FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4386886402> ?p ?o ?g. }

• W4386886402 abstract "<h3>Background</h3> Progressive familial intrahepatic cholestasis (PFIC) is a group of rare cholestatic liver diseases characterised by intractable pruritus, elevated serum bile acids (sBAs), and progressive liver damage. NAPPED (NAtural course and Prognosis of PFIC and Effect of biliary Diversion) is a large retrospective database investigating the natural history of PFIC. Odevixibat, an ileal bile acid transporter inhibitor, reduced sBAs and pruritus in patients with PFIC in the phase 3 PEDFIC 1 and PEDFIC 2 studies. We compared clinical outcomes of surgical biliary diversion (SBD), liver transplantation (LT), and death in patients from NAPPED (not treated with odevixibat) with odevixibat-treated patients from the PEDFIC studies. <h3>Methods</h3> The analysis population comprised odevixibat-naive patients from NAPPED and odevixibat-treated patients from PEDFIC 1 and/or PEDFIC 2. Patients with bile salt export pump subtype 3 (BSEP3) mutations were excluded. Eligibility criteria were aligned across cohorts and included genetically proven diagnosis of PFIC1 or PFIC2, sBAs ≥100 µmol/L, alanine aminotransaminase and total bilirubin ≤10× the upper limit of normal, and no prior SBD or LT. Propensity scores, inverse probability of treatment weighting, and matching methods were used to identify and balance baseline covariates, including PFIC1, PFIC2-BSEP1, or PFIC2-BSEP2. The primary endpoint was event-free survival (EFS; time to first event of SBD, LT, or death); secondary endpoints included native liver survival (NLS), SBD-free survival (DFS), and overall survival (OS). Survival outcomes were measured from study day 1; treatment differences were evaluated by weighted log-rank tests and Cox regression. <h3>Results</h3> A cohort of 80 NAPPED patients (controls) was compared with 69 odevixibat-treated patients. Median study duration in the odevixibat cohort was 22.6 months (range: 1.9–39.2 months). Follow-up duration in the NAPPED cohort was truncated accordingly. Odevixibat-treated patients showed significantly higher EFS and DFS than controls (hazard ratio [HR] [95% CI]: 0.20 [0.09−0.45] and 0.13 [0.04−0.39], respectively); numerical improvements in NLS and OS were also observed. Results were consistent when different sensitivity analyses were performed. Additional subgroup analyses indicated that EFS was higher in odevixibat-treated patients with PFIC1 (HR [95% CI]: 0.10 [0.02−0.55]) and PFIC2 (HR [95% CI]: 0.34 [0.12−1.00]) vs controls. <h3>Conclusions</h3> Odevixibat treatment is associated with higher EFS in patients with PFIC without prior SBD upon comparison with matched non-odevixibat-treated patients from the NAPPED registry." @default.
• W4386886402 created "2023-09-21" @default.
• W4386886402 creator A5000708233 @default.
• W4386886402 creator A5005111900 @default.
• W4386886402 creator A5006405927 @default.
• W4386886402 creator A5011058995 @default.
• W4386886402 creator A5013526326 @default.
• W4386886402 creator A5016566105 @default.
• W4386886402 creator A5017063355 @default.
• W4386886402 creator A5019267788 @default.
• W4386886402 creator A5020830252 @default.
• W4386886402 creator A5021839917 @default.
• W4386886402 creator A5022234144 @default.
• W4386886402 creator A5024769297 @default.
• W4386886402 creator A5028213193 @default.
• W4386886402 creator A5029948175 @default.
• W4386886402 creator A5031051209 @default.
• W4386886402 creator A5031718124 @default.
• W4386886402 creator A5032411876 @default.
• W4386886402 creator A5034630836 @default.
• W4386886402 creator A5038401549 @default.
• W4386886402 creator A5038766843 @default.
• W4386886402 creator A5040722876 @default.
• W4386886402 creator A5042062122 @default.
• W4386886402 creator A5045763842 @default.
• W4386886402 creator A5045989725 @default.
• W4386886402 creator A5046006475 @default.
• W4386886402 creator A5048108916 @default.
• W4386886402 creator A5048122975 @default.
• W4386886402 creator A5048242267 @default.
• W4386886402 creator A5048367095 @default.
• W4386886402 creator A5052720375 @default.
• W4386886402 creator A5054821969 @default.
• W4386886402 creator A5054857852 @default.
• W4386886402 creator A5055569132 @default.
• W4386886402 creator A5055875110 @default.
• W4386886402 creator A5064084607 @default.
• W4386886402 creator A5064221142 @default.
• W4386886402 creator A5065353690 @default.
• W4386886402 creator A5065632110 @default.
• W4386886402 creator A5066163290 @default.
• W4386886402 creator A5068992178 @default.
• W4386886402 creator A5073711492 @default.
• W4386886402 creator A5075399641 @default.
• W4386886402 creator A5075460799 @default.
• W4386886402 creator A5075625228 @default.
• W4386886402 creator A5076444077 @default.
• W4386886402 creator A5082541510 @default.
• W4386886402 creator A5083405368 @default.
• W4386886402 creator A5084054094 @default.
• W4386886402 creator A5087598046 @default.
• W4386886402 creator A5089809017 @default.
• W4386886402 creator A5091744236 @default.
• W4386886402 creator A5092510698 @default.
• W4386886402 creator A5092565018 @default.
• W4386886402 creator A5092907880 @default.
• W4386886402 date "2023-09-01" @default.
• W4386886402 modified "2023-10-02" @default.
• W4386886402 title "P1 Analysis of long-term treatment effects of odevixibat on clinical outcomes in children with progressive familial intrahepatic cholestasis in odevixibat clinical studies vs external controls from the NAPPED database" @default.
• W4386886402 doi "https://doi.org/10.1136/gutjnl-2023-basl.17" @default.
• W4386886402 hasPublicationYear "2023" @default.
• W4386886402 type Work @default.
• W4386886402 citedByCount "0" @default.
• W4386886402 crossrefType "proceedings-article" @default.
• W4386886402 hasAuthorship W4386886402A5000708233 @default.
• W4386886402 hasAuthorship W4386886402A5005111900 @default.
• W4386886402 hasAuthorship W4386886402A5006405927 @default.
• W4386886402 hasAuthorship W4386886402A5011058995 @default.
• W4386886402 hasAuthorship W4386886402A5013526326 @default.
• W4386886402 hasAuthorship W4386886402A5016566105 @default.
• W4386886402 hasAuthorship W4386886402A5017063355 @default.
• W4386886402 hasAuthorship W4386886402A5019267788 @default.
• W4386886402 hasAuthorship W4386886402A5020830252 @default.
• W4386886402 hasAuthorship W4386886402A5021839917 @default.
• W4386886402 hasAuthorship W4386886402A5022234144 @default.
• W4386886402 hasAuthorship W4386886402A5024769297 @default.
• W4386886402 hasAuthorship W4386886402A5028213193 @default.
• W4386886402 hasAuthorship W4386886402A5029948175 @default.
• W4386886402 hasAuthorship W4386886402A5031051209 @default.
• W4386886402 hasAuthorship W4386886402A5031718124 @default.
• W4386886402 hasAuthorship W4386886402A5032411876 @default.
• W4386886402 hasAuthorship W4386886402A5034630836 @default.
• W4386886402 hasAuthorship W4386886402A5038401549 @default.
• W4386886402 hasAuthorship W4386886402A5038766843 @default.
• W4386886402 hasAuthorship W4386886402A5040722876 @default.
• W4386886402 hasAuthorship W4386886402A5042062122 @default.
• W4386886402 hasAuthorship W4386886402A5045763842 @default.
• W4386886402 hasAuthorship W4386886402A5045989725 @default.
• W4386886402 hasAuthorship W4386886402A5046006475 @default.
• W4386886402 hasAuthorship W4386886402A5048108916 @default.
• W4386886402 hasAuthorship W4386886402A5048122975 @default.
• W4386886402 hasAuthorship W4386886402A5048242267 @default.
• W4386886402 hasAuthorship W4386886402A5048367095 @default.
• W4386886402 hasAuthorship W4386886402A5052720375 @default.
• W4386886402 hasAuthorship W4386886402A5054821969 @default.
• W4386886402 hasAuthorship W4386886402A5054857852 @default.
• W4386886402 hasAuthorship W4386886402A5055569132 @default.
• W4386886402 hasAuthorship W4386886402A5055875110 @default.
• W4386886402 hasAuthorship W4386886402A5064084607 @default.
• W4386886402 hasAuthorship W4386886402A5064221142 @default.

• next