Value Evidence & Outcomes, GlaxoSmithKline, Brentford, Middlesex, UK.
Adelphi Real World, Adelphi Group, Macclesfield, Cheshire, UK.
Rheumatology (Oxford). 2021 Apr 6;60(4):1871-1881. doi: 10.1093/rheumatology/keaa508.
To describe how patients with primary SS (pSS) and systemic organ involvement are classified and clustered in routine practice.
This multinational, cross-sectional survey of real-world quantitative data was conducted across Europe and the US. Rheumatologists who treated seven or more adult patients per month with pSS and current/past systemic manifestations undertook a survey before completing a patient record form capturing demographic, clinical and treatment information for their next six eligible patients. Patients with a completed patient record form were invited to complete a patient self-completion questionnaire capturing insights into their disease and treatment. Subgroups were defined by physicians' assessment of disease severity; clusters were derived based on key clinical characteristics using latent class analysis.
Rheumatologists completed 316 physician surveys and 1879 patient record forms; 888 patients completed the patient self-completion questionnaire. pSS severity reflected organ involvement and symptomatology. Latent class analysis produced five clusters distinguished by the organ systems involved and the presence of pain and fatigue symptoms at the time of the survey. A minority of patients [n = 67 (4%)] were categorized with multiple organ involvement and the highest frequency of pain and fatigue. A total of 324 patients (17%) were categorized as 'low burden'. The remaining three clusters exhibited high frequencies of articular involvement but were distinguished by the extent of other organ system involvement.
Cluster analysis using a real-world cohort of patients with pSS and systemic organ involvement highlights the heterogeneous presentation of patients with pSS and confirms the importance of pain and fatigue as well as organ involvement when determining disease burden.
描述原发性干燥综合征（pSS）伴系统器官受累患者在常规实践中的分类和聚类情况。
这是一项在欧洲和美国进行的多国、横断面、真实世界定量数据调查。每月治疗 7 例或以上成人 pSS 患者且有当前/既往系统表现的风湿病医生，在完成包含其下 6 例合格患者人口统计学、临床和治疗信息的患者病历表单前，先进行调查。有完整患者病历表单的患者被邀请完成一份患者自我报告问卷，以了解他们的疾病和治疗情况。亚组由医生评估的疾病严重程度定义；根据关键临床特征使用潜在类别分析得出聚类。
风湿病医生完成了 316 份医生调查和 1879 份患者病历表单；888 名患者完成了患者自我报告问卷。pSS 的严重程度反映了器官受累和症状。潜在类别分析产生了五个聚类，根据参与的器官系统以及调查时疼痛和疲劳症状的存在情况进行区分。少数患者（n=67 [4%]）被归类为多器官受累，疼痛和疲劳的频率最高。共有 324 名患者（17%）被归类为“低负担”。其余三个聚类表现出高频率的关节受累，但通过其他器官系统受累的程度进行区分。
使用伴有系统器官受累的 pSS 患者真实队列进行聚类分析，突出了 pSS 患者表现的异质性，并证实了疼痛和疲劳以及器官受累在确定疾病负担方面的重要性。
