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风湿病领域未被满足的需求:2016年靶向治疗会议报告

The unmet need in rheumatology: reports from the Targeted Therapies meeting 2016.

作者信息

Winthrop Kevin L, Strand Vibeke, van der Heijde Désiréé M, Mease Philip J, Crow Mary K, Weinblatt Michael, Bathon Joan M, Buch Maya H, Burmester Gerd R, Dougados Maxime, Kay Jonathan, Mariette Xavier, Breedveld Ferry C, Kalden Joachim R, Smolen Josef S, Furst Daniel E

机构信息

Oregon Health and Science University, Portland, OR, USA.

Stanford University School of Medicine, Portola Valley, CA, USA.

出版信息

Clin Exp Rheumatol. 2016 Jul-Aug;34(4 Suppl 98):69-76. Epub 2016 Aug 3.

Abstract

The 18th annual international Targeted Therapies meeting brought together over 100 leading scientists and clinicians from around the world in the field of rheumatology. During the meeting, breakout sessions were held consisting of 5 disease-specific groups each with 20-40 experts assigned to each group based on clinical or scientific expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis/spondyloarthritis, systemic lupus erythematous, and other connective tissue diseases (e.g. Sjögren's, Behçet's, others). In each group, experts were asked to identify unmet needs in 3 categorical areas: basic/translational science, clinical science and therapeutic development, and clinical care. Needs were prioritised as primary or secondary. Overall, similar primary unmet needs were identified within each disease foci. Within translational science, these included the need for better understanding the heterogeneity within each disease, such that predictive tools for therapeutic response could be developed. Within clinical science and therapeutic trials, the ability to prevent progression to disease onset in those at risk, and the ability to cure disease were identified. A further unmet need was to develop new and accessible therapeutics, as well as to conduct strategic trials of currently approved therapies. Within the clinical care realm, improved co-morbidity management and patient-centered care were identified as unmet needs. Lastly, it was strongly felt there was a need to develop a scientific infrastructure for well-characterised, longitudinal cohorts married with biobanks and mechanisms to support data-sharing. This infrastructure could facilitate many of the unmet needs identified within each disease area.

摘要

第18届年度国际靶向治疗会议汇聚了全球100多位风湿病领域的顶尖科学家和临床医生。会议期间,举行了分组会议,由5个疾病特定小组组成,根据临床或科学专业知识,每个小组分配了20至40名专家。具体小组包括:类风湿关节炎、银屑病关节炎、强直性脊柱炎/脊柱关节炎、系统性红斑狼疮以及其他结缔组织疾病(如干燥综合征、白塞病等)。在每个小组中,专家们被要求确定三个分类领域中未满足的需求:基础/转化科学、临床科学与治疗开发以及临床护理。需求被优先分为主要或次要。总体而言,在每个疾病焦点中都确定了类似的主要未满足需求。在转化科学领域,这些需求包括需要更好地了解每种疾病内部的异质性,以便能够开发治疗反应的预测工具。在临床科学和治疗试验领域,确定了预防高危人群疾病进展至发病的能力以及治愈疾病的能力。另一个未满足的需求是开发新的、可及的治疗方法,以及对目前已批准的治疗方法进行战略试验。在临床护理领域,改善合并症管理和以患者为中心的护理被确定为未满足的需求。最后,与会者强烈认为,有必要为特征明确的纵向队列研究建立科学基础设施,并结合生物样本库和支持数据共享的机制。这种基础设施可以促进每个疾病领域中确定的许多未满足需求的实现。

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