Department of Women's and Children's Health, Institute of Life Course and Medical Sciences, University of Liverpool, Liverpool, UK.
Royal Liverpool and Broadgreeen University Hospital Trusts, Liverpool, UK.
Pediatr Rheumatol Online J. 2023 Aug 14;21(1):85. doi: 10.1186/s12969-023-00872-1.
IgA vasculitis (IgAV) is a small vessel vasculitis that is more common in childhood. Very limited evidence exists on patients who experience an atypical disease course. The aim of this study was to describe a cohort of children diagnosed with recurrent or persisting IgAV to identify any themes associated with their disease course and areas of unmet needs.
A single centre retrospective study of children diagnosed with recurrent or persisting IgAV at Alder Hey Children's Hospital (Liverpool, UK). Clinical data, including features at presentation and during follow up, potential triggers, abnormal laboratory and histology results, treatment and outcome at last clinical review were retrospectively collected. Key themes were identified.
A total of 13 children met the inclusion criteria (recurrent disease, n = 4; persisting disease, n = 9). Median age at first presentation was 10.2 years [2.6-15.5], female:male ratio 1.2:1. Children in the atypical cohort were significantly older than a larger cohort of children who followed a non-complicated disease course (median age 5.5 years (range [0.6-16.7], p = 0.003)). All children re-presented with a purpuric rash (either recurring or persisting), accompanied by joint involvement in 92% of patients (12/13). Disease-modifying anti-rheumatic drugs (DMARDs) were used in 8/13 (62%) children. The median time from first presentation to diagnosis of atypical disease was 18.4 months [5.3-150.8] and the time from first presentation to treatment was 24.1 months [1.8-95.4]. Use of corticosteroids was significantly higher in children with renal involvement (p = 0.026). During follow up, 8/13 (62%) children were admitted at least once, whilst 10/13 (77%) had re-presented at least once to the emergency department. Five (38%) children were referred to psychology services and 7 (54%) children reported feelings of frustration.
This series describes some characteristics of a small cohort of children with atypical IgAV. It also identifies unmet needs in children with atypical IgAV, which includes delays in diagnosis and lengthy waits for treatment, lack of high-quality evidence regarding treatment choices and a high unrecognised disease burden. Further research is needed to study this subgroup of children as evidence is lacking.
IgA 血管炎（IgAV）是一种小血管血管炎，在儿童中更为常见。关于经历非典型病程的患者，仅有非常有限的证据。本研究的目的是描述一组被诊断为复发性或持续性 IgAV 的儿童，以确定与疾病过程相关的任何主题和未满足的需求领域。
这是一项在英国利物浦阿尔德海儿童医院（Alder Hey Children's Hospital）进行的回顾性单中心研究，纳入了被诊断为复发性或持续性 IgAV 的儿童。回顾性收集了临床数据，包括就诊时和随访期间的特征、潜在诱因、异常实验室和组织学结果、最后临床复查时的治疗和结局。确定了关键主题。
共有 13 名儿童符合纳入标准（复发性疾病，n=4；持续性疾病，n=9）。首次就诊时的中位年龄为 10.2 岁[2.6-15.5]，男女比例为 1.2:1。非典型组的儿童明显比遵循非复杂疾病病程的更大一组儿童年龄更大（中位年龄为 5.5 岁[0.6-16.7]，p=0.003）。所有儿童均以紫癜性皮疹（复发性或持续性）再次就诊，92%的患者伴有关节受累（13 例中有 12 例）。8/13（62%）名儿童使用了疾病修饰抗风湿药物（DMARDs）。从首次就诊到诊断为非典型疾病的中位时间为 18.4 个月[5.3-150.8]，从首次就诊到开始治疗的时间为 24.1 个月[1.8-95.4]。有肾脏受累的儿童使用皮质类固醇的比例显著更高（p=0.026）。在随访期间，13 名儿童中有 8 名（62%）至少住院一次，13 名中有 10 名（77%）至少因急症就诊一次。5 名（38%）儿童被转介至心理科，7 名（54%）儿童报告有挫折感。
本系列描述了一组非典型 IgAV 儿童的一些特征。它还确定了非典型 IgAV 儿童的未满足需求，包括诊断延迟和治疗等待时间长、缺乏高质量的治疗选择证据以及高未被识别的疾病负担。需要进一步研究这一组儿童，因为缺乏证据。
