Prospects for Stratified and Precision Medicine in Systemic Sclerosis Treatment

Prospects for Stratified and Precision Medicine in Systemic Sclerosis Treatment
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分层精准医学在系统性硬化症治疗中的前景

DOI:
10.1007/s40674-019-00124-y
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发表时间:
2019
影响因子:
1.2
通讯作者:
Clark K
Clark K
中科院分区:
--
文献类型:
--
作者:
Clark K

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精准医学是源于肿瘤学研究的一个新兴领域,在自身免疫性疾病中发挥着越来越重要的作用。系统性硬化症的临床表现和对治疗的不同反应的异质性,强调了发展患者分层方法的重要性,以确保正确的患者在疾病期间接受最适当的治疗,因为这将对疾病的病程和缓解产生有意义的影响。这篇综述的目的是讨论到目前为止在系统性硬化症患者分层中探索的不同方法。我们强调最近的临床试验已应用分层技术,以提供一种形式的精准医学管理的系统性硬化症。最近的研究进展集中在利用全皮肤活检或成纤维细胞的基因表达技术来了解哪组患者更有可能对哪种治疗产生反应。该技术已成功用于了解tocilizumab, abatacept和fresolimumab对系统性硬化症的影响,并有助于识别那些更可能对治疗有反应的患者。利用高输出平台对患者进行分层进行靶向治疗仍处于起步阶段,但在确保最有可能对特定治疗产生反应的患者进行试验方面具有巨大潜力。它已经被证明在那些具有高IL-6的人身上是成功的,并且很可能在未来被证明是非常有用的。
Purpose of reviewPrecision medicine is an evolving field stemming from Oncology research, with an increasingly important role in autoimmune diseases. The heterogeneity, both of clinical presentations of systemic sclerosis and differing response to treatment, emphasises the importance of developing means of patient stratification to ensure that the correct patients are managed with the most appropriate treatments at a disease duration when this will have meaningful impact on disease course and resolution. This review aims to discuss the different means explored so far in stratifying patients with systemic sclerosis. We highlight recent clinical trials which have applied stratification techniques in order to provide a form of precision medicine in the management of systemic sclerosis.Recent findingsAdvances have focused on utilising gene expression techniques on whole skin biopsies or fibroblasts to understand which groups of patients are more likely to respond to which treatments. This technique has been used successfully to understand the effect of tocilizumab, abatacept, and fresolimumab on systemic sclerosis, and helped identify those that are more likely to respond to treatment.SummaryUtilising high output platforms to stratify patients for targeted treatment is still in its infancy but has huge potential for ensuring the patients most likely to respond to a specific therapy are put forward to trials. It has already been shown to be successful in those with a high IL-6 profile and will most likely prove hugely informative in the future.
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