背景:免疫性血小板减少症(ITP)的特点是血小板计数低,常导致出血,疲劳,降低了与健康相关的生活质量。方法这种观察,回顾性,使用BIG-PAC®数据库的基于人群的研究纳入了2014年至2020年在初级保健和医院诊断为原发性ITP的西班牙儿科和成年患者(中位随访时间:4年).流行病学,基线/临床特征,治疗趋势,对医疗资源和成本进行了分析。结果BIG-PAC®数据库包含1,818,588名患者的记录;170名成人和27名ITP儿童被纳入我们的分析。估计每100,000例ITP患病率和年发病率分别为10.8(慢性ITP[cITP]患者为2.8)和1.5(cITP患者为0.3),分别。鼻出血是最常见的出血事件,其次是泌尿生殖系统和消化道出血;>50%/>75%的ITP/cITP患者报告疲劳。慢性患者基线时血小板计数较低,需要更多输血。皮质类固醇,免疫抑制剂,血小板生成素受体激动剂是最常用的药物,二线和三线治疗,分别。35个病人,他们都处于慢性期,做了脾切除术.患者平均每年有13.9、6.6和1.2次就诊于初级保健,血液学/内科,和急诊科,分别。超过四分之一的成年患者平均每年请病假16.3天。平均年度总医疗保健费用为10,741欧元(ITP患者)和19,809欧元(cITP患者)。结论这是第一项从流行病学角度对西班牙ITP人群的状况提供整体观点的研究,治疗趋势,医疗保健资源和成本,突出未满足的患者需求,以及2014年至2020年之间的直接和间接成本/资源使用。
Background Immune thrombocytopenia (ITP) is characterised by low platelet counts and often leads to bleeding, fatigue, and reduced health-related quality of life. Methods This observational, retrospective, population-based study using BIG-PAC® database included Spanish paediatric and adult patients with primary ITP diagnosed in primary care and hospitals between 2014 and 2020 (median follow-up: 4 years). Epidemiology, baseline/clinical characteristics, treatment trends, healthcare resources and costs were analysed. Results The BIG-PAC® database contains records of 1,818,588 patients; 170 adults and 27 children with ITP were included in our analysis. ITP prevalence and annual incidence per 100,000 were estimated in 10.8 (2.8 in chronic ITP [cITP] patients) and 1.5 (0.3 in cITP patients), respectively. Epistaxis was the most common bleeding event, followed by genitourinary and gastrointestinal bleeding; >50%/> 75% of ITP/cITP patients reported fatigue. Chronic patients had lower platelet counts at baseline and required more transfusions. Corticosteroids, immunosuppressants, and thrombopoietin receptor agonists were the most used agents in first-, second- and third-line treatment, respectively. Thirty-five patients, all of them in chronic phase, underwent splenectomy. Patients had on average 13.9, 6.6, and 1.2 visits/year to primary care, haematology/internal medicine, and emergency departments, respectively. More than one-fourth of adult patients took on average 16.3 days of sick leave annually. Mean annual total health care costs were €10,741 (ITP patients) and €19,809 (cITP patients). Conclusion This is the first study to provide an overall perspective on the situation of the Spanish ITP population in terms of epidemiology, treatment trends, health care resources and costs, highlighting unmet patient needs, and direct and indirect costs/resource use between 2014 and 2020.