Health Assessment Questionnaire-Disability Index (HAQ-DI) use in modelling disease progression in diffuse cutaneous systemic sclerosis: an analysis from the EUSTAR database.

Health Assessment Questionnaire-Disability Index (HAQ-DI) use in modelling disease progression in diffuse cutaneous systemic sclerosis: an analysis from the EUSTAR database.
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DOI:
10.1186/s13075-020-02329-2
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发表时间:
2020-10-28
影响因子:
4.9
通讯作者:
EUSTAR Collaborators
EUSTAR Collaborators
中科院分区:
医学2区
文献类型:
--
作者:
Allanore Y;Bozzi S;Terlinden A;Huscher D;Amand C;Soubrane C;Siegert E;Czirják L;Carreira PE;Hachulla E;Zanatta E;Li M;Airò P;Mendoza FA;Rosato E;Distler O;EUSTAR Collaborators

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弥漫性皮肤系统性硬化症(dcSSc)患者预后不良。监测功能和残疾的主观指标(如健康评估量表-残疾指数(HAQ-DI))非常重要,因为患者认为dcSSc的生活质量损害比糖尿病或血液透析更严重。进行欧洲硬皮病试验和研究(EUSTAR)数据库分析,以检查功能受损在dcSSc预后中的重要性。主要目的是确定1年内死亡和HAQ-DI评分进展的预测因素。HAQ-DI评分、主要晚期器官受累和死亡率也用于开发预测终生dcSSc进展的综合模型。这是一项在EUSTAR登记的dcSSc患者中进行的观察性纵向研究。分别通过与基线协变量相关的考克斯回归和线性回归分析对死亡和HAQ-DI评分进行分析。开发了一种微观模拟马尔可夫模型来估计/预测dcSSc在患者一生中的自然进展。该分析包括来自EUSTAR数据库的具有(N = 690)和不具有(N = 4132)HAQ-DI评分评估的dcSSc患者。在多变量分析中,基线HAQ-DI评分、皮质类固醇治疗和主要晚期器官受累预测死亡;基线HAQ-DI评分增加1分,死亡风险增加2.7倍(p < 0.001),多个晚期主要器官受累增加2.8倍(p < 0.05)。多变量分析显示,基线改良Rodnan皮肤评分(mRSS)和基线HAQ-DI评分与1年时HAQ-DI评分进展相关(p < 0.05),但基线器官受累与1年时HAQ-DI评分进展无关。HAQ-DI评分、主要晚期器官受累和死亡成功用于模拟dcSSc的长期疾病进展。HAQ-DI评分和主要晚期器官受累是dcSSc患者死亡风险的可比预测因素。基线mRSS和基线HAQ-DI评分可预测1年时HAQ-DI评分进展,表明这些终点在监测疾病进展方面具有相关性。希望EUSTAR分析可以改变医生对HAQ-DI评分在dcSSc中的重要性的看法。
Patients with diffuse cutaneous systemic sclerosis (dcSSc) have a poor prognosis. The importance of monitoring subjective measures of functioning and disability, such as the Health Assessment Questionnaire-Disability Index (HAQ-DI), is important as dcSSc is rated by patients as worse than diabetes or hemodialysis for quality of life impairment. This European Scleroderma Trials and Research (EUSTAR) database analysis was undertaken to examine the importance of impaired functionality in dcSSc prognosis. The primary objectives were to identify predictors of death and HAQ-DI score progression over 1 year. HAQ-DI score, major advanced organ involvement, and death rate were also used to develop a comprehensive model to predict lifetime dcSSc progression. This was an observational, longitudinal study in patients with dcSSc registered in EUSTAR. Death and HAQ-DI scores were, respectively, analyzed by Cox regression and linear regression analyses in relation to baseline covariates. A microsimulation Markov model was developed to estimate/predict natural progression of dcSSc over a patient’s lifetime. The analysis included dcSSc patients with (N = 690) and without (N = 4132) HAQ-DI score assessments from the EUSTAR database. Baseline HAQ-DI score, corticosteroid treatment, and major advanced organ involvement were predictive of death on multivariable analysis; a 1-point increase in baseline HAQ-DI score multiplied the risk of death by 2.7 (p <  0.001) and multiple advanced major organ involvement multiplied the risk of death by 2.8 (p <  0.05). Multivariable analysis showed that baseline modified Rodnan Skin Score (mRSS) and baseline HAQ-DI score were associated with HAQ-DI score progression at 1 year (p <  0.05), but there was no association between baseline organ involvement and HAQ-DI score progression at 1 year. HAQ-DI score, major advanced organ involvement, and death were successfully used to model long-term disease progression in dcSSc. HAQ-DI score and major advanced organ involvement were comparable predictors of mortality risk in dcSSc. Baseline mRSS and baseline HAQ-DI score were predictive of HAQ-DI score progression at 1 year, indicating a correlation between these endpoints in monitoring disease progression. It is hoped that this EUSTAR analysis may change physician perception about the importance of the HAQ-DI score in dcSSc.
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