Epidemiology and treatment of children with hereditary angioedema in Germany: A retrospective database study

IF 4.6 2区 医学 Q2 ALLERGY
Freerk Prenzel, Susanne Abraham, Christoph Hirche, Gerrit Müller, Stephan Kaiser, Leonarda Serdani-Neuhaus, Rebecca Zingel, Inmaculada Martinez-Saguer
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引用次数: 0

Abstract

Background

Hereditary angioedema (HAE) is a potentially life-threatening inherited disease that causes recurrent, serious, and debilitating episodes of swelling. While evidence has improved in adult patients, data on the epidemiology and treatment of pediatric patients with HAE remain very limited. The aim of this study was to determine the incidence and prevalence of pediatric patients with HAE aged <12 years, as well as treatment patterns, co-medication, and specialties involved.

Methods

In this retrospective study (2016–2021), the German IQVIATM pharmacy claims (LRx) database was used to analyze prescriptions of HAE-specific treatments and co-medications.

Results

We found an HAE prevalence in pediatric patients aged <12 years of 2.51:100,000 and a 12-month prevalence of up to 1.02:100,000 between 2016 and 2021. Most HAE treatments were prescribed by outpatient clinics and pediatricians, with an increasing proportion of icatibant as an on-demand treatment and low rates of long-term prophylaxis (LTP). The prescription rate of analgesics as the most common co-medication decreased notably after HAE diagnosis.

Conclusion

Our findings provide insights into the epidemiology and current pediatric HAE treatment landscape in Germany. The obtained HAE prevalence in pediatric patients aged <12 years was even higher than the previously reported average of overall cohorts, whereas the LTP rate was low, which might indicate an unmet need for newer LTP treatment options in pediatric patients.

Abstract Image

德国儿童遗传性血管性水肿的流行病学和治疗:回顾性数据库研究
背景:遗传性血管性水肿(HAE)是一种潜在威胁生命的遗传性疾病,可引起反复、严重和使人衰弱的肿胀发作。虽然成人患者的证据有所改善,但关于儿童HAE患者的流行病学和治疗的数据仍然非常有限。本研究的目的是确定12岁儿童HAE患者的发病率和患病率,以及治疗模式、联合用药和涉及的专科。方法回顾性研究(2016-2021年)采用德国IQVIATM药房索赔(LRx)数据库对hae特异性治疗和联合用药处方进行分析。结果我们发现,2016年至2021年间,12岁儿童HAE患病率为2.51:10万,12个月患病率高达1.02:10万。大多数HAE治疗是由门诊诊所和儿科医生开出的,伊卡替特作为按需治疗的比例越来越高,长期预防(LTP)率很低。在HAE诊断后,镇痛药作为最常见的联合用药的处方率显著下降。结论:我们的研究结果为了解德国儿童HAE的流行病学和治疗现状提供了见解。在12岁儿童患者中获得的HAE患病率甚至高于之前报道的总体队列的平均值,而LTP率很低,这可能表明儿科患者对较新的LTP治疗方案的需求尚未得到满足。
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来源期刊
Clinical and Translational Allergy
Clinical and Translational Allergy Immunology and Microbiology-Immunology
CiteScore
7.50
自引率
4.50%
发文量
117
审稿时长
12 weeks
期刊介绍: Clinical and Translational Allergy, one of several journals in the portfolio of the European Academy of Allergy and Clinical Immunology, provides a platform for the dissemination of allergy research and reviews, as well as EAACI position papers, task force reports and guidelines, amongst an international scientific audience. Clinical and Translational Allergy accepts clinical and translational research in the following areas and other related topics: asthma, rhinitis, rhinosinusitis, drug hypersensitivity, allergic conjunctivitis, allergic skin diseases, atopic eczema, urticaria, angioedema, venom hypersensitivity, anaphylaxis, food allergy, immunotherapy, immune modulators and biologics, animal models of allergic disease, immune mechanisms, or any other topic related to allergic disease.
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