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001-es BibID:	BIBFORM113535
035-os BibID:	(scopus)85153208380 (WoS)001129085700001
Első szerző:	Pócsi Marianna (klinikai laboratóriumi kutató)
Cím:	Human epididymis protein 4 (HE4) plasma concentration inversely correlates with the improvement of cystic fibrosis lung disease in p.Phe508del-CFTR homozygous cases treated with the CFTR modulator lumacaftor/ivacaftor combination / Marianna Pócsi, Zsolt Fejes, Zsolt Bene, Attila Nagy, István Balogh, Margarida D. Amaral, Milan Macek Jr., Béla Nagy Jr.
Dátum:	2023
ISSN:	1569-1993 1873-5010
Megjegyzések:	Background: We previously documented that elevated HE4 plasma concentration decreased in people with CF (pwCF) bearing the p.Gly551Asp-CFTR variant in response to CFTR modulator (CFTRm) ivacaftor (IVA), and this level was inversely correlated with the FEV1% predicted values (ppFEV1). Although the effectiveness of lumacaftor (LUM)/IVA in pwCF homozygous for the p.Phe508del-CFTR variant has been evaluated, plasma biomarkers were not used to monitor treatment efficacy thus far. Methods: Plasma HE4 concentration was examined in 68 pwCF drawn from the PROSPECT study who were homozygous for the p.Phe508del-CFTR variant before treatment and at 1, 3, 6 and 12 months after administration of LUM/IVA therapy. Plasma HE4 was correlated with ppFEV1 using their absolute and delta values. The discriminatory power of delta HE4 was evaluated for the detection of lung function improvements based on ROC-AUC analysis and multiple regression test. Results: HE4 plasma concentration was significantly reduced below baseline following LUM/IVA administration during the entire study period. The mean change of ppFEV1 was 2.6% (95% CI, 0.6 to 4.5) by 6 months of therapy in this sub-cohort. A significant inverse correlation between delta values of HE4 and ppFEV1 was observed especially in children with CF (r=-0.7053; p<0.0001). Delta HE4 predicted a 2.6% mean change in ppFEV1 (AUC: 0.7898 [95% CI 0.6823?0.8972]; P < 0.0001) at a cut-off value of -10.7 pmol/L. Moreover, delta HE4 independently represented the likelihood of being a responder with ? 5% delta ppFEV1 at 6 months (OR: 0.89, 95% CI: 0.82?0.95; P = 0.001). Conclusions: Plasma HE4 level negatively correlates with lung function improvement assessed by ppFEV1 in pwCF undergoing LUM/IVA CFTRm treatment. ? 2023
Tárgyszavak:	Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban folyóiratcikk
Megjelenés:	Journal Of Cystic Fibrosis. - 22 : 6 (2023), p. 1085-1092. -
További szerzők:	Fejes Zsolt (1988-) (molekuláris biológus) Bene Zsolt (1981-) (orvos) Nagy Attila Csaba (1981-) (megelőző orvostan és népegészségtan szakorvos, epidemiológus) Balogh István (1972-) (molekuláris biológus, genetikus) Amaral, Margarida D. Macek Jr., Milan Nagy Béla Jr. (1980-) (labordiagnosztikai szakorvos)
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