Retrospective Database Analysis of Liver-Related Clinical Events in Adult and Pediatric Patients with Alpha-1 Antitrypsin Deficiency in the United States.

IF 2.6 Q2 GASTROENTEROLOGY & HEPATOLOGY
Hepatic Medicine : Evidence and Research Pub Date : 2024-07-24 eCollection Date: 2024-01-01 DOI:10.2147/HMER.S469769
May Hagiwara, Victoria Divino, Swapna Munnangi, Mark Delegge, Suna Park, Ed G Marins, Kaili Ren, Charlie Strange
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引用次数: 0

Abstract

Background and aims: Real-world analyses on burden of illness in patients with alpha-1 antitrypsin deficiency (AATD) are limited. We investigated the real-world burden of liver-related clinical events among adult and pediatric patients with AATD in the USA.

Methods: This was a retrospective, observational analysis of administrative claims data from the IQVIA PharMetrics® Plus and Ambulatory Electronic Medical Records databases from 2011 to 2022. Patients had a diagnosis of liver and/or lung disease with ≥180 days of continuous enrollment in the IQVIA PharMetrics Plus database before and ≥90 days after their first diagnosis. Follow-up time was assigned to the AATD with liver disease health state or AATD with both liver and lung disease health state (for patients aged ≥18 years only). Baseline demographic characteristics and liver-related clinical events of interest were reported.

Results: Of 5136 eligible patients, 771 adult and 123 pediatric patients contributed time to the AATD with liver disease health state; 541 adults contributed time to the AATD with both liver and lung disease health state. Among adults, patients with both liver and lung disease had higher rates of liver-related clinical events than patients with liver disease alone. Ascites was the most frequently observed clinical event among adults in both health states, and the median time to the composite of any liver-related clinical event was 26.5 days among all adults combined. Across all pediatric age groups, ascites, gastrointestinal bleed and hepatic encephalopathy were more common than spontaneous bacterial peritonitis and hepatocellular carcinoma, but median time to liver-related clinical event varied by age group at index date and type of event. No liver transplantations occurred in patients aged 6-17 years.

Conclusion: Diagnosed AATD with liver disease carries a substantial burden on adult and pediatric patients; new treatment options are warranted to avoid disease progression to decompensating events.

美国成人和儿童 Alpha-1 抗胰蛋白酶缺乏症患者肝脏相关临床事件的回顾性数据库分析。
背景和目的:有关α-1抗胰蛋白酶缺乏症(AATD)患者疾病负担的真实世界分析非常有限。我们调查了美国成人和儿童 AATD 患者肝脏相关临床事件的实际负担:这是一项回顾性观察分析,研究对象是2011年至2022年期间来自IQVIA PharMetrics® Plus和非住院电子病历数据库的行政报销数据。患者被诊断为肝病和/或肺病,在首次诊断前和首次诊断后≥90天连续加入IQVIA PharMetrics Plus数据库≥180天。随访时间被分配到有肝病健康状态的 AATD 或有肝病和肺病健康状态的 AATD(仅适用于年龄≥18 岁的患者)。报告了基线人口统计学特征和与肝脏相关的临床事件:在5136名符合条件的患者中,771名成人患者和123名儿童患者在AATD中贡献了肝脏疾病健康状态的时间;541名成人患者在AATD中贡献了肝脏和肺部疾病健康状态的时间。在成人患者中,同时患有肝病和肺病的患者发生肝脏相关临床事件的比例高于仅患有肝病的患者。腹水是两种健康状况的成人中最常观察到的临床事件,所有成人中发生任何肝脏相关临床事件的复合时间中位数为 26.5 天。在所有儿童年龄组中,腹水、消化道出血和肝性脑病比自发性细菌性腹膜炎和肝细胞癌更常见,但发生肝脏相关临床事件的中位时间因发病日年龄组和事件类型而异。6-17岁的患者没有发生肝移植:结论:确诊的AATD合并肝脏疾病给成人和儿童患者带来了沉重的负担;需要新的治疗方案来避免疾病发展到失代偿期。
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来源期刊
Hepatic Medicine : Evidence and Research
Hepatic Medicine : Evidence and Research GASTROENTEROLOGY & HEPATOLOGY-
自引率
0.00%
发文量
15
审稿时长
16 weeks
期刊介绍: Hepatic Medicine: Evidence and Research is an international, peer-reviewed, open access, online journal. Publishing original research, reports, editorials, reviews and commentaries on all aspects of adult and pediatric hepatology in the clinic and laboratory including the following topics: Pathology, pathophysiology of hepatic disease Investigation and treatment of hepatic disease Pharmacology of drugs used for the treatment of hepatic disease Although the main focus of the journal is to publish research and clinical results in humans; preclinical, animal and in vitro studies will be published where they will shed light on disease processes and potential new therapies. Issues of patient safety and quality of care will also be considered. As of 1st April 2019, Hepatic Medicine: Evidence and Research will no longer consider meta-analyses for publication.
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