欧洲血友病 A 成人队列中出血事件频率对健康相关生活质量和工作生产力结局的影响：来自 CHESS II 研究的见解。

The impact of bleeding event frequency on health-related quality of life and work productivity outcomes in a European cohort of adults with haemophilia A: insights from the CHESS II study.

机构信息

Pfizer Ltd, Walton-on-the-Hill Tadworth, Surrey, UK.

Pfizer Inc, Collegeville, PA, USA.

出版信息

Orphanet J Rare Dis. 2023 Aug 3;18(1):227. doi: 10.1186/s13023-023-02690-w.

DOI:10.1186/s13023-023-02690-w

PMID:37537683

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10398941/

Abstract

BACKGROUND

Haemophilia A carries a substantial healthcare burden, affecting health-related quality of life (HRQoL). The Cost of Haemophilia in Men: a Socioeconomic Survey II (CHESS II), a retrospective real-world study, characterised the burden of haemophilia and its impact on HRQoL and work productivity. The current analysis explored the impact of bleeding events on HRQoL and work productivity in Europe. This analysis focused on data collected from males aged 18 to 64 years with haemophilia A without inhibitors who were receiving replacement factor products or a monoclonal antibody and were not participating in a clinical trial at the time of study recruitment. Descriptive statistics were analysed using scores from EuroQoL's EQ-5D-5L index and EQ-VAS analogue scale and the Work Productivity and Activity Index Specific Health Problem (WPAI:SHP) percentage scores stratified by the number of annual bleeding events (ABs) 0, 1, 2, 3-4, or ≥ 5.

RESULTS

Of 918 males with haemophilia A in CHESS II, 318 met inclusion criteria and had data available for HRQoL measures; mean age (SD) was 33.8 (12.1) years and 96% were White. Mean (SD) ABs of 2.7 (2.9) occurred over the preceding 12 months: 20% had 3 or 4 ABs; 17% had ≥ 5 ABs. Mean EQ-5D-5L index scores for patients with 0, 1, 2, 3-4, or ≥ 5 ABs were 0.92, 0.76, 0.76, 0.71, and 0.56, respectively. Mean (SD) EQ-VAS scores were 86.9 (13.6), with 0 ABs versus 69.5 (19.1) for 3 or 4 ABs and 61.2 (17.2) for ≥ 5 ABs. Mean percentage of overall work productivity loss on the WPAI:SHP questionnaire ranged from 9.70 to 0 ABs to 47.65 for ≥ 5 ABs.

CONCLUSIONS

In this European sample of adult men with haemophilia A, HRQoL and work productivity scores were lower among those reporting more AB events. Bleeding burden appears to affect HRQoL and productivity; however, this cross-sectional analysis limits the ability to draw firm conclusions on causality.

摘要

背景

甲型血友病会给患者带来沉重的医疗负担，影响其健康相关生活质量（HRQoL）。《男性血友病的成本：一项社会经济学调查 II（CHESS II）》是一项回顾性真实世界研究，描述了血友病的负担及其对 HRQoL 和工作生产力的影响。目前的分析探讨了出血事件对欧洲甲型血友病患者 HRQoL 和工作生产力的影响。本分析重点关注了在研究招募时未参加临床试验且无抑制剂的 18-64 岁甲型血友病男性患者的医疗数据，这些患者正在接受替代因子产品或单克隆抗体治疗。使用 EuroQoL 的 EQ-5D-5L 指数和 EQ-VAS 模拟量表的评分以及按每年出血事件（ABs）数量（0、1、2、3-4 或≥5）分层的工作生产力和活动指数特定健康问题（WPAI:SHP）百分比评分分析了描述性统计数据。

结果

在 CHESS II 中的 918 名甲型血友病男性中，有 318 名符合纳入标准，且有 HRQoL 测量数据；平均年龄（标准差）为 33.8（12.1）岁，96%为白人。过去 12 个月中 ABs 的平均（标准差）为 2.7（2.9）次：20%有 3 或 4 次 ABs；17%有≥5 次 ABs。ABs 数量为 0、1、2、3-4 或≥5 的患者的 EQ-5D-5L 指数平均（标准差）分别为 0.92、0.76、0.76、0.71 和 0.56。EQ-VAS 平均（标准差）评分分别为 86.9（13.6）和 0 ABs 与 3 或 4 ABs 时的 69.5（19.1）和≥5 ABs 时的 61.2（17.2）。WPAI:SHP 问卷中总工作生产力损失的平均百分比范围从 ABs 数量为 0 时的 9.70%到 ABs 数量为 5 时的 47.65%。