Elexacaftor/Tezacaftor/Ivacaftor for Mycobacterium abscessus Eradication in a Child With Cystic Fibrosis
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00669806%3A_____%2F25%3A10499633" target="_blank" >RIV/00669806:_____/25:10499633 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/25:10499633 RIV/00216208:11140/25:10499633 RIV/00064203:_____/25:10499633
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Uw4DJdaMT8" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Uw4DJdaMT8</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1002/ppul.71208" target="_blank" >10.1002/ppul.71208</a>
Alternative languages
Result language
angličtina
Original language name
Elexacaftor/Tezacaftor/Ivacaftor for Mycobacterium abscessus Eradication in a Child With Cystic Fibrosis
Original language description
A boy was diagnosed with cystic fibrosis (CF) through nationwide newborn screening using an immunoreactive trypsinogen genetic algorithm. The first chest computed tomography (CT) scan, performed at 4.7 years of age, showed significant bronchiectasis, airway wall thickening, and mucus plugging. At the age of 7.5 years, chest CT revealed significant progression of bronchiectasis in the right lung accompanied by worsening peripheral airway obstruction on spirometry. Lumacaftor/ivacaftor cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy was initiated at the age of 10 years. Elexacaftor/tezacaftor/ivacaftor (ETI) therapy was initiated at the age of 12 years. After 1 year of ETI therapy (age, 13 years), the first MAB-negative culture was documented, along with significant regression in CT findings. Our case report supports the potential efficacy of CFTR modulator therapy for eradicating MAB infections in individuals with CF. In this case, MAB negativity could not be achieved with prolonged (52 months) guideline-based antibiotic therapy but occurred after 12 months of combined antibiotic and ETI treatment. Although a direct causal relationship between ETI and MAB eradication cannot be definitively established, our findings support this hypothesis through unique, detailed longitudinal data. The observed clinical, microbiological, and radiological improvements, including normalization of spirometric values and substantial regression of structural lung disease as quantified by the Brody II score, highlight the potential added benefit of ETI in people with CF (pwCF) with advanced lung disease and provide a meaningful contribution to the existing literature.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30203 - Respiratory systems
Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2025
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Pediatric Pulmonology
ISSN
8755-6863
e-ISSN
1099-0496
Volume of the periodical
60
Issue of the periodical within the volume
7
Country of publishing house
US - UNITED STATES
Number of pages
4
Pages from-to
e71208
UT code for WoS article
001529008100001
EID of the result in the Scopus database
2-s2.0-105010890012