■Elexacaftor/tezacaftor/ivacaftor(ELX/TEZ/IVA)已被证明对年龄≥2岁的囊性纤维化(CF)患者安全有效。这里,我们描述了一项观察性研究的结果,该研究评估了在现实环境中启动ELX/TEZ/IVA后疾病负担的变化.
■这家总部位于美国的,多中心,观察性研究使用来自电子病历的数据,评估年龄≥12岁的CF为F508del杂合子且存在最小功能突变(F/MF)或未表征的CFTR突变的患者在ELX/TEZ/IVA启动前后的真实世界疾病负担.终点包括1s内预测用力呼气量百分比(ppFEV1)相对于基线的绝对变化,体重指数(BMI)和BMI-年龄z-得分,糖化血红蛋白(HbA1c),和肺加重(PEx)的数量。
■总的来说,206例CF患者入组(平均[SD]年龄22.5[11.1]岁;192例F/MF基因型[93.2%])。平均随访时间为15.6(SD,1.6)个月。从基线到随访观察到ppFEV1的改善(7.3[95%CI:5.7,8.8]个百分点)。在12个月时,BMI(1.40[95%CI:1.07,1.77]kg/m2)和BMI-年龄z评分(0.14[95%CI:0.00,0.28])也从基线开始增加。任何PEx的估计年化比率在基线时为1.31,在随访期间为0.61(比率为0.47[95%CI:0.39,0.55]),基线期间需要抗生素和住院的PEx年率为0.55和0.88,随访期间为0.12和0.36(比率为0.22[95%CI:0.15,0.31]和0.41[95%CI:0.32,0.51]),分别。HbA1c的绝对变化从基线到随访为-0.22(95%CI:-0.38,-0.06)。
■ELX/TEZ/IVA治疗与肺功能改善有关,BMI增加,降低PEx的频率,和改进(即,降低)HbA1c。这些结果证实了在临床试验中看到的ELX/TEZ/IVA的广泛临床益处,并且显示了ELX/TEZ/IVA改善葡萄糖代谢标志物的潜力。
UNASSIGNED: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings.
UNASSIGNED: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for F508del and a minimal function mutation (F/MF) or an uncharacterized CFTR mutation. Endpoints included absolute change from baseline in percent predicted forced expiratory volume in 1 s (ppFEV1), body mass index (BMI) and BMI-for-age z-score, glycated hemoglobin (HbA1c), and numbers of pulmonary exacerbations (PEx).
UNASSIGNED: Overall, 206 people with CF were enrolled (mean [SD] age 22.5 [11.1] years; 192 [93.2%] with F/MF genotype). Mean follow-up was 15.6 (SD, 1.6) months. Improvements in ppFEV1 (7.3 [95% CI: 5.7, 8.8] percentage points) were observed from baseline through follow-up. Increases in BMI (1.40 [95% CI: 1.07, 1.77] kg/m2) and BMI-for-age z-score (0.14 [95% CI: 0.00, 0.28]) were also observed from baseline at 12 months. The estimated annualized rate of any PEx was 1.31 at baseline and 0.61 over follow-up (rate ratio 0.47 [95% CI: 0.39, 0.55]), with annualized rates of PEx requiring antibiotics and hospitalizations of 0.55 and 0.88 in the baseline period and 0.12 and 0.36 over follow-up (rate ratios 0.22 [95% CI: 0.15, 0.31] and 0.41 [95% CI: 0.32, 0.51]), respectively. Absolute change in HbA1c was -0.22 (95% CI: -0.38, -0.06) from baseline through follow-up.
UNASSIGNED: ELX/TEZ/IVA treatment was associated with improved lung function, increased BMI, reduced frequency of PEx, and improved (i.e., reduced) HbA1c. These results confirm the broad clinical benefits of ELX/TEZ/IVA seen in clinical trials and show the potential for ELX/TEZ/IVA to improve markers of glucose metabolism.