Precision medicine in cystic fibrosis: predictive role of forskolin-induced swelling assay
Identifikátory výsledku
Kód výsledku v IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F24%3A10478554" target="_blank" >RIV/00216208:11130/24:10478554 - isvavai.cz</a>
Nalezeny alternativní kódy
RIV/00064203:_____/24:10478554 RIV/00216208:11110/24:10478554
Výsledek na webu
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=I8k1WeRy62" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=I8k1WeRy62</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1183/13993003.00156-2024" target="_blank" >10.1183/13993003.00156-2024</a>
Alternativní jazyky
Jazyk výsledku
angličtina
Název v původním jazyce
Precision medicine in cystic fibrosis: predictive role of forskolin-induced swelling assay
Popis výsledku v původním jazyce
People with CF (PwCF) carrying at least one copy of the F508del allele can benefit from a small molecule therapy referred to as CFTR modulators (CFTRm). Triple CFTRm combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) resulted in marked improvement in clinical outcomes such as the percentage of predicted forced expiratory volume in 1 s (ppFEV1) and body mass index (BMI) [1]; however, granular data from the clinical trial also showed that the treatment response to CFTRm varied among PwCF, even among PwCF carrying the same CFTR genotype. To date, only short-circuit-current measurements on human nasal epithelial cells have been validated as a predictive biomarker of CFTRm treatment response [2]. The forskolin-induced swelling assay (FIS) performed on patient-derived intestinal organoids (IOs) has been tested as another candidate tool for predicting the CFTRm response [3-5]. To date, no studies have compared the extent of FIS (i.e., numeric values of the area under the curve, AUC) and the magnitude of clinical response to ELX/TEZ/IVA in real life. Thus, we investigated this correlation to assess whether the FIS can predict clinical outcomes in F508del homozygous PwCF treated with ELX/TEZ/IVA.
Název v anglickém jazyce
Precision medicine in cystic fibrosis: predictive role of forskolin-induced swelling assay
Popis výsledku anglicky
People with CF (PwCF) carrying at least one copy of the F508del allele can benefit from a small molecule therapy referred to as CFTR modulators (CFTRm). Triple CFTRm combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) resulted in marked improvement in clinical outcomes such as the percentage of predicted forced expiratory volume in 1 s (ppFEV1) and body mass index (BMI) [1]; however, granular data from the clinical trial also showed that the treatment response to CFTRm varied among PwCF, even among PwCF carrying the same CFTR genotype. To date, only short-circuit-current measurements on human nasal epithelial cells have been validated as a predictive biomarker of CFTRm treatment response [2]. The forskolin-induced swelling assay (FIS) performed on patient-derived intestinal organoids (IOs) has been tested as another candidate tool for predicting the CFTRm response [3-5]. To date, no studies have compared the extent of FIS (i.e., numeric values of the area under the curve, AUC) and the magnitude of clinical response to ELX/TEZ/IVA in real life. Thus, we investigated this correlation to assess whether the FIS can predict clinical outcomes in F508del homozygous PwCF treated with ELX/TEZ/IVA.
Klasifikace
Druh
J<sub>imp</sub> - Článek v periodiku v databázi Web of Science
CEP obor
—
OECD FORD obor
30101 - Human genetics
Návaznosti výsledku
Projekt
Výsledek vznikl pri realizaci vícero projektů. Více informací v záložce Projekty.
Návaznosti
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)
Ostatní
Rok uplatnění
2024
Kód důvěrnosti údajů
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Údaje specifické pro druh výsledku
Název periodika
European Respiratory Journal
ISSN
0903-1936
e-ISSN
1399-3003
Svazek periodika
63
Číslo periodika v rámci svazku
4
Stát vydavatele periodika
CH - Švýcarská konfederace
Počet stran výsledku
4
Strana od-do
2400156
Kód UT WoS článku
001225873200003
EID výsledku v databázi Scopus
2-s2.0-85191102199