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比较在两家欧洲三级转诊中心接受治疗的幼年皮肌炎患儿的治疗方法和结局。

Comparison of treatments and outcomes of children with juvenile dermatomyositis followed at two European tertiary care referral centers.

机构信息

Dipartimento di Neuroscienze Riabilitazione Oftalmologia Genetica e Scienze MaternoInfantili, Università degli Studi di Genova, Genoa, Italy.

Department of Paediatric Rheumatology, Royal Manchester Children's Hospital, Manchester, UK.

出版信息

Rheumatology (Oxford). 2021 Nov 3;60(11):5419-5423. doi: 10.1093/rheumatology/keab089.

DOI:10.1093/rheumatology/keab089
PMID:33528490
Abstract

OBJECTIVES

To compare the treatment approaches and disease outcomes of children with JDM followed in two European tertiary care peadiatric rheumatology centres.

METHODS

The medical notes of patients with JDM seen at Istituto Giannina Gaslini (IGG) of Genoa, Italy or Great Ormond Street Hospital (GOSH) of London, UK between January 2000 and December 2015 within 6 months after disease onset and followed for at least 6 months were reviewed. Demographic, clinical and therapeutic data were collected. At each visit, the caring physician was asked to rate the disease state subjectively.

RESULTS

A total of 127 patients were included, 88 at GOSH and 39 at IGG. At 24 months, the median values of muscle strength and disease activity were at the normal end of the scale and around three quarters of patients were said to have inactive disease. Also, at 2 years, 38.6% and 36% of British and Italian patients, respectively, had damage. Cyclophosphamide, azathioprine, infliximab, rituximab and mycophenolate mofetil were used more frequently by UK physicians, whereas ciclosporin, intravenous immunoglobulin and hydroxychloroquine were prescribed by Italian physicians.

CONCLUSION

This study shows a significant difference in the choice of medications between pediatric rheumatologists practising in the two centres. Despite this, a high proportion of patients had inactive disease at 2 years and there was a low frequency of damage: modern treatments have improved outcomes.

摘要

目的

比较在两家欧洲三级儿童风湿病学中心接受治疗的儿童肌炎患者的治疗方法和疾病结局。

方法

回顾了 2000 年 1 月至 2015 年 12 月间在意大利热那亚 Giannina Gaslini 研究所(IGG)或英国伦敦大奥蒙德街医院(GOSH)发病后 6 个月内就诊并至少随访 6 个月的 JDM 患者的病历。收集了人口统计学、临床和治疗数据。每次就诊时,都会询问负责治疗的医生对疾病状况进行主观评估。

结果

共纳入 127 例患者,其中 88 例来自 GOSH,39 例来自 IGG。24 个月时,肌肉力量和疾病活动度的中位数值处于正常范围的末端,约四分之三的患者被认为患有不活跃的疾病。此外,在 2 年时,分别有 38.6%和 36%的英国和意大利患者出现了损害。英国医生更频繁地使用环磷酰胺、硫唑嘌呤、英夫利昔单抗、利妥昔单抗和霉酚酸酯,而意大利医生更常处方环孢素、静脉注射免疫球蛋白和羟氯喹。

结论

本研究表明,在两个中心工作的儿科风湿病医生在药物选择上存在显著差异。尽管如此,仍有相当比例的患者在 2 年内患有不活跃的疾病,且损害的发生率较低:现代治疗方法改善了预后。

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