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Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis
Journal of Cystic Fibrosis ( IF 5.2 ) Pub Date : 2020-07-01 , DOI: 10.1016/j.jcf.2020.07.002
Emily DiMango 1 , Jonathan Overdevest 2 , Claire Keating 1 , Sarah Fracasso Francis 1 , David Dansky 1 , David Gudis 2
Affiliation  

BACKGROUND Elexacaftor-tezacaftor-ivacaftor is a highly effective modulator for cystic fibrosis (CF) patients homozygous or heterozygous for F508del. Effects of the drug on sinonasal symptoms have not been studied. METHODS Adult participants were prospectively evaluated at baseline and after three months of treatment using validated questionnaires assessing sinonasal symptoms (SNOT-22) and CF-related quality of life (CFQ-R). RESULTS Forty-three participants completed the study; 23 were taking other CF transmembrane conductance (CFTR) modulators at the time of study participation. There was a significant improvement in mean SNOT-22 from 34.8 (29.4-40, 95% confidence interval) to 24.4 (19.9-29.0) (p = 0.000003) and in the Respiratory domain of the CFQR from 60.6 (57.1-64.1) to 83.3 (79.4-87.2) (p = 0.0000002), both achieving a minimal clinically important difference. Patients previously taking CFTR modulators experienced a greater benefit in sinonasal and respiratory symptoms. CONCLUSIONS Elexacaftor-tezacaftor-ivacaftor is associated with significant improvement in sinonasal symptoms; previous use of CFTR modulators is associated with greater benefit.

中文翻译:

高效调节剂治疗对囊性纤维化鼻窦症状的影响

背景 Elexacaftor-tezacaftor-ivacaftor 是 F508del 纯合子或杂合子囊性纤维化 (CF) 患者的高效调节剂。尚未研究该药物对鼻窦症状的影响。方法 使用经过验证的问卷评估鼻窦症状 (SNOT-22) 和 CF 相关生活质量 (CFQ-R) 在基线和治疗三个月后对成年参与者进行前瞻性评估。结果 43 名参与者完成了研究;23 人在参与研究时正在服用其他 CF 跨膜电导 (CFTR) 调节剂。平均 SNOT-22 从 34.8(29.4-40,95% 置信区间)显着改善至 24.4(19.9-29.0)(p = 0.000003),CFQR 的呼吸域从 60.6(57.1-64.1)显着改善至83.3 (79.4-87.2) (p = 0.0000002), 两者都实现了最小的临床重要差异。先前服用 CFTR 调节剂的患者在鼻窦和呼吸道症状方面的益处更大。结论 Elexacaftor-tezacaftor-ivacaftor 与鼻窦症状的显着改善相关;以前使用 CFTR 调制器会带来更大的好处。
更新日期:2020-07-01
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