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风湿学未满足的需求:2019 年靶向治疗会议报告。

Unmet need in rheumatology: reports from the Targeted Therapies meeting 2019.

机构信息

Oregon Health Sciences University, Portland, Oregon, USA

Brigham and Womens Hospital, Boston, Massachusetts, USA.

出版信息

Ann Rheum Dis. 2020 Jan;79(1):88-93. doi: 10.1136/annrheumdis-2019-216151. Epub 2019 Oct 29.

Abstract

OBJECTIVES

To detail the greatest areas of unmet scientific and clinical needs in rheumatology.

METHODS

The 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20-30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area.

RESULTS

Overarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases.

CONCLUSIONS

Unmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.

摘要

目的

详细说明风湿病学中最大的未满足科学和临床需求领域。

方法

第 21 届年度国际靶向治疗进展会议汇集了 100 多位领先的风湿病学、免疫学、流行病学、分子生物学等专业的基础科学家和临床研究人员。会议期间,召开了分组会议,由 5 个特定疾病组组成,每组有 20-30 名专家根据专业知识分配到每个组。具体分组包括:类风湿关节炎、银屑病关节炎、中轴型脊柱关节炎、系统性红斑狼疮和其他系统性自身免疫性风湿病。在每个小组中,专家们被要求确定一般未满足的临床和转化研究需求,然后对每个疾病领域内最重要的具体需求进行优先排序和详细说明。

结果

所有疾病状态的总体主题包括需要创新临床试验设计,重点研究难治性疾病患者,开发考虑疾病内型和重叠炎症性疾病患者的试验,需要更好地了解世界发展中地区炎症性疾病的患病率和发病率,最终开发出能够治愈炎症性自身免疫性疾病的疗法。

结论

风湿病学中仍迫切需要新疗法和试验设计,特别是针对那些治疗难治性疾病的患者。

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