Precision medicine in cystic fibrosis: predictive role of forskolin-induced swelling assay

Result code in 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>
Alternative codes found
RIV/00064203:_____/24:10478554 RIV/00216208:11110/24:10478554
Result on the web
<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>

Result language
angličtina
Original language name
Precision medicine in cystic fibrosis: predictive role of forskolin-induced swelling assay
Original language description
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.
Czech name
—
Czech description
—

Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
—
OECD FORD branch
30101 - Human genetics

Project
Result was created during the realization of more than one project. More information in the Projects tab.
Continuities
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)

Publication year
2024
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Similar results(10)