Sophia Children's Hospital, Erasmus University Medical Center, Rotterdam, The Netherlands, and Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands.
Sophia Children's Hospital, Erasmus University Medical Center, Rotterdam, The Netherlands.
Arthritis Rheumatol. 2019 Feb;71(2):290-301. doi: 10.1002/art.40697.
Childhood-onset systemic lupus erythematosus (SLE) is a severe, lifelong, multisystem autoimmune disease. Long-term outcome data are limited. This study was undertaken to identify clinical characteristics and health-related quality of life (HRQoL) of adults with childhood-onset SLE.
Patients participated in a single study visit comprising a structured history and physical examination. Disease activity (scored using the SLE Disease Activity Index 2000 [SLEDAI-2K]), damage (scored using the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index [SDI]), and HRQoL (scored using the Short Form 36 Health Survey) were assessed. Medical records were reviewed.
In total, 111 childhood-onset SLE patients were included; the median disease duration was 20 years, 91% of patients were female, and 72% were white. Disease activity was low (median SLEDAI-2K score 4), and 71% of patients received prednisone, hydroxychloroquine (HCQ), and/or other disease-modifying antirheumatic drugs. The vast majority of new childhood-onset SLE-related manifestations developed within 2 years of diagnosis. Damage such as myocardial infarctions began occurring after 5 years. Most patients (62%) experienced damage, predominantly in the musculoskeletal, neuropsychiatric, and renal systems. Cerebrovascular accidents, renal transplants, replacement arthroplasties, and myocardial infarctions typically occurred at a young age (median age 20 years, 24 years, 34 years, and 39 years, respectively). Multivariate logistic regression analysis showed that damage accrual was associated with disease duration (odds ratio [OR] 1.15, P < 0.001), antiphospholipid antibody positivity (OR 3.56, P = 0.026), and hypertension (OR 3.21, P = 0.043). Current HCQ monotherapy was associated with an SDI score of 0 (OR 0.16, P = 0.009). In this cohort, HRQoL was impaired compared to the overall Dutch population. The presence of damage reduced HRQoL scores in 1 domain. High disease activity (SLEDAI-2K score ≥8) and changes in physical appearance strongly reduced HRQoL scores (in 4 of 8 domains and 7 of 8 domains, respectively).
The majority of adults with childhood-onset SLE in this large cohort developed significant damage at a young age and had impaired HRQoL without achieving drug-free remission, illustrating the substantial impact of childhood-onset SLE on future life.
儿童发病的系统性红斑狼疮(SLE)是一种严重的、终身的、多系统自身免疫性疾病。长期预后数据有限。本研究旨在确定儿童发病的 SLE 成年患者的临床特征和健康相关生活质量(HRQoL)。
患者参与了一次单中心研究,包括结构化病史和体检。疾病活动(使用 SLE 疾病活动指数 2000 评分[SLEDAI-2K]评分)、损伤(使用系统性红斑狼疮国际合作临床/美国风湿病学会损伤指数[SDI]评分)和 HRQoL(使用简短 36 健康调查评分)进行评估。回顾了病历。
共纳入 111 例儿童发病的 SLE 患者;中位病程 20 年,91%的患者为女性,72%为白人。疾病活动度低(中位 SLEDAI-2K 评分 4),71%的患者接受泼尼松、羟氯喹(HCQ)和/或其他疾病修饰抗风湿药物治疗。绝大多数新的儿童发病的 SLE 相关表现发生在诊断后 2 年内。心肌梗死等损伤在 5 年后才开始发生。大多数患者(62%)出现损伤,主要发生在肌肉骨骼、神经精神和肾脏系统。中风、肾移植、关节置换和心肌梗死通常发生在年轻的时候(中位年龄分别为 20 岁、24 岁、34 岁和 39 岁)。多变量逻辑回归分析显示,损伤累积与疾病持续时间(比值比[OR]1.15,P<0.001)、抗磷脂抗体阳性(OR 3.56,P=0.026)和高血压(OR 3.21,P=0.043)相关。目前的 HCQ 单药治疗与 SDI 评分 0 相关(OR 0.16,P=0.009)。在本队列中,与荷兰一般人群相比,HRQoL 受损。损伤的存在降低了 1 个领域的 HRQoL 评分。高疾病活动度(SLEDAI-2K 评分≥8)和外貌变化强烈降低了 HRQoL 评分(分别在 8 个领域中的 4 个和 8 个领域中的 7 个)。
在这项大型队列研究中,大多数儿童发病的 SLE 成年患者在年轻时就出现了显著的损伤,且 HRQoL 受损,并未达到无药物缓解,表明儿童发病的 SLE 对未来生活有重大影响。