Mignani Renzo, Pieroni Maurizio, Pisani Antonio, Spada Marco, Battaglia Yuri, Verrecchia Elena, Mangeri Mario, Feriozzi Sandro, Tanini Ilaria, De Danieli Gianluca, Pieruzzi Federico
Nephrology and Dialysis Department, Infermi Hospital, Italy.
Cardiovascular Department, San Donato Hospital, Italy.
Clin Kidney J. 2018 Nov 14;12(1):65-70. doi: 10.1093/ckj/sfy108. eCollection 2019 Feb.
The FAbry STabilization indEX (FASTEX) is an innovative index allowing the assessment of clinical stability over time in Fabry disease patients. This index was developed in a population of 28 male patients with the classical form of Fabry disease.
The aim of the study was to test the accuracy of the FASTEX in evaluating Fabry disease stability in 132 male and female patients with classical and non-classical Fabry disease from nine Italian centres and it also aimed to define the sensitivity and specificity of this new tool. In particular, we aimed to investigate the correlation between the FASTEX and clinical judgement in a large-scale cohort of the study population.
Statistical methods applied to this investigation included the calculation of accuracy, specificity and sensitivity, receiver operating characteristic (ROC) curves and Cohen's κ index related to the FASTEX and clinical judgement.
The patient population included 58 males (43.9%). The mean age of the overall population was 46.3 ± 15. 1 years (range 31.2-61.4). The median interval between the two multidisciplinary evaluations used for FASTEX calculation was 398 days. Since no gold standard method is available to define the overall clinical condition of Fabry patients over time, the results of the FASTEX were compared with clinical judgements given by the physicians involved in this study. In this way, the FASTEX classified 121 of 132 (92%) patients correctly. In particular, the FASTEX correctly identified 93% (41/44) of clinically 'unstable' and 91% (80/88) of clinically 'stable' patients. The area under the curve of the ROC related to the FASTEX index cut-off (20) was equal to 0.967, very close to its theoretical maximum (1), which means that it is an excellent test for classifying patients as 'stable' or 'unstable' compared with clinical judgement. In addition, the FASTEX cut-off >20 provides the most acceptable balance between sensitivity and specificity. The Cohen's κ index value obtained in our study was 0.82, showing a highly statistically significant P-value < 0.01 related to the agreement between the FASTEX and clinical judgement.
The FASTEX is demonstrated here to be a specific and sensitive tool. When applied to a large cohort of Fabry patients, it was shown to be a valid instrument in helping physicians to discriminate objectively the clinical stability of individual Fabry patients.
法布里病稳定性指数（FASTEX）是一项创新指数，可用于评估法布里病患者随时间推移的临床稳定性。该指数是在28例患有经典型法布里病的男性患者群体中开发出来的。
本研究旨在检测FASTEX在评估来自意大利九个中心的132例患有经典型和非经典型法布里病的男性和女性患者的疾病稳定性方面的准确性，同时还旨在确定这一新型工具的敏感性和特异性。特别是，我们旨在研究在大规模研究人群队列中FASTEX与临床判断之间的相关性。
应用于本调查的统计方法包括计算FASTEX和临床判断的准确性、特异性和敏感性、受试者工作特征（ROC）曲线以及科恩κ指数。
患者群体包括58名男性（43.9%）。总体人群的平均年龄为46.3±15.1岁（范围31.2 - 61.4岁）。用于计算FASTEX的两次多学科评估之间的中位间隔时间为398天。由于没有金标准方法可用于确定法布里病患者随时间推移的整体临床状况，因此将FASTEX的结果与参与本研究的医生给出的临床判断进行了比较。通过这种方式，FASTEX正确分类了132例患者中的121例（92%）。特别是，FASTEX正确识别出93%（41/44）临床“不稳定”患者和91%（80/88）临床“稳定”患者。与FASTEX指数临界值（20）相关的ROC曲线下面积等于0.967，非常接近其理论最大值（1），这意味着与临床判断相比，它是将患者分类为“稳定”或“不稳定”的一项出色测试。此外，FASTEX临界值>20在敏感性和特异性之间提供了最可接受的平衡。我们研究中获得的科恩κ指数值为0.82，显示出与FASTEX和临床判断之间的一致性相关且具有高度统计学意义的P值<0.01。
在此证明FASTEX是一种特异且敏感的工具。当应用于一大队列法布里病患者时，它被证明是帮助医生客观区分个体法布里病患者临床稳定性的有效工具。
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