REAL-world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations-A case report

被引：2

作者：

Fuchs, Teresa ^{[1
]}

Appelt, Dorothea ^{[1
]}

Niedermayr, Katharina ^{[1
]}

Ellemunter, Helmut ^{[1
]}

机构：

[1] Med Univ Innsbruck, Cyst Fibrosis Ctr Innsbruck, Anichstr 35, A-6020 Innsbruck, Austria

来源：

CLINICAL CASE REPORTS | 2022年 / 10卷 / 02期

关键词：

cystic fibrosis; highly efficient CFTR modulator therapy; pancreas insufficiency; sweat chloride;

D O I：

10.1002/ccr3.5364

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

This study summarizes efficacy of ivacaftor treatment in 2 infants in a real-world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.

引用

页数：4

共 2 条

[1] Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization
Dominique Hubert
Christophe Marguet
Jacques Benichou
Cynthia DeSouza
Catherine Payen-Champenois
Nils Kinnman
Keval Chandarana
Anne Munck
Isabelle Fajac
Pulmonary Therapy, 2021, 7 : 455 - 468
[2] Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization (Jun, 10.1007/s41030-021-00158-5, 2021)
Hubert, Dominique
Marguet, Christophe
Benichou, Jacques
DeSouza, Cynthia
Payen-Champenois, Catherine
Kinnman, Nils
Chandarana, Keval
Munck, Anne
Fajac, Isabelle
PULMONARY THERAPY, 2021, 7 (02) : 469 - 470

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