血友病伴踝关节关节病患者健康相关生活质量和足踝关节患者报告结局测量指标下降。

Decline in health-related quality of life and foot and ankle patient reported outcomes measures in patients with haemophilia and ankle haemarthropathy.

机构信息

Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK.

Leeds Haemophilia Comprehensive Care Centre, Leeds Teaching Hospitals NHS Trust, Leeds, UK.

出版信息

J Foot Ankle Res. 2023 Mar 10;16(1):12. doi: 10.1186/s13047-023-00611-5.

DOI:10.1186/s13047-023-00611-5

PMID:36899385

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10007846/

Abstract

BACKGROUND

Haemophilia is an X-linked recessive genetic disorder characterised by bleeding within soft tissue and joints. The ankle is disproportionally affected by haemarthropathy when compared to the elbows and knees; reported as the most affected joints in patients with haemophilia. Despite advances in treatment, patients still report ongoing pain and disability, however, the impact has not been evaluated, nor has the effect on health-related quality of life (HRQoL) or foot and ankle patient-reported outcome measures (PROMs). The primary aim of this study was to establish the impact of ankle haemarthropathy in patients with severe and moderate haemophilia A and B. Secondly to identify the clinical outcomes associated with a decline in HRQoL and foot and ankle PROMs.

METHODS

A cross-sectional multi-centre questionnaire study was conducted across 18 haemophilia centres in England, Scotland and Wales with a recruitment target of 245 participants. The HAEMO-QoL-A and Manchester-Oxford Foot Questionnaire (MOXFQ) (foot and ankle) with total and domain scores measured impact on HRQOL and foot and ankle outcomes. Demographics, clinical characteristics, ankle haemophilia joint health scores, multi-joint haemarthropathy and Numerical Pain Rating Scales (NPRS) of "ankle pain over the past six months" were collected as a measure of chronic ankle pain.

RESULTS

A total of 243 of 250 participants provided complete data. HAEMO-QoL-A and MOXFQ (foot and ankle) total and index scores indicated worse HRQoL with total scores ranging from a mean of 35.3 to 35.8 (100 best-health) and 50.5 to 45.8 (0 best-health) respectively. NPRS (mean (SD)) ranged from 5.0 (2.6) to 5.5 (2.5), with median (IQR) ankle haemophilia joint health score of 4.5 (1 to 12.5) to 6.0 (3.0 to 10.0) indicating moderate to severe levels of ankle haemarthropathy. Ankle NPRS over six months and inhibitor status were associated with decline in outcome.

CONCLUSIONS

HRQoL and foot and ankle PROMs were poor in participants with moderate to severe levels of ankle haemarthropathy. Pain was a major driver for decline in HRQoL and foot and ankle PROMs and use of NPRS has the potential to predict worsening HRQoL and PROMs at the ankle and other affected joints.

摘要

背景

血友病是一种 X 连锁隐性遗传疾病，其特征是软组织和关节内出血。与肘部和膝盖相比，踝关节更容易受到关节积血的影响；据报道，这是血友病患者中最常受影响的关节。尽管治疗取得了进展，但患者仍报告持续存在疼痛和残疾，然而，这种影响尚未得到评估，也未评估对健康相关生活质量（HRQoL）或足部和踝关节患者报告的结果测量（PROMs）的影响。本研究的主要目的是确定严重和中度血友病 A 和 B 患者踝关节关节积血的影响。其次，确定与 HRQoL 和足部和踝关节 PROM 下降相关的临床结果。

方法

在英格兰、苏格兰和威尔士的 18 个血友病中心进行了一项横断面多中心问卷调查研究，目标招募 245 名参与者。使用 HAEMO-QoL-A 和曼彻斯特-牛津足部问卷（MOXFQ）（足部和踝关节）测量总得分和领域得分，以衡量对 HRQoL 和足部和踝关节结果的影响。收集人口统计学、临床特征、踝关节血友病关节健康评分、多关节关节积血和“过去六个月踝关节疼痛”的数字疼痛评分量表（NPRS），作为慢性踝关节疼痛的衡量标准。

结果

共有 243 名 250 名参与者提供了完整的数据。HAEMO-QoL-A 和 MOXFQ（足部和踝关节）总分和指数评分表明 HRQoL 更差，总分范围从平均 35.3 到 35.8（最佳健康 100）和 50.5 到 45.8（最佳健康 0）。NPRS（平均值（标准差））范围从 5.0（2.6）到 5.5（2.5），中位数（IQR）踝关节血友病关节健康评分 4.5（1 到 12.5）到 6.0（3.0 到 10.0）表明踝关节关节积血处于中度至重度水平。过去六个月的踝关节 NPRS 和抑制剂状态与结果下降有关。