患者可接受的症状状态和最小临床重要差异的患者报告结局在系统性硬化症:一项比较个体化物理治疗与常规护理的随机对照试验的二次分析。

Patient acceptable symptom state and minimal clinically important difference for patient-reported outcomes in systemic sclerosis: A secondary analysis of a randomized controlled trial comparing personalized physical therapy to usual care.

机构信息

AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France; Sorbonne Paris Cité, Université Paris Descartes, Faculté de Médecine, Paris, France; INSERM UMR 1124, Laboratoire de Pharmacologie, Toxicologie et Signalisation Cellulaire, Faculté des Sciences Fondamentales et Biomédicales, Centre Universitaire des Saints-Pères, Paris, France.

出版信息

Semin Arthritis Rheum. 2019 Feb;48(4):694-700. doi: 10.1016/j.semarthrit.2018.03.013. Epub 2018 Mar 27.

DOI:10.1016/j.semarthrit.2018.03.013

PMID:29685482

Abstract

BACKGROUND

To estimate patient acceptable symptom state (PASS) and minimal clinically important difference (MCID) for patient-reported outcomes in systemic sclerosis (SSc).

METHODS

We conducted a secondary analysis of the SCLEREDUC trial, a 12-month randomized controlled trial comparing the efficacy of physical therapy to usual care in 220 SSc patients followed-up from September 2005 to October 2010. Self-rated state and change in patient health at 12 months were assessed by using 2 external anchors extracted from the Medical Outcomes Study 36-Item Short-Form. Patients who self-rated their health as "excellent", "very good" or "good" were the PASS group and those who self-rated their health change as "somewhat better" were the MCID group. Main outcomes were the estimates of PASS by using the 75th percentile method and of MCID by using the mean change in scores method for pain and activity limitation.

RESULTS

PASS (95% confidence interval) and mean (SD) MCID estimates at 12 months were 53.75 (34.00 to 68.00) and -6.74 (32.02) for the joint-pain visual analog scale (range 0-100), 1.41 (1.13 to 1.63) and -0.21 (0.48) for the Health Assessment Questionnaire (HAQ, range 0-3), 1.27 (1.07 to 1.62) and -0.13 (0.45) for the scleroderma HAQ (range 0-3), 26.00 (17.00 to 37.00) and -3.38 (9.87) for the Cochin Hand Function Scale (range 0-90), and 19.40 (17.20 to 21.90) and -5.69 (6.79) for the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (range 0-30), respectively.

CONCLUSIONS

We provide, for the first time, the PASS and MCID estimates for pain and activity limitation in SSc.

TRIAL REGISTRATION

ClinicalTrials.gov Identifier: NCT00318188. First Posted: April 26, 2006.

摘要

背景

评估系统性硬化症（SSc）患者报告结局的可接受症状状态（PASS）和最小临床重要差异（MCID）。

方法

我们对 SCLEREDUC 试验进行了二次分析，这是一项为期 12 个月的随机对照试验，比较了物理治疗与常规护理对 220 例 SSc 患者的疗效，这些患者于 2005 年 9 月至 2010 年 10 月接受随访。使用从医疗结局研究 36 项短式健康调查中提取的 2 个外部锚点，评估患者在 12 个月时的自我报告状态和健康变化。自我报告健康状况为“优秀”、“非常好”或“好”的患者为 PASS 组，自我报告健康变化为“稍有改善”的患者为 MCID 组。主要结局是使用第 75 百分位数法估计 PASS，使用评分变化均值法估计疼痛和活动受限的 MCID。

结果

12 个月时 PASS（95%置信区间）和平均（SD）MCID 估计值分别为关节疼痛视觉模拟量表（范围 0-100）的 53.75（34.00-68.00）和-6.74（32.02）、健康评估问卷（HAQ，范围 0-3）的 1.41（1.13-1.63）和-0.21（0.48）、硬皮病 HAQ（范围 0-3）的 1.27（1.07-1.62）和-0.13（0.45）、Cochin 手功能量表（范围 0-90）的 26.00（17.00-37.00）和-3.38（9.87）以及麦克马斯特-多伦多关节炎患者偏好残疾问卷（范围 0-30）的 19.40（17.20-21.90）和-5.69（6.79）。