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

Autor:	Picker N; Real-World Evidence, Cytel Inc. Ingress-Health HWM GmbH, Wismar 23966, Germany., Hagiwara M; R&D, Global Evidence and Outcomes, Takeda Development Center Americas, Inc., Cambridge, MA 02139, United States., Baumann S; Real-World Evidence, Cytel Inc. Ingress-Health HWM GmbH, Wismar 23966, Germany., Marins EG; Global Medical Affairs, Takeda Development Center Americas, Inc., Cambridge, MA 02139, United States., Wilke T; IPAM Institute, IPAM E.V., Wismar 23966, Germany., Ren K; Statistics and Quantitative Sciences, Data Science Institute, Takeda Development Center Americas, Inc., Cambridge, MA 02139, United States., Maywald U; Drug Department, AOK PLUS, Dresden 01058, Germany., Karki C; R&D, Global Evidence and Outcomes, Takeda Development Center Americas, Inc., Cambridge, MA 02139, United States., Strnad P; Medical Clinic III, Gastroenterology, Metabolic Diseases and Intensive Care, University Hospital RWTH Aachen, Aachen 52074, Germany. pstrnad@ukaachen.de.
Jazyk:	angličtina
Zdroj:	World journal of hepatology [World J Hepatol] 2023 Oct 27; Vol. 15 (10), pp. 1127-1139.
DOI:	10.4254/wjh.v15.i10.1127
Abstrakt:	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 deficiency. Methods: 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-10 th 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 disease. Outcomes were incidence/prevalence of liver disease in patients with APPM, demographics/baseline characteristics, diagnostic procedures, progression-free survival (PFS), disease progression and mortality. Results: Overall, 2680 and 26299 patients were included in the APPM (fibrosis, 96; cirrhosis, 2584) and control (fibrosis, 1444; cirrhosis, 24855) cohorts, respectively. Per 100000 individuals, annual incidence and prevalence of APPM and liver disease was 10-15 and 36-51, respectively. In 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, cirrhosis, hepatic failure and esophageal/gastric varices than patients with fibrosis in the APPM cohort ( P < 0.001). In the APPM cohort, the most common diagnostic procedures within 12 mo after the first diagnosis of liver disease were imaging procedures (66.3%) and laboratory tests (51.0%). Conclusion: Among patients with liver disease, those with APPM experience substantial burden and earlier liver disease progression than patients without APPM. Competing Interests: Conflict-of-interest statement: Dr. Strnad reports grants and other from CSL Behring, grants and other from Grifols, grants and other from Arrowhead Pharmaceuticals, grants and other from Dicerna Pharmaceuticals, grants from Vertex Pharmaceuticals, other from Albireo, other from GlaxoSmithKline, other from Intellia Pharmaceuticals, other from Ono Pharmaceuticals, other from Takeda Pharmaceuticals, during the conduct of the study. (©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.)
Databáze:	MEDLINE
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