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Liver disease epidemiology and burden in patients with alterations in plasma protein metabolism:German retrospective insurance claims analysis

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World Journal of Hepatology 2023年第10期15卷 1127-1139页

作者： Nils Picker May Hagiwara severin baumann Ed G Marins Thomas Wilke Kaili Ren Ulf Maywald ChitraKarki Pavel Strnad Real-World Evidence Cytel Inc.Ingress-Health HWM GmbHWismar 23966Germany R&D Global Evidence and OutcomesTakeda Development Center AmericasInc.CambridgeMA 02139United States Global Medical Affairs Takeda Development Center AmericasInc.CambridgeMA 02139United States IPAM Institute IPAM E.V.Wismar 23966Germany Statistics and Quantitative Sciences Data Science InstituteTakeda Development Center AmericasInc.CambridgeMA 02139United States Drug Department AOK PLUSDresden 01058Germany Medical Clinic III GastroenterologyMetabolic Diseases and Intensive CareUniversity Hospital RWTH AachenAachen 52074Germany

BACKGROUND Alpha-1 antitrypsin deficiency is a rare genetic disease and a leading cause of inherited alterations in plasma protein metabolism(APPM).AIM To understand the prevalence,burden and progression of liver disease in patients with APPM including alpha-1 antitrypsin *** We conducted a retrospective analysis of anonymized patient-level claims data from a German health insurance provider(AOK PLUS).The APPM cohort comprised patients with APPM(identified using the German Modification of the International Classification of Diseases-10th Revision[ICD-10-GM]code E88.0 between 01/01/2010-30/09/2020)and incident liver disease(ICD-10-GM codes K74,K70.2-3 and K71.7 between 01/01/2012-30/09/2020).The control cohort comprised patients without APPM but with incident liver *** were incidence/prevalence of liver disease in patients with APPM,demographics/baseline characteristics,diagnostic procedures,progression-free survival(PFS),disease progression and *** Overall,2680 and 26299 patients were included in the APPM(fibrosis,96;cirrhosis,2584)and control(fibrosis,1444;cirrhosis,24855)cohorts,*** 100000 individuals,annual incidence and prevalence of APPM and liver disease was 10-15 and 36-51,*** the APPM cohort,median survival was 4.7 years[95%confidence interval(CI):3.5-7.0]and 2.5 years(95%CI:2.3-2.8)in patients with fibrosis and cirrhosis,respectively.A higher proportion of patients in the APPM cohort experienced disease progression(92.0%)compared with the control cohort(67.2%).Median PFS was shorter in the APPM cohort(0.9 years,95%CI:0.7-1.1)compared with the control cohort(3.7 years,95%CI:3.6-3.8;P<0.001).Patients with cirrhosis in the control cohort had longer event-free survival for ascites,hepatic encephalopathy,hepatic failure and esophageal/gastric varices than patients with cirrhosis in the APPM cohort(P<0.001).Patients with fibrosis in the control cohort had longer event-free survival for ascites,cir

关键词： Alpha-1 antitrypsin deficiency Epidemiology Germany Liver diseases Retrospective study

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