METHODS: This was a single-center, prospective cohort study enrolling adults with CF at a major center in Italy. We used linear regression models to identify a set of potential predictors (including CFTR genotype, sex, age, and baseline clinical characteristics) and estimate the variability in treatment response.
RESULTS: The study included 211 patients (median age: 29 years, range: 12-58). Median changes (10-90th percentile) from baseline were: - 56 mEq/L (-76; -27) for sweat chloride concentration, +14.5 points (2.5; 32.0) for ppFEV1, +0.33 standard deviation scores (-0.13; 1.05) for BMI and +17 points (0; 39) for the CFQ-R respiratory domain score. The selected predictors explained 23 % of the variability in sweat chloride concentration changes, 18 % of the variability in ppFEV1 changes, 39 % of the variability in BMI changes, and 65 % of the variability in CFQ-R changes.
CONCLUSIONS: This study highlights a high level of heterogeneity in treatment response to ETI, which can only be partially explained by the baseline characteristics of the disease.
方法:这是一个单中心,前瞻性队列研究在意大利的一个主要中心招募患有CF的成年人。我们使用线性回归模型来确定一组潜在的预测因子(包括CFTR基因型,性别,年龄,和基线临床特征)并估计治疗反应的变异性。
结果:该研究包括211名患者(中位年龄:29岁,范围:12-58)。与基线相比的中位数变化(10-90百分位数)为:-56mEq/L(-76;-27),ppFEV1+14.5分(2.5;32.0),BMI+0.33标准差评分(-0.13;1.05),CFQ-R呼吸域评分+17分(0;39)。选定的预测因子解释了23%的汗液氯化物浓度变化的变异性,PPFEV1变化的变异性为18%,39%的变异性在BMI的变化,CFQ-R变化的变异性为65%。
结论:这项研究强调了ETI治疗反应的高度异质性,这只能部分由疾病的基线特征来解释。