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Effects of elexacaftor/tezacaftor/ivacaftor therapy in children with cystic fibrosis - a comprehensive assessment using lung clearance index, spirometry, and functional and structural lung MRI.

Author: BaumanGrzegorz, BieriOliver, BrabandtBen, BulloMarina, CasaultaCarmen, CurdyMarion, FrauchigerBettina Sarah, KieningerElisabeth, KortenInsa, KrügerLinn, LatzinPhilipp, PusterlaOrso, RatjenFelix, StranzingerEnno, StreibelCarmen, WillersCorin C

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

This study investigates the effects of a combination regimen of elexacaftor, tezacaftor, and ivacaftor on lung abnormalities in patients with cystic fibrosis. The study aims to assess the treatment effect of this regimen on measures of structural and functional lung abnormalities using functional lung MRI with matrix-pencil decomposition.
Paper Details　
Original Abstract of the Article :
With improvement in supportive therapies and the introduction of cystic fibrosis transmembrane conductance regulator (CFTR)-modulator treatment in patients with cystic fibrosis (CF), milder disease courses are expected. Therefore, sensitive parameters are needed to monitor disease course and effects...See full text at original site
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引用元：
https://doi.org/10.1016/j.jcf.2022.12.012

データ提供：米国国立医学図書館（NLM）

Monitoring Cystic Fibrosis Treatment: A Multifaceted Approach

Cystic fibrosis, a genetic disorder that affects the lungs and other organs, can be a challenging journey. This study focuses on the effectiveness of a new combination therapy, elexacaftor/tezacaftor/ivacaftor, in children with cystic fibrosis. The researchers used a variety of tools, including lung clearance index, spirometry, and functional and structural lung MRI, to assess the treatment's impact on lung function and structure. They aimed to determine if this combination therapy could improve lung health and slow disease progression.

A Multifaceted Approach to Assessing Treatment

The study highlights the importance of using multiple methods to assess the effectiveness of new therapies for cystic fibrosis. By combining lung clearance index, spirometry, and functional and structural lung MRI, the researchers obtained a comprehensive picture of the treatment's impact on various aspects of lung health. This multifaceted approach provides a more accurate assessment of the therapy's effectiveness and helps to identify potential benefits and limitations.

Monitoring Cystic Fibrosis Treatment: A Multifaceted Approach

The study's findings emphasize the need for careful monitoring of patients with cystic fibrosis, particularly those receiving new therapies. The researchers found that while the combination therapy showed promise in improving lung function and structure, further research is needed to fully understand its long-term effects and potential benefits for children with cystic fibrosis.

Dr.Camel's Conclusion

This study reminds us that the journey of caring for patients with cystic fibrosis is a long and intricate one, like navigating a vast desert landscape. By embracing new technologies and using multiple approaches to monitor treatment, we can hope to find new oases of hope and better outcomes for patients. This research is a valuable step in the ongoing quest for effective and safe treatments for this complex disease.

Date :
  1. Date Completed 2023-09-11
  2. Date Revised 2023-09-18
Further Info :

Pubmed ID

36635199

DOI: Digital Object Identifier

10.1016/j.jcf.2022.12.012

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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