Wildflowers abundant in the garden of systemic sclerosis research, while hopeful exotics will one day bloom.
Wildflowers abundant in the garden of systemic sclerosis research, while hopeful exotics will one day bloom.
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系统性硬化症研究的花园里野花盛开，而充满希望的外来植物总有一天会绽放。
DOI：
10.1093/rheumatology/kex420
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发表时间：
2018
期刊：
影响因子：
--
通讯作者：
Saketkoo,LesleyAnn
中科院分区：
文献类型：
--
作者：
Saketkoo,LesleyAnn
Jaeger等人[1]发布了欧洲硬皮病试验和研究(EUSTAR)注册中心的扩展研究得出的许多预期出版物中的第一篇，以破译系统性硬化症的最佳治疗(DeSScipher)。2004年开始使用的EUSTAR具有任何注册记录中可预测的缺陷，它可靠地表征了SSC临床试验的自然历史、预测模型、治疗和对队列充实的影响。EUSTAR的新成功基本上没有资金，它以可行的数据集和强有力的合作努力为基础，以便将新的、小型的和地理位置偏远的中心纳入出版物，并通过广泛的教育、领导和指导机会培养南南合作专门知识。正如欧洲卫星导航卫星系统一直是利用国际专家协作的一个基础性例子一样，在欧洲联盟种子资金的支持下，其DeSScipher扩展计划旨在优化对这种极其复杂、异质性和稀有的危及生命的神秘疾病的调查机会。DeSScipher是一个既是天才又是常识的实验模型；使用适度扩展的数据集，捕获与治疗相关的潜在变化，将符合标准的登记病例分流到一个或多个特定于观察表现的手臂。DeSScipher预测了其他罕见的异质多器官疾病的模型，这些疾病正在与试验设计、招募和治疗有效性作斗争，如结节病。这是DeSScipher的第一篇出版物，确定了SSC相关残疾的预测因素，对SSC相关损害进行了最广泛的检查，样本量是类似研究的150倍。Jaeger等人[1]发现，指部溃疡、疼痛、肌肉无力、胃肠道症状和呼吸困难是SSC相关残疾的最主要驱动因素，令人惊讶的是，这些因素与与生存相关的客观临床措施无关。这一发现本身就是一个至关重要的发现，与SSC研究界高度专注于开发和研究消除死亡的治疗方法形成了鲜明对比。抑制两种最致命的SSC表现-肺纤维化和肺动脉高压-进展的新药稳步扩大SSC的武器库。然而，除非进行干细胞移植，
Jaeger et al.[1] dispatch the first of many anticipated publications resulting from the European Scleroderma Trials and Research (EUSTAR) registry’s extension study, to Decipher the optimal management of Systemic Sclerosis (DeSScipher). EUSTAR, incepted in 2004, with the predictable flaws of any registry, has reliably characterized natural history, prediction modelling, treatment and influences on cohort enrichment for clinical trials in SSc. The novel success of EUSTAR, largely unfunded, pivots upon a feasible data set and strong collaborative drive to include new, small and geographically remote centres in publication and to cultivate SSc expertise through broad educational, leadership and mentorship opportunities. Just as EUSTAR has been a foundational example harnessing international expert collaboration, its DeSScipher extension, with European Union seed funding, was designed to optimize investigative opportunity in a life-threatening enigmatic disease of extreme complexity, heterogeneity and rarity. DeSScipher is an experimental model that is at once genius and common sense; streaming criteria-compliant registry cases into one or multiple observational manifestation-specific arms using a modestly expanded dataset that captures potential treatment-related changes. DeSScipher foretells a model for other rare heterogeneous multi-organ diseases battling with trial design, recruitment and treatment efficacy, such as sarcoidosis.This first DeSScipher publication identifies predictors of SSc-related disability, undertaking the broadest examination of SSc-related impairment and with a sample size 150 times greater than similar investigations. Jaeger et al.[1] found that digital ulcers, pain, muscle weakness, gastrointestinal symptoms and dyspnoea are the most prominent drivers of SSc-related disability and, strikingly, were not correlated with objective clinical measures associated with survival. This finding alone is a crucial revelation contrasting the SSc research community’s heavily focused drive to develop and investigate treatments that eliminate mortality. New medications dampening progression of the two deadliest SSc manifestations, pulmonary fibrosis and pulmonary hypertension, steadily expand the SSc arsenal. However, barring stem cell transplantation,
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影响因子：
5.5
作者：
Jaeger, Veronika K.;Distler, Oliver;Walker, Ulrich A.
通讯作者：
Walker, Ulrich A.
影响因子：
4.1
作者：
Irwin, Kelly E.;Greer, Joseph A.;Pirl, William F.
通讯作者：
Pirl, William F.
影响因子：
45.3
作者：
Giese-Davis, Janine;Collie, Kate;Spiegel, David
通讯作者：
Spiegel, David
影响因子：
5.5
作者：
Saketkoo, Lesley Ann;Escorpizo, Reuben;Distler, Oliver
通讯作者：
Distler, Oliver
影响因子：
12.6
作者：
Kanwal, Fasiha;Gralnek, Ian M.;Spiegel, Brennan M. R.
通讯作者：
Spiegel, Brennan M. R.