Skin involvement in early diffuse cutaneous systemic sclerosis: an unmet clinical need.
Skin involvement in early diffuse cutaneous systemic sclerosis: an unmet clinical need.
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皮肤参与早期弥漫性皮肤硬化症：未满足的临床需求。
DOI：
10.1038/s41584-022-00765-9
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发表时间：
2022-05
期刊：
影响因子：
--
通讯作者：
Denton CP
中科院分区：
文献类型：
--
作者：
Herrick AL;Assassi S;Denton CP
弥漫性皮肤系统性硬化症（dcSSc）与早期内脏受累导致的高死亡率相关。因此，临床医生往往侧重于早期诊断和治疗可能危及生命的心肺和肾脏疾病。然而，dcSSc的特征性症状是迅速进行性疼痛、瘙痒、皮肤紧绷，这对患者的生活质量影响最大，目前还没有有效的疾病改善治疗方法。在预测皮肤病进展的程度和速度（患者之间存在差异）方面已经取得了相当大的进展，包括皮肤活检样本的分子分析等技术的发展。由于造血干细胞移植是一种治疗选择，进行性皮肤病的风险分层尤其重要，因为分层将为每位患者提供风险与获益的平衡。皮肤病的测量是一个重大挑战。临床试验的结果突出了改良Rodnan皮肤评分（目前的金标准）的局限性。已经并正在开发替代的患者报告和其他潜在的结局指标。早期dcSSc患者应转诊至专科中心，以确保最佳实践管理，包括皮肤病管理，并最大限度地增加纳入临床试验的机会。弥漫性皮肤系统性硬化症患者的生活质量受到进行性疼痛和皮肤瘙痒的严重影响。在这里，Herrick，Assassi和丹顿描述了皮肤受累，进展预测，结果测量，成像技术和最佳实践管理。早期弥漫性皮肤系统性硬化症（dcSSc）的大部分疼痛和残疾是由皮肤增厚（硬皮病）引起的，其可以快速进行，从远端开始，然后向近端延伸。现在可以通过考虑疾病持续时间，皮肤病的程度，自身抗体状态和（潜在的）皮肤活检标本的基因表达谱来确定皮肤病方面的“进展者”。预测进行性皮肤病的能力的提高将为选择造血干细胞移植患者提供信息，以及更有针对性地将患者纳入临床试验。改良Rodnan皮肤评分的局限性是刺激皮肤疾病的其他结局指标的发展，包括患者报告的结局指标、非侵入性成像方法和综合评分。早期dcSSc的最佳实践管理包括早期转诊到专科中心，疼痛管理，多学科输入，免疫抑制治疗，并在可能的情况下纳入临床试验。
Diffuse cutaneous systemic sclerosis (dcSSc) is associated with high mortality resulting from early internal-organ involvement. Clinicians therefore tend to focus on early diagnosis and treatment of potentially life-threatening cardiorespiratory and renal disease. However, the rapidly progressive painful, itchy skin tightening that characterizes dcSSc is the symptom that has the greatest effect on patients’ quality of life, and there is currently no effective disease-modifying treatment for it. Considerable advances have been made in predicting the extent and rate of skin-disease progression (which vary between patients), including the development of techniques such as molecular analysis of skin biopsy samples. Risk stratification for progressive skin disease is especially relevant now that haematopoietic stem-cell transplantation is a treatment option, because stratification will inform the balance of risk versus benefit for each patient. Measurement of skin disease is a major challenge. Results from clinical trials have highlighted limitations of the modified Rodnan skin score (the current gold standard). Alternative patient-reported and other potential outcome measures have been and are being developed. Patients with early dcSSc should be referred to specialist centres to ensure best-practice management, including the management of their skin disease, and to maximize opportunities for inclusion in clinical trials. The quality of life of patients with diffuse cutaneous systemic sclerosis is greatly affected by progressive painful and itchy skin tightening. Here, Herrick, Assassi and Denton describe skin involvement, the prediction of progression, outcome measures, imaging techniques and best-practice management. Much of the pain and disability of early diffuse cutaneous systemic sclerosis (dcSSc) results from skin thickening (scleroderma), which can be rapidly progressive, commencing distally then extending proximally. ‘Progressors’ in terms of skin disease can now be identified by considering disease duration, extent of skin disease, autoantibody status and (potentially) gene-expression profiling of skin biopsy specimens. Improvement in the ability to predict progressive skin disease will inform the selection of patients for haematopoietic stem-cell transplantation, as well as more targeted inclusion of patients in clinical trials. Limitations of the modified Rodnan skin score are stimulating development of other outcome measures of skin disease, including patient-reported outcome measures, non-invasive imaging methods and composite scores. Best-practice management of early dcSSc includes early referral to a specialist centre, pain management, multidisciplinary input, immunosuppressive therapy and, when at all possible, inclusion in a clinical trial.
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影响因子：
4.9
作者：
Allanore Y;Bozzi S;Terlinden A;Huscher D;Amand C;Soubrane C;Siegert E;Czirják L;Carreira PE;Hachulla E;Zanatta E;Li M;Airò P;Mendoza FA;Rosato E;Distler O;EUSTAR Collaborators
通讯作者：
EUSTAR Collaborators
影响因子：
4.9
作者：
Campochiaro C;Allanore Y
通讯作者：
Allanore Y
影响因子：
158.5
作者：
Distler, Oliver;Highland, Kristin B.;Maher, Toby M.
通讯作者：
Maher, Toby M.
影响因子：
--
作者：
Amjadi, Sogol;Maranian, Paul;Khanna, Dinesh
通讯作者：
Khanna, Dinesh
DOI：
10.1093/rheumatology/kex504
发表时间：
2018-05-01
期刊：
Rheumatology (Oxford, England)
影响因子：
--
作者：
Distler O;Allanore Y;Denton CP;Matucci-Cerinic M;Pope JE;Hinzmann B;Davies S;de Oliveira Pena J;Khanna D
通讯作者：
Khanna D