PDF(Pubmed)
Abstract：
UNASSIGNED: The CFTR-modulating therapy Elexaftor - Tezacaftor - Ivacaftor (ETI) has been widely prescribed since its approval in 2020 in the European Union. The aim of this study was to methodically evaluate the effects of an ETI treatment on clinical, biochemical data and Pseudomonas colonization in order to demonstrate its efficacy.
UNASSIGNED: This prospective monocentric study comprised 69 patients diagnosed with cystic fibrosis aged at least 12 years and treated with ETI between September 2020 and November 2021. Clinical and laboratory data of each patient and study visit were collected before and after 24 weeks of ETI treatment. Follow-up status of Pseudomonas aeruginosa (PsA) colonization was assessed after one year of therapy by regularly determined sputum or throat swab samples.
UNASSIGNED: Marked improvements biochemical markers of systemic inflammation as white blood cell count, levels of immunoglobulins A, G and M and albumin within 24 weeks of therapy were observed. ETI treatment proved to be effective as seen by amelioration of lung function and sweat chloride concentration. Assessment of PsA colonization status revealed a conversion from a positive to negative detection in 36% of the cases after one year of therapy.
UNASSIGNED: ETI treatment effectively improves systemic inflammation parameters and shows promising results in PsA status conversion.
UNASSIGNED: This prospective monocentric study comprised 69 patients diagnosed with cystic fibrosis aged at least 12 years and treated with ETI between September 2020 and November 2021. Clinical and laboratory data of each patient and study visit were collected before and after 24 weeks of ETI treatment. Follow-up status of Pseudomonas aeruginosa (PsA) colonization was assessed after one year of therapy by regularly determined sputum or throat swab samples.
UNASSIGNED: Marked improvements biochemical markers of systemic inflammation as white blood cell count, levels of immunoglobulins A, G and M and albumin within 24 weeks of therapy were observed. ETI treatment proved to be effective as seen by amelioration of lung function and sweat chloride concentration. Assessment of PsA colonization status revealed a conversion from a positive to negative detection in 36% of the cases after one year of therapy.
UNASSIGNED: ETI treatment effectively improves systemic inflammation parameters and shows promising results in PsA status conversion.
摘要：
■自2020年在欧盟获得批准以来，CFTR调节疗法Elexaftor-Tezacaftor-Ivacaftor（ETI）已被广泛使用。这项研究的目的是有条不紊地评估ETI治疗对临床的影响，生化数据和假单胞菌定植以证明其功效。
■这项前瞻性单中心研究包括69名诊断为囊性纤维化的患者，年龄至少为12岁，在2020年9月至2021年11月期间接受ETI治疗。在ETI治疗24周之前和之后收集每个患者的临床和实验室数据以及研究访视。治疗一年后，通过定期测定痰液或咽拭子样本评估铜绿假单胞菌（PsA）定植的随访状态。
■标记改善全身炎症的生化标志物，如白细胞计数，免疫球蛋白A的水平，观察治疗24周内的G和M以及白蛋白。通过改善肺功能和汗液氯化物浓度，证明ETI治疗有效。对PsA定植状态的评估显示，治疗一年后，有36％的病例从阳性检测转变为阴性检测。
■ETI治疗可有效改善全身炎症参数，并在PsA状态转换方面显示出有希望的结果。
■这项前瞻性单中心研究包括69名诊断为囊性纤维化的患者，年龄至少为12岁，在2020年9月至2021年11月期间接受ETI治疗。在ETI治疗24周之前和之后收集每个患者的临床和实验室数据以及研究访视。治疗一年后，通过定期测定痰液或咽拭子样本评估铜绿假单胞菌（PsA）定植的随访状态。
■标记改善全身炎症的生化标志物，如白细胞计数，免疫球蛋白A的水平，观察治疗24周内的G和M以及白蛋白。通过改善肺功能和汗液氯化物浓度，证明ETI治疗有效。对PsA定植状态的评估显示，治疗一年后，有36％的病例从阳性检测转变为阴性检测。
■ETI治疗可有效改善全身炎症参数，并在PsA状态转换方面显示出有希望的结果。
