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德国镰状细胞病伴复发性血管闭塞性危象以及输血依赖型β地中海贫血传统治疗相关的临床并发症和医疗资源利用情况

Clinical Complications and Healthcare Resource Utilization Associated with Conventional Management of Sickle Cell Disease with Recurrent Vaso-occlusive Crises and Transfusion-Dependent β-Thalassemia in Germany.

作者信息

Udeze Chuka, Li Nanxin, Kunzweiler Colin, Baldwin Jessica, Tuzin Petra, Zingel Sebastian Dietmar, Vetter Céline, Dombrowski Silvia, Georgiadou-Schmidt Elena, Alba Aranzazu, Meisel Roland

机构信息

Health Economics and Outcomes Research, Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA.

IQVIA Commercial GmbH & Co. OHG, Frankfurt, Germany.

出版信息

Pharmacoecon Open. 2025 Mar;9(2):291-300. doi: 10.1007/s41669-024-00550-2. Epub 2025 Jan 3.

Abstract

OBJECTIVE

The purpose of this study was to describe clinical complications and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and patients with transfusion-dependent β-thalassemia (TDT) in Germany.

METHODS

The Betriebskrankenkasse (BKKs) Database was used to identify patients with SCD or TDT. To be eligible for inclusion, patients with SCD were required to have ≥ 2 VOCs/year in any two consecutive years and ≥ 12 months of available data before and after the index date (second VOC in the second consecutive year). Patients with TDT were required to have ≥ 8 red blood cell transfusions (RBCTs) in any 12-month period and ≥ 12 months of available data after the index date (first RBCT). Clinical and HCRU outcomes were analyzed during follow-up.

RESULTS

Overall, 84 patients with SCD with recurrent VOCs and 68 patients with TDT were identified in the BKKs database. Among patients with SCD with recurrent VOCs, the most prevalent complications were retinopathy (45.2%), multisystem organ disease/failure (40.5%), and mental health complications (31.0%); among patients with TDT, they were endocrine (69.1%) and cardiopulmonary (55.9%) complications and malignancies (44.1%). Patients with SCD experienced a mean of 4.0 (standard deviation [SD] 3.9) VOCs and 1.9 (SD 2.5) hospitalizations per patient per year (PPPY) during follow-up. Patients with TDT had a mean (SD) of 16.4 (11.2) RBCTs and 59.4 (40.8) outpatient visits PPPY.

CONCLUSIONS

Patients with SCD with recurrent VOCs or TDT in Germany experience significant clinical complications and HCRU.

摘要

目的

本研究旨在描述德国镰状细胞病(SCD)伴复发性血管闭塞性危机(VOCs)患者以及输血依赖型β地中海贫血(TDT)患者的临床并发症和医疗资源利用情况(HCRU)。

方法

利用企业健康保险基金(BKKs)数据库识别SCD或TDT患者。SCD患者若符合纳入标准,需在任意连续两年中每年发生≥2次VOCs,且在索引日期(连续第二年的第二次VOC)前后有≥12个月的可用数据。TDT患者需在任意12个月期间接受≥8次红细胞输血(RBCT),且在索引日期(首次RBCT)后有≥12个月的可用数据。在随访期间分析临床和HCRU结局。

结果

总体而言,在BKKs数据库中识别出84例SCD伴复发性VOCs患者和68例TDT患者。在SCD伴复发性VOCs患者中,最常见的并发症是视网膜病变(45.2%)、多系统器官疾病/功能衰竭(40.5%)和心理健康并发症(31.0%);在TDT患者中,是内分泌(69.1%)和心肺(55.9%)并发症以及恶性肿瘤(44.1%)。在随访期间,SCD患者每位患者每年平均发生4.0次(标准差[SD] 3.9)VOCs和1.9次(SD 2.5)住院。TDT患者每位患者每年平均(SD)接受16.4次(11.2次)RBCT和59.4次(40.8次)门诊就诊。

结论

德国SCD伴复发性VOCs或TDT患者经历了显著的临床并发症和HCRU。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1fcf/11865411/610c94928ca7/41669_2024_550_Fig1_HTML.jpg

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