Wenzel Katrin, Schulze-Rothe Sarah, Haberland Annekathrin, Müller Johannes, Wallukat Gerd, Davideit Hanna
Berlin Cures GmbH, Berlin, Germany.
Heliyon. 2017 Jul 31;3(7):e00362. doi: 10.1016/j.heliyon.2017.e00362. eCollection 2017 Jul.
Autoantibodies specific for the adrenergic beta1-receptor were identified to be an essential factor for the pathogenesis of dilated cardiomyopathy. For the detection of these autoantibodies, a bioassay was developed and has been used, measuring the positive chronotropic effect on spontaneously beating neonatal rat cardiomyocytes. In order to use this bioassay as an analytical tool to monitor the effectiveness of autoantibody neutralizing therapy in a regulated field, there is a need to assess its analytical performance and validate it according to current guidelines.
Using standard autoantibody samples, the increased beat rate compared to the basal rate [delta beats/min] was recorded when investigating guideline required assay performance parameters.
The analytical specificity and sensitivity of the bioassay was demonstrated. The limit of detection and positivity cut-off level were determined to be 3.56 and 7.97 delta beats/min, respectively. The coefficient of variation (CV) of all tested single values (four technical replicates each) was ≤15.2%. The CV of precision within each measuring series did not exceed 20%. Furthermore, the sample stability under a variety of different storage conditions was assessed, as well as the robustness of the cardiomyocyte preparations, which were both given.
This bioassay fulfilled guideline determined quality requirements and proved to be appropriate for its application in clinical trials.
已确定肾上腺素能β1受体特异性自身抗体是扩张型心肌病发病机制的一个关键因素。为检测这些自身抗体,开发并使用了一种生物测定法,该方法通过测量对自发搏动的新生大鼠心肌细胞的正性变时作用来进行检测。为了将这种生物测定法用作一种分析工具,以便在受监管领域监测自身抗体中和疗法的有效性,有必要评估其分析性能并根据现行指南进行验证。
在研究指南要求的测定性能参数时,使用标准自身抗体样本记录与基础心率相比增加的心率[每分钟心跳次数变化量]。
证明了该生物测定法的分析特异性和灵敏度。检测限和阳性截断水平分别确定为3.56和7.97每分钟心跳次数变化量。所有测试的单个值(每个进行四次技术重复)的变异系数(CV)≤15.2%。每个测量系列内精密度的CV不超过20%。此外,还评估了在各种不同储存条件下样本的稳定性以及心肌细胞制剂的稳健性。
这种生物测定法符合指南确定的质量要求,并证明适用于临床试验。