Personalized Medicine for Systemic Sclerosis-Associated Interstitial Lung Disease.

Angela Ma, Sydney B Montesi
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Abstract

Purpose of the review: Systemic sclerosis (SSc) is a rare immune-mediated connective tissue disease with high morbidity and mortality. Interstitial lung disease (ILD) is now the leading cause of death for patients with SSc. While several therapeutic agents have been approved for SSc-ILD, opportunities remain for a personalized medicine approach to improve patient outcomes. The purpose of this narrative review is to summarize the current state of personalized medicine for SSc-ILD and future directions to facilitate earlier diagnosis, disease stratification, prognostication, and determination of treatment response. We also review opportunities for personalized medicine approaches within clinical trial design for SSc-ILD.

Recent findings: The management of SSc-ILD remains challenging due to its variable clinical course and current deficits in predicting which individuals will develop progressive pulmonary fibrosis. There have additionally been many challenges in clinical trial design due to limitations in enrichment strategies. Emerging data suggest that serum, radiologic, and other novel biomarkers could be utilized to assess disease activity and treatment response on an individual level.

Summary: Personalized medicine is emerging as a way to address unmet challenges in SSc-ILD and has applicability for identifying stratifying, prognostic, and therapeutic markers for routine clinical care and clinical trial design.

系统性硬化症相关间质性肺病的个性化治疗。
综述目的:系统性硬化症(SSc)是一种罕见的免疫介导的结缔组织疾病,发病率和死亡率高。间质性肺疾病(ILD)现在是SSc患者死亡的主要原因。虽然几种治疗药物已被批准用于SSc-ILD,但仍有机会采用个性化的药物方法来改善患者的预后。这篇叙述性综述的目的是总结SSc-ILD个体化治疗的现状和未来的发展方向,以促进早期诊断、疾病分层、预后和治疗反应的确定。我们还回顾了在SSc-ILD临床试验设计中个性化医疗方法的机会。近期发现:SSc-ILD的管理仍然具有挑战性,因为其多变的临床病程和目前在预测哪些个体将发展为进行性肺纤维化方面的缺陷。此外,由于富集策略的局限性,临床试验设计也面临许多挑战。新出现的数据表明,血清、放射学和其他新的生物标志物可用于评估个体水平上的疾病活动性和治疗反应。摘要:个性化医疗正在成为解决SSc-ILD尚未解决的挑战的一种方法,并适用于确定常规临床护理和临床试验设计的分层、预后和治疗标志物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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