Fairley Jessica L, Hansen Dylan, Proudman Susanna, Sahhar Joanne, Ngian Gene-Siew, Walker Jennifer, Apostolopoulos Diane, Host Lauren V, Stevens Wendy, Nikpour Mandana, Ross Laura
The University of Melbourne, Melbourne, VIC, Australia.
St Vincent's Hospital Melbourne, Melbourne, VIC, Australia.
J Scleroderma Relat Disord. 2025 Aug 19:23971983251360883. doi: 10.1177/23971983251360883.
Functional disability is a major concern for individuals with systemic sclerosis (SSc). The Health Assessment Questionnaire-Disability Index (HAQ-DI) measures the ability to perform activities of daily living, with higher scores indicating poorer function.
To define the frequency and clinical associations of minimum clinically important difference (MCID) change in HAQ-DI scores in SSc.
Australian Scleroderma Cohort Study participants with two or more HAQ-DI scores ⩽ 2 visits apart were included. Generalised estimating equations were used to model the correlates of the MCID improvement (-0.125 points) and MCID worsening (+0.14 points) of HAQ-DI scores. Subgroup analysis in those with incident (⩽5 years SSc duration at recruitment) and diffuse SSc (dcSSc) were performed.
Of 1117 participants, 712 (64%) recorded worsening of HAQ-DI scores. Of 827 participants with baseline HAQ-DI ⩾ 0.125 units, 585 (71%) had recorded improvement. Across 3229 study visits, older age (odds ratio (OR) 1.1, 95% confidence interval (CI) 1.1-1.1, < 0.01), higher skin score (OR 1.1, 95% CI 1.0-1.2, = 0.01), digital ulcers (OR 1.3, 95% CI 1.0-1.5, = 0.02), raised C-reactive protein (CRP; OR 1.3, 95% CI 1.1-1.6, < 0.01) and patient-reported worsening Raynaud's phenomenon (RP; OR 1.2, 95% CI 1.0-1.4, = 0.04) and dyspnoea (OR 1.3, 95% CI 1.1-1.6, < 0.01) were associated with worsening of HAQ-DI scores. In those with incident SSc, raised CRP and patient-reported worsening RP and dyspnoea were associated with worsening of HAQ-DI scores. In dcSSc, only raised CRP was associated with worsening HAQ-DI scores, while in lcSSc higher baseline HAQ-DI score, older age, higher skin score, proximal weakness and worsening dyspnoea were associated with worsening scores. Only higher baseline HAQ-DI score was associated with improvement in HAQ-DI scores in the overall cohort (OR 1.3, 95% CI 1.2-1.5, < 0.01).
Two-thirds of a large SSc cohort demonstrated significant change in physical function. Worsening symptom burden and elevated CRP were important determinants of worsening function.
功能残疾是系统性硬化症（SSc）患者的主要担忧。健康评估问卷残疾指数（HAQ-DI）用于衡量日常生活活动能力，分数越高表明功能越差。
确定SSc患者HAQ-DI评分中最小临床重要差异（MCID）变化的频率及临床相关性。
纳入澳大利亚硬皮病队列研究中两次或更多次HAQ-DI评分间隔≤2次就诊的参与者。采用广义估计方程对HAQ-DI评分的MCID改善（-0.125分）和MCID恶化（+0.14分）的相关因素进行建模。对发病（招募时SSc病程≤5年）和弥漫性SSc（dcSSc）患者进行亚组分析。
1117名参与者中，712名（64%）记录到HAQ-DI评分恶化。在827名基线HAQ-DI≥0.125单位的参与者中，585名（71%）记录到改善。在3229次研究就诊中，年龄较大（优势比（OR）1.1，95%置信区间（CI）1.1 - 1.1，P<0.01）、皮肤评分较高（OR 1.1，95%CI 1.0 - 1.2，P = 0.0l）、指端溃疡（OR 1.3，95%CI 1.0 - 1.5，P = 0.02）、C反应蛋白（CRP）升高（OR 1.3，95%CI 1.1 - 1.6，P<0.01）、患者报告的雷诺现象（RP）恶化（OR 1.2，95%CI 1.0 - 1.4，P = 0.04）和呼吸困难（OR 1.3，95%CI 1.1 - 1.6，P<0.01）与HAQ-DI评分恶化相关。在发病性SSc患者中，CRP升高、患者报告的RP和呼吸困难恶化与HAQ-DI评分恶化相关。在dcSSc中，仅CRP升高与HAQ-DI评分恶化相关，而在局限性皮肤型SSc（lcSSc）中，较高的基线HAQ-DI评分、年龄较大、皮肤评分较高、近端肌无力和呼吸困难恶化与评分恶化相关。在整个队列中，仅较高的基线HAQ-DI评分与HAQ-DI评分改善相关（OR 1.3，95%CI 1.2 - 1.5，P<0.01）。
一大群SSc患者中有三分之二表现出身体功能的显著变化。症状负担加重和CRP升高是功能恶化的重要决定因素。
