,QIN Shiwei,et al.Study on the influencing factors of venetoclax trough concentration and its association with efficacy in patients with acute myeloid leukemia[J].ZHONGGUO YAOFANG,2026,37(09):1200-1205. DOI： 10.6039/j.issn.1001-0408.2026.09.16.

摘要

目的

探讨急性髓系白血病（AML）患者维奈托克血浆谷浓度、疗效及其影响因素之间的关系。

方法

AML患者服用维奈托克5 d后，于下次给药前采集静脉血，采用高效液相色谱-串联质谱法测定维奈托克血浆谷浓度。采用Spearman相关性分析评估维奈托克血浆谷浓度与各指标（包括患者一般资料、维奈托克相关指标、肝功能指标、肾功能指标、血常规指标等）之间的相关性，采用多元线性回归分析维奈托克血浆谷浓度的独立影响因素；以疗效为因变量[完全缓解（CR）+部分缓解（PR）vs.未缓解（NR）]，采用单因素和多因素二元Logistic回归分析影响疗效的因素。采用受试者工作特征（ROC）曲线评估维奈托克血浆谷浓度对临床疗效（以CR计）的预测价值。

结果

本研究共纳入101例患者的172个维奈托克血浆谷浓度检测值。维奈托克中位血浆谷浓度为2.38（1.18，3.85）μg/mL；维奈托克血浆谷浓度中位检测时间为10（7，15）d；维奈托克使用天数为（34±12）d。多元线性回归分析结果显示，碱性磷酸酶（

＝14.65，95%CI为5.35～23.95，

＝0.002）、总胆红素（

＝－101.71，95%CI为－197.16～－6.25，

＝0.037）和白细胞计数（

＝－106.84，95%CI为－187.61～－26.07，

＝0.010）是维奈托克血浆谷浓度的独立影响因素。因治疗过程中有患者脱落，共纳入69例患者的114个维奈托克血浆谷浓度进行疗效评估，结果显示，CR、PR、NR患者分别有46例（66.7%）、11例（15.9%）和12例（17.4%）。多因素二元Logistic回归分析结果显示，年龄、血红蛋白、维奈托克血浆谷浓度、红细胞压积、平均血红蛋白含量是患者疗效的独立影响因素（

＜0.05）；ROC曲线分析结果显示，预测患者疗效（以CR计）的维奈托克血浆谷浓度截断值为1.68 μg/mL（AUC＝0.66，95%CI为0.54～0.78，

＝0.014）。

结论

维奈托克血浆谷浓度在AML患者中存在较大的个体差异，与碱性磷酸酶、总胆红素、白细胞计数存在显著相关性；维奈托克血浆谷浓度是患者疗效的独立影响因素，血浆谷浓度在1.68 μg/mL以上时可能获得更好的治疗效果。

Abstract

OBJECTIVE

To investigate the effect of plasma trough concentration of venetoclax and its influencing factors in patients with acute myeloid leukemia （AML）.

METHODS

After 5 days of venetoclax administration， venous blood samples were collected from AML patients before the next dose. Plasma trough concentrations of venetoclax were determined using high-performance liquid chromatography-tandem mass spectrometry. Spearman correlation was used to assess the correlations between venetoclax plasma trough concentration and various parameters （including patients’ general information， venetoclax-related indicators， liver function indicators， kidney function indicators， and blood routine indicators）. Multiple linear regression analysis was performed to identify independent factors influencing plasma trough concentration of venetoclax. Using efficacy as dependent variable [complete remission （CR）+partial remission （PR） vs. no remission （NR）]， univariate and multivariate binary Logistic regression analyses were conducted to identify factors affecting efficacy. The receiver operating characteristic （ROC） curve was used to evaluate the predictive value of venetoclax plasma trough concentration for clinical efficacy （assessed as CR）.

RESULTS

A total of 172 venetoclax plasma trough concentration measurements from 101 patients were included in this study. The median plasma trough concentration of venetoclax was 2.38 （1.18， 3.85） μg/mL； the median sampling time for plasma trough concentration of venetoclax was 10 （7， 15） d； the duration of venetoclax use was （34±12） d. Multiple linear regression analysis showed that alkaline phosphatase （

＝14.65， 95%CI： 5.35-23.95，

＝0.002）， total bilirubin （

＝－101.71， 95%CI：－197.16 to －6.25，

＝0.037）， and white blood cell count （

＝－106.84， 95%CI：－187.61 to －26.07，

＝0.010） were independent factors influencing plasma trough concentration of venetoclax. Due to patient attrition during treatment， 114 venetoclax plasma trough concentration measurements from 69 patients were included for efficacy evaluation. The results showed that 46 patients （66.7%） achieved CR， 11 patients （15.9%） achieved PR， and 12 patients （17.4%） were NR. Multivariate binary Logistic regression analysis showed that age， hemoglobin， venetoclax plasma trough concentration， hematocrit， and mean corpuscular hemoglobin were independent factors affecting patient efficacy （

＜0.05）. ROC curve analysis showed that the cut-off value of plasma trough concentration of venetoclax for predicting patient efficacy （assessed as CR） was 1.68 μg/mL （AUC＝0.66， 95%CI： 0.54-0.78，

＝0.014）.

CONCLUSIONS

There is considerable inter-individual variability in plasma trough concentration of venetoclax among AML patients. Plasma trough concentration of venetoclax is significantly correlated with alkaline phosphatase， total bilirubin， and white blood cell count. Plasma trough concentration of venetoclax is an independent factor affecting patient’s efficacy， and when the cut-off value for predicting CR is above 1.68 μg/mL， better effects may be achieved.

关键词

Keywords

references

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