From the Johns Hopkins University School of Medicine, Baltimore, Maryland; United BioSource Corporation, Bethesda, Maryland; and MedImmune LLC, Gaithersburg, Maryland, USA.
J Rheumatol. 2013 Nov;40(11):1865-74. doi: 10.3899/jrheum.130046. Epub 2013 Oct 1.
Our study evaluated the impaired health status of clinical trial patients with systemic lupus erythematosus (SLE) and explored the relationship between changes in fatigue and pain and their effect on overall health status.
Pooled treatment and placebo data from a phase Ib clinical trial of adults with moderate/severe SLE were analyzed. Measures included patient-reported Medical Outcome Study Short Form-36 Survey, Version 2 (SF-36v2), Fatigue Severity Scale, and numeric rating scales (NRS) for pain and global health assessment and clinician-reported global assessment of disease activity (MDGA). Disease burden was compared to the US general population. Health status of responders and nonresponders on pain or fatigue were compared.
The sample included 161 patients with SLE, predominantly female (96%) and white (72%), with average age of 43 ± 11 years. Mean SF-36v2 component summary scores reflected overall problems with physical [physical component summary (PCS); 35.2 ± 9.7] and mental health (mental component summary; 40.9 ± 12.9). Patients with SLE had worse health status on all SF-36v2 subscales than the US general population and comparable age and sex norms (effect size -0.51 to -2.15). Pain and fatigue responders had greater improvements on SF-36v2 scores (bodily pain, physical functioning, social functioning, PCS), patient global health assessment NRS, and MDGA than nonresponders. There was moderate agreement in responder status, based on global assessments by patients and clinicians (68.1%), with some discrepancy between patients who were MDGA responders but patient assessment nonresponders (27.7%).
Improvements in patient-reported pain or fatigue correlated with improvements in overall health. Patient assessments offer a unique perspective on treatment outcomes. Patient-reported outcomes add value in understanding clinical trial treatment benefits.
我们的研究评估了系统性红斑狼疮（SLE）临床试验患者的健康受损状况，并探讨了疲劳和疼痛变化与其对整体健康状况的影响之间的关系。
对一项成人中度/重度SLE 患者的 Ib 期临床试验的汇总治疗和安慰剂数据进行了分析。评估指标包括患者报告的医疗结局研究 36 项简短健康调查简表（SF-36v2）、疲劳严重程度量表、疼痛和总体健康评估的数字评分量表（NRS）以及临床医生报告的疾病活动整体评估（MDGA）。将疾病负担与美国一般人群进行比较。比较了疼痛或疲劳的应答者和无应答者的健康状况。
该样本包括 161 名 SLE 患者，主要为女性（96%）和白人（72%），平均年龄为 43±11 岁。SF-36v2 成分综合评分均值反映了躯体健康（躯体成分综合评分；35.2±9.7）和心理健康（精神成分综合评分；40.9±12.9）方面的总体问题。与美国一般人群和具有可比性年龄及性别常模相比，SLE 患者在所有 SF-36v2 子量表上的健康状况均更差（效应大小 -0.51 至 -2.15）。疼痛和疲劳应答者在 SF-36v2 评分（躯体疼痛、躯体功能、社会功能、PCS）、患者总体健康评估 NRS 和 MDGA 方面的改善程度大于无应答者。基于患者和临床医生的总体评估，应答者状态具有中度一致性（68.1%），但在 MDGA 应答者但患者评估无应答者之间存在一定差异（27.7%）。
患者报告的疼痛或疲劳改善与整体健康状况改善相关。患者评估为治疗结局提供了独特的视角。患者报告的结局在理解临床试验治疗益处方面具有重要价值。
