Elexacaftor/Tezacaftor/Ivacaftor for Mycobacterium abscessus Eradication in a Child With Cystic Fibrosis

Kód výsledku v 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>

Nalezeny alternativní kódy

RIV/00216208:11130/25:10499633 RIV/00216208:11140/25:10499633 RIV/00064203:_____/25:10499633

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

Elexacaftor/Tezacaftor/Ivacaftor for Mycobacterium abscessus Eradication in a Child With Cystic Fibrosis

Popis výsledku v původním jazyce

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.

Název v anglickém jazyce

Elexacaftor/Tezacaftor/Ivacaftor for Mycobacterium abscessus Eradication in a Child With Cystic Fibrosis

Popis výsledku anglicky

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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30203 - Respiratory systems

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Pediatric Pulmonology

ISSN

8755-6863

e-ISSN

1099-0496

Svazek periodika

60

Číslo periodika v rámci svazku

7

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

4

Strana od-do

e71208

Kód UT WoS článku

001529008100001

EID výsledku v databázi Scopus

2-s2.0-105010890012