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非重型血友病:它是良性的吗?-来自 PROBE 研究的见解。

Non-severe haemophilia: Is it benign? - Insights from the PROBE study.

机构信息

Department of Medicine, McMaster University, Hamilton, ON, Canada.

Department of Internal Medicine, Chiang Mai University, Chiang Mai, Thailand.

出版信息

Haemophilia. 2021 Jan;27 Suppl 1:17-24. doi: 10.1111/hae.14105. Epub 2020 Sep 1.

Abstract

INTRODUCTION

There are limited data on the impact of haemophilia on health status and health-related quality of life (HRQL) in people with non-severe (mild and moderate) haemophilia.

AIM

To evaluate the health status of people living with mild or moderate haemophilia.

METHODS

Data on respondents with no bleeding disorder (NoBD), mild and moderate haemophilia patients were drawn from the PROBE study. Respondents were enrolled using network patient organizations. This analysis was performed as a cross-sectional study. Primary outcomes were reported bleeding, acute and chronic pain, activities of daily living and HRQL.

RESULTS

A total of 862 respondents with NoBD (n = 173), mild (n = 102) and moderate (n = 134) haemophilia were eligible, with a median age of 33, 42 and 43, respectively. In relation to haemophilia-related sequalae, 53% of male and 29% of female patients with mild and 83% of males with moderate haemophilia had more than 2-3 bleeds in the last 12 months. Reporting of acute and chronic pain is less in those with NoBD compared to the mild and moderate cohorts for both genders. Multivariate analysis demonstrates significant reductions in quality of life using VAS, EQ-5D-5L and PROBE for males with mild and moderate haemophilia (P ≤ .001) with only PROBE indicating a significant reduction for females with mild (P = .002).

CONCLUSION

People affected by mild or moderate haemophilia report a significant HRQL impact due to haemophilia-related bleeding. Future research is needed to identify the optimal care management of patients with mild and moderate haemophilia.

摘要

简介

关于非重度(轻度和中度)血友病患者的健康状况和健康相关生活质量(HRQL)的影响,数据有限。

目的

评估轻度或中度血友病患者的健康状况。

方法

使用网络患者组织招募无出血性疾病(NoBD)、轻度和中度血友病患者的数据来自 PROBE 研究。本分析作为横断面研究进行。主要结局为报告出血、急性和慢性疼痛、日常生活活动和 HRQL。

结果

共有 862 名无出血性疾病(n=173)、轻度(n=102)和中度(n=134)血友病患者符合条件,中位数年龄分别为 33、42 和 43 岁。关于血友病相关后遗症,53%的男性和 29%的女性轻度患者和 83%的男性中度患者在过去 12 个月中有 2-3 次以上出血。与轻度和中度队列相比,无出血性疾病的患者报告的急性和慢性疼痛较少。多变量分析表明,男性轻度和中度血友病患者的 VAS、EQ-5D-5L 和 PROBE 的生活质量显著降低(P≤.001),仅 PROBE 表明女性轻度患者的生活质量显著降低(P=.002)。

结论

受轻度或中度血友病影响的患者因与血友病相关的出血而报告了显著的 HRQL 影响。需要进一步研究以确定轻度和中度血友病患者的最佳护理管理。

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