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1054 例系统性硬化症患者的临床特征和转归:Reuma.pt/SSc 登记处分析。

Clinical features and outcome of 1054 patients with Systemic Sclerosis: analysis of Reuma.pt/SSc registry.

机构信息

Serviço de Reumatologia, Hospital Garcia de Orta.

Serviço de Reumatologia, Centro Hospitalar Universitário Lisboa Norte; Unidade de Investigação em Reumatologia, Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa.

出版信息

ARP Rheumatol. 2022 Jan-Mar;1(1):21-29.

Abstract

BACKGROUND

Systemic sclerosis (SSc) is a rare connective tissue disorder with heterogeneous manifestations and outcomes. Besides differences in disease characteristics among distinct ethnic groups and geographical regions, several questions regarding the impact of the disease and the effectiveness of treatments remain unanswered. To address these questions, the Rheumatic Diseases Portuguese Register (Reuma.pt) launched a specific protocol for the prospective follow-up of SSc patients.

OBJECTIVES

To describe the baseline characteristics, disease subsets, treatments used and survival of SSc patients registered in Reuma.pt/SSc.

METHODS

Data from adult patients with SSc included in Reuma.pt up to November 2020 were analysed. Demographic features, SSc subsets, fulfilment of classification criteria, main clinical and immunological features, comorbidities, treatments used and survival data were described and compared between diffuse cutaneous (dc) and limited cutaneous (lc) disease subsets. Survival was calculated for patients included in Reuma.pt within the first two years of diagnosis.

RESULTS

In total, 1054 patients were included, 87.5% female, with a mean age at diagnosis of 52.7 +/- 14.8 years. The most common subset was lcSSc (56.3%), followed by dcSSc (17.5%), preclinical SSc (13%), overlap syndrome (9.8%) and SSc sine scleroderma (3.3%). Raynaud's phenomenon (93.4%) and skin thickening (76.9%) were the most frequently observed clinical manifestations. Gastrointestinal (62.8% versus 47.8%), pulmonary (59.5% versus 23%) and cardiac (12.8% versus 6.9%) involvements were significantly more prevalent in dcSSc than lcSSc. Ninety per-cent of patients were Antinuclear antibody positive, 52.5% were Anti-centromere antibody positive and 21% anti-topoisomerase positive, with significant differences between lcSSc and dcSSc. One-third of patients were treated with immunomodulators, 53.6% with vasodilators, 23% with glucocorticoids and 2.3% with biologics. During follow-up, 83 deaths (7.9%) were reported. The overall 1-, 2- and 5-year survivals were 98.0%, 96.8% and 92.6%, respectively, without significant differences between lcSSc and dcSSc.

CONCLUSION

Reuma.pt/SSc data highlights the importance of registries in improving knowledge about rare and complex diseases, such as SSc. Clinical features of Portuguese SSc patients are similar to those of other populations. In recently diagnosed patients, 5-year survival is over 92%. To the best of our knowledge, this is the first study showing that clinical features of Portuguese SSc are similar to those of other cohorts.

摘要

背景

系统性硬化症(SSc)是一种罕见的结缔组织疾病,具有异质性表现和结局。除了不同种族和地理区域之间疾病特征的差异外,关于疾病的影响和治疗效果的几个问题仍未得到解答。为了解决这些问题,风湿疾病葡萄牙登记处(Reuma.pt)为 SSc 患者的前瞻性随访制定了专门的方案。

目的

描述 Reuma.pt/SSc 登记的 SSc 患者的基线特征、疾病亚组、使用的治疗方法和生存情况。

方法

分析截至 2020 年 11 月纳入 Reuma.pt 的成年 SSc 患者的数据。描述和比较弥漫性皮肤(dc)和局限性皮肤(lc)疾病亚组之间的人口统计学特征、SSc 亚组、分类标准的满足情况、主要临床和免疫学特征、合并症、使用的治疗方法和生存数据。计算 Reuma.pt 中诊断后两年内纳入的患者的生存情况。

结果

共纳入 1054 例患者,其中 87.5%为女性,平均诊断年龄为 52.7 +/- 14.8 岁。最常见的亚组是 lcSSc(56.3%),其次是 dcSSc(17.5%)、临床前 SSc(13%)、重叠综合征(9.8%)和 SSc sine scleroderma(3.3%)。雷诺现象(93.4%)和皮肤增厚(76.9%)是最常见的临床表现。dcSSc 比 lcSSc 更常见胃肠道(62.8%比 47.8%)、肺部(59.5%比 23%)和心脏(12.8%比 6.9%)受累。90%的患者抗核抗体阳性,52.5%抗着丝点抗体阳性,21%抗拓扑异构酶阳性,lcSSc 和 dcSSc 之间存在显著差异。三分之一的患者接受免疫调节剂治疗,53.6%接受血管扩张剂治疗,23%接受糖皮质激素治疗,2.3%接受生物制剂治疗。在随访期间,报告了 83 例死亡(7.9%)。总的 1 年、2 年和 5 年生存率分别为 98.0%、96.8%和 92.6%,lcSSc 和 dcSSc 之间无显著差异。

结论

Reuma.pt/SSc 的数据强调了登记处在提高对罕见和复杂疾病(如 SSc)的认识方面的重要性。葡萄牙 SSc 患者的临床特征与其他人群相似。在最近诊断的患者中,5 年生存率超过 92%。据我们所知,这是第一项表明葡萄牙 SSc 患者的临床特征与其他队列相似的研究。

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