PDF(Pubmed)
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.
CONCLUSIONS: 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.
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.
CONCLUSIONS: 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.
摘要：
背景：遗传性血管性水肿（HAE）是一种可能危及生命的遗传性疾病，严肃,和衰弱的肿胀发作。虽然成年患者的证据有所改善，关于HAE儿科患者的流行病学和治疗的数据仍然非常有限.这项研究的目的是确定儿童HAE患者年龄<12岁的发病率和患病率。以及治疗模式，联合用药,和涉及的专业。
方法：在这项回顾性研究（2016-2021年）中，使用德国IQVIATM药房声明(LRx)数据库分析HAE特异性治疗和联合用药的处方.
结果：我们发现，在2016年至2021年期间，年龄<12岁的儿科患者中HAE患病率为2.51:100,000，12个月患病率高达1.02:100,000。大多数HAE治疗是由门诊和儿科医生开出的，icatibant作为按需治疗的比例不断增加，长期预防（LTP）的比例较低。在HAE诊断后，作为最常见的联合用药的镇痛药的处方率明显下降。
结论：我们的发现提供了对德国流行病学和当前儿科HAE治疗前景的见解。在<12岁的儿科患者中获得的HAE患病率甚至高于先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更新的LTP治疗方案的需求未得到满足。
方法：在这项回顾性研究（2016-2021年）中，使用德国IQVIATM药房声明(LRx)数据库分析HAE特异性治疗和联合用药的处方.
结果：我们发现，在2016年至2021年期间，年龄<12岁的儿科患者中HAE患病率为2.51:100,000，12个月患病率高达1.02:100,000。大多数HAE治疗是由门诊和儿科医生开出的，icatibant作为按需治疗的比例不断增加，长期预防（LTP）的比例较低。在HAE诊断后，作为最常见的联合用药的镇痛药的处方率明显下降。
结论：我们的发现提供了对德国流行病学和当前儿科HAE治疗前景的见解。在<12岁的儿科患者中获得的HAE患病率甚至高于先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更新的LTP治疗方案的需求未得到满足。
