确定系统性硬化症(SSc)中功能性残疾的轨迹和临床关联。
澳大利亚硬皮病队列研究(ASCS)参与者符合SSc的ACR/EULAR标准,在疾病发作后5年内招募,纳入≥2分的健康评估问卷-残疾指数(HAQ-DI)。基于组的轨迹建模(GBTM)用于识别HAQ-DI轨迹的数量和形状。组间比较采用卡方检验,适当时采用双样本t检验或Wilcoxon秩和检验。使用多元逻辑回归来识别与轨迹组成员身份相关的特征。使用KaplanMeier和Cox比例风险模型进行生存分析。
我们在426名患有SSc的ASCS参与者中确定了两个HAQ-DI轨迹组:低稳定性残疾(n=221,52%),和高度增加的残疾(n=205,48%)。高残疾的参与者在疾病发作时年龄较大,更有可能有弥漫性SSc(DCSSc),心肺疾病,多浊度,数字溃疡,和胃肠道受累(所有p≤0.01),使用免疫抑制(p<0.01)。多症与高增加的轨迹组成员相关(OR3.1,95CI1.1-8.8,p=0.04);独立地,多个SSc特征也强烈相关,包括dcSSc(OR2.3,95CI1.3-4.2,p<0.01),近端无力(OR7.3,95CI2.0-27.1,p<0.01)和关节挛缩(OR2.7,95CI1.3-5.3,p<0.01)。身体残疾增加与死亡风险增加几乎两倍相关(HR1.9,95CI1.0-3.8,p=0.05),和更高的症状负担。
确定了SSc功能障碍的两个轨迹。与具有低稳定性功能残疾的人相比,具有高度增加的功能残疾的人具有明显的临床表型和更差的生存率。这些数据突出了SSc中身体残疾的普遍性,及其预后的重要性。
To identify the trajectories and clinical associations of functional disability in systemic sclerosis (SSc).
Australian Scleroderma Cohort Study (ASCS) participants meeting ACR/EULAR criteria for SSc recruited within 5 years of disease onset, with ≥2 Health Assessment Questionnaire-Disability Index (HAQ-DI) scores were included. Group based trajectory modelling (GBTM) was used to identify the number and shape of HAQ-DI trajectories. Between group comparisons were made using the chi-squared test, two-sample t-test or Wilcoxon rank-sum test as appropriate. Multiple logistic regression was used to identify features associated with trajectory group membership. Survival analyses were performed using Kaplan Meier and Cox proportional hazard modelling.
We identified two HAQ-DI trajectory groups within 426 ASCS participants with incident SSc: low-stable disability (n=221, 52%), and high-increasing disability (n=205, 48%). Participants with high-increasing disability were older at disease onset, more likely to have diffuse SSc (dcSSc), cardiopulmonary disease, multimorbidity, digital ulcers, and gastrointestinal involvement (all p≤0.01), as was use of immunosuppression (p<0.01). Multimorbidity was associated with high-increasing trajectory group membership (OR3.1, 95%CI1.1-8.8, p=0.04); independently, multiple SSc features were also strongly associated including dcSSc (OR2.3, 95%CI1.3-4.2, p<0.01), proximal weakness (OR7.3, 95%CI2.0-27.1, p<0.01) and joint contractures (OR2.7, 95%CI1.3-5.3, p<0.01). High-increasing physical disability was associated with an almost two-fold increased risk of mortality (HR1.9, 95%CI1.0-3.8, p=0.05), and higher symptom burden.
Two trajectories of functional disability in SSc were identified. Those with high-increasing functional disability had a distinct clinical phenotype and worse survival compared to those with low-stable functional disability. These data highlight the pervasive nature of physical disability in SSc, and its prognostic importance.