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苯丙酮尿症（PKU）患者的健康经济负担——一项基于德国医疗保险理赔数据的回顾性研究。

Health economic burden of patients with phenylketonuria (PKU) - A retrospective study of German health insurance claims data.

作者信息

Trefz Friedrich, Muntau Ania C, Schneider Kim M, Altevers Julia, Jacob Christian, Braun Sebastian, Greiner Wolfgang, Jha Ashok, Jain Mohit, Alvarez Ignacio, Lane Paul, Zeiss Claudia, Rutsch Frank

机构信息

Zentrum für Kinder- und Jugendmedizin, Universitätsklinikum Heidelberg, Heidelberg, Germany.

University Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

出版信息

Mol Genet Metab Rep. 2021 May 13;27:100764. doi: 10.1016/j.ymgmr.2021.100764. eCollection 2021 Jun.

DOI:10.1016/j.ymgmr.2021.100764
PMID:34036045
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8138676/
Abstract

This retrospective matched-cohort analysis compared health-economic burdens of adults (≥18 years;  = 377) with phenylketonuria (PKU) and age/gender-matched non-PKU controls ( = 3770) in Germany. Healthcare costs and resource-utilization were analyzed for the year 2015. Differences between groups were tested using 95% CI of mean differences (MD). PKU patients had significantly higher mean costs in total (MD €3307, 95% CI €1736-€4879), for pharmaceuticals (MD €1912, 95% CI €1195-€2629) [including dietary amino-acid supplements (MD €1268, 95% CI €864-€1672)], and outpatient costs (MD €395, 95% CI €115-€675). Inpatient costs (MD €904, 95% CI -€293 to €2100) and costs for aids and remedies (MD €97, 95% CI -€10 to €203) were also higher in PKU patients. PKU patients had more outpatient visits and stayed longer in hospital. Adult PKU patients incur higher total healthcare costs than non-PKU controls, especially regarding pharmaceuticals and outpatient costs, and more frequent resource-utilization, resulting in higher health-economic burden for the statutory healthcare system.

摘要

这项回顾性匹配队列分析比较了德国18岁及以上的苯丙酮尿症（PKU）成人患者（n = 377）与年龄/性别匹配的非PKU对照者（n = 3770）的健康经济负担。分析了2015年的医疗保健成本和资源利用情况。使用平均差异（MD）的95%置信区间（CI）对组间差异进行检验。PKU患者的总平均成本显著更高（MD 3307欧元，95% CI 1736欧元 - 4879欧元），药品成本（MD 1912欧元，95% CI 1195欧元 - 2629欧元）[包括膳食氨基酸补充剂（MD 1268欧元，95% CI 864欧元 - 1672欧元）]以及门诊成本（MD 395欧元，95% CI 115欧元 - 675欧元）。PKU患者的住院成本（MD 904欧元，95% CI -293欧元至2100欧元）以及辅助器具和药物成本（MD 97欧元，95% CI -10欧元至203欧元）也更高。PKU患者的门诊就诊次数更多，住院时间更长。成年PKU患者产生的总医疗保健成本高于非PKU对照者，尤其是在药品和门诊成本方面，且资源利用更频繁，给法定医疗保健系统带来了更高的健康经济负担。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/508edeecfabf/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/10d1a740878b/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/98bcc1f6eac9/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/508edeecfabf/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/10d1a740878b/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/98bcc1f6eac9/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/07ac/8138676/508edeecfabf/gr3.jpg

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2
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[ expression and functional analyses of the mutants p.R243Q, p.R241C and p.Y356X of the human phenylalanine hydroxylase].[人苯丙氨酸羟化酶突变体p.R243Q、p.R241C和p.Y356X的表达及功能分析]
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