Division of Rheumatology, Jewish General Hospital, McGill University, Montreal, QC, Canada.
Jewish General Hospital, Lady Davis Institute for Medical Research, Montreal, QC, Canada.
Arthritis Res Ther. 2020 Jun 5;22(1):132. doi: 10.1186/s13075-020-02220-0.
Outcomes of therapeutic studies in diffuse cutaneous systemic sclerosis (dcSSc) have mainly been measured for specific organs, particularly the skin and lungs. A new composite response index in dcSSc (CRISS) has been developed for clinical trials. The goal of this study was to determine whether, in an observational dcSSc cohort, immunosuppression was associated with global disease improvement measured with the CRISS.
We conducted a retrospective cohort study in a multi-centered SSc registry comparing 47 patients newly exposed to immunosuppression for ≥ 1 year to 254 unexposed patients. Inverse probability of treatment weighting (IPTW) was performed to create comparable exposed and unexposed groups by balancing for age, sex, disease duration, modified Rodnan skin score (mRSS), forced vital capacity, patient and physician global assessments, and Health Assessment Questionnaire score. A CRISS score ≥ 0.6 at 1 year was defined as improvement.
Exposed patients had shorter disease duration (5.5 versus 11.7 years, p < 0.01), more interstitial lung disease (67.4% versus 40.3%, p < 0.01), and worse physician global severity scores (4.2 versus 2.5 points, p < 0.01) compared to unexposed patients. Improvement in CRISS scores was more common in exposed patients after IPTW (odds ratio 1.85, 95% confidence interval 1.11, 3.09). Of the individual CRISS variables, only mean patient global assessment scores were significantly better among exposed than unexposed patients (- 0.4 versus 0 points, p = 0.03) while other variables including mRSS were similar.
Using a composite response measure, immunosuppression was associated with better outcomes at 1 year in a dcSSc cohort. These results provide real-world data that align with clinical trials to support our current use of immunosuppression.
弥漫性皮肤系统性硬化症（dcSSc）治疗研究的结果主要针对特定器官进行测量，特别是皮肤和肺部。已经为临床试验开发了一种新的 dcSSc 综合反应指数（CRISS）。本研究的目的是确定在观察性 dcSSc 队列中，免疫抑制是否与使用 CRISS 测量的整体疾病改善相关。
我们在一个多中心 SSc 登记处进行了一项回顾性队列研究，比较了 47 例新接受免疫抑制治疗≥1 年的患者和 254 例未接受免疫抑制治疗的患者。通过平衡年龄、性别、疾病持续时间、改良 Rodnan 皮肤评分（mRSS）、用力肺活量、患者和医生的总体评估以及健康评估问卷评分，使用逆概率治疗加权（IPTW）创建可比的暴露组和未暴露组。1 年时 CRISS 评分≥0.6 定义为改善。
暴露组患者的疾病持续时间更短（5.5 年 vs 11.7 年，p<0.01），间质性肺病更多（67.4% vs 40.3%，p<0.01），医生总体严重程度评分更高（4.2 分 vs 2.5 分，p<0.01）。与未暴露组相比，经过 IPTW 后，暴露组 CRISS 评分改善更为常见（比值比 1.85，95%置信区间 1.11-3.09）。在 CRISS 变量中，只有患者的平均总体评估评分在暴露组中显著优于未暴露组（-0.4 分 vs 0 分，p=0.03），而其他变量包括 mRSS 相似。
使用综合反应测量，免疫抑制与 dcSSc 队列中 1 年时的更好结果相关。这些结果提供了与临床试验一致的真实世界数据，支持我们目前对免疫抑制的使用。
