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儿科风湿病学中的精准医学。

Precision medicine in pediatric rheumatology.

作者信息

Yeo Joo Guan, Ng Chin Teck, Albani Salvatore

机构信息

aSingHealth Translational Immunology and Inflammation Centre, Singapore Health Services Pte Ltd. bDivision of Medicine, KK Women's and Children's Hospital cDepartment of Rheumatology and Immunology, Singapore General Hospital dDuke-NUS Medical School, Singapore, Singapore.

出版信息

Curr Opin Rheumatol. 2017 Sep;29(5):500-505. doi: 10.1097/BOR.0000000000000419.

Abstract

PURPOSE OF REVIEW

Precision medicine is the tailoring of medical care to subcategories of disease. In pediatric rheumatology, these subcategories must first be defined by their specific molecular immunological profiles, and then the effects of growth and puberty, developmental immunological changes, and differences in treatment options and adherence considered when designing therapeutic strategies. In the present review, we summarize the unmet needs in pediatric rheumatology before such precision medical care can be effectively delivered to affected patients.

RECENT FINDINGS

The current clinical classification of pediatric rheumatic diseases does not provide all the information necessary for prognostication and accurate therapeutic selection. Many studies have highlighted the molecular differences between disease subcategories and the dissimilarities in the molecular manifestations of the same disease between patients. Harnessing such discoveries by collaborating with various research networks and laboratories is required to interrogate the multifactorial nature of rheumatic diseases in a holistic manner.

SUMMARY

Integration of big data sets generated from well defined pediatric cohorts with rheumatic diseases using different high-dimensional technological platforms will help to elucidate the underlying disease mechanisms. Distilling these data will be necessary for accurate disease stratification and will have a positive impact on prognosis and treatment choice.

摘要

综述目的

精准医学是根据疾病亚类来定制医疗护理。在儿科风湿病领域,这些亚类首先必须依据其特定的分子免疫学特征来定义,然后在设计治疗策略时要考虑生长和青春期的影响、发育过程中的免疫学变化以及治疗选择和依从性的差异。在本综述中,我们总结了在能够将这种精准医疗有效提供给受影响患者之前,儿科风湿病领域尚未满足的需求。

最新发现

儿科风湿性疾病目前的临床分类并未提供预后评估和准确治疗选择所需的全部信息。许多研究强调了疾病亚类之间的分子差异以及同一疾病在不同患者之间分子表现的不同。需要通过与各种研究网络和实验室合作来利用这些发现,以便全面探究风湿性疾病的多因素性质。

总结

利用不同的高维技术平台,将来自定义明确的儿科风湿病队列产生的大数据集进行整合,将有助于阐明潜在的疾病机制。提炼这些数据对于准确的疾病分层是必要的,并且将对预后和治疗选择产生积极影响。

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