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风湿病学中未满足的需求:2022年靶向治疗进展会议报告

Unmet need in rheumatology: reports from the Advances in Targeted Therapies meeting, 2022.

作者信息

Winthrop Kevin L, Isaacs John D, Mease Philip J, Boumpas Dimitrios T, Baraliakos Xenofon, Gottenberg Jacques-Eric, Siebert Stefan, Mosca Marta, Basu Neil, Orange Dana, Lories R, Aletaha Daniel, McInnes Iain B, Huizinga Tom W J, Voll Reinhard E, Gravallese Ellen M, Breedveld Ferry C, Smolen Josef S

机构信息

Department of Medicine, Oregon Health and Sciences University (OHSU), Portland, Oregon, USA

Department of Clinical Rheumatology, Newcastle University, Newcastle upon Tyne, UK.

出版信息

Ann Rheum Dis. 2023 May;82(5):594-598. doi: 10.1136/ard-2022-223528. Epub 2023 Jan 26.

Abstract

To detail the unmet clinical and scientific needs in the field of rheumatology. After a 2-year hiatus due to the SARS-CoV-2 pandemic, the 22nd 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. Breakout sessions were convened with experts in five rheumatological disease-specific groups including: rheumatoid arthritis (RA), psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and connective tissue diseases (CTDs). In each group, experts were asked to identify and prioritise current unmet needs in clinical and translational research, as well as highlight recent progress in meeting formerly identified unmet needs. Clinical trial design innovation was emphasised across all disease states. Within RA, developing therapies and trials for refractory disease patients remained among the most important identified unmet needs and within lupus and spondyloarthritis the need to account for disease endotypes was highlighted. The RA group also identified the need to better understand the natural history of RA, pre-RA states and the need ultimately for precision medicine. In CTD generally, experts focused on the need to better identify molecular, cellular and clinical signals of early and undifferentiated disease in order to identify novel drug targets. There remains a strong need to develop therapies and therapeutic strategies for those with treatment-refractory disease. Increasingly it is clear that we need to better understand the natural history of these diseases, including their 'predisease' states, and identify molecular signatures, including at a tissue level, which can facilitate disease diagnosis and treatment. As these unmet needs in the field of rheumatic diseases have been identified based on consensus of expert clinicians and scientists in the field, this document may serve individual researchers, institutions and industry to help prioritise their scientific activities.

摘要

详细阐述风湿病领域尚未满足的临床和科学需求。由于新冠疫情,间隔两年之后,第22届年度靶向治疗进展国际会议汇聚了100多位风湿病学、免疫学、流行病学、分子生物学及其他专业领域的顶尖基础科学家和临床研究人员。会议组织了分组讨论,邀请了五个特定风湿病组的专家,包括:类风湿关节炎(RA)、银屑病关节炎、中轴型脊柱关节炎、系统性红斑狼疮和结缔组织病(CTD)。在每个组中,专家们被要求确定临床和转化研究中当前未满足的需求并排出优先级,同时强调在满足先前确定的未满足需求方面取得的最新进展。所有疾病状态下都强调了临床试验设计创新。在类风湿关节炎领域,为难治性疾病患者开发治疗方法和试验仍然是最重要的未满足需求之一,在狼疮和脊柱关节炎领域,强调了考虑疾病内型的必要性。类风湿关节炎组还确定需要更好地了解类风湿关节炎的自然病史、类风湿关节炎前期状态以及最终对精准医学的需求。总体而言,在结缔组织病方面,专家们关注的重点是需要更好地识别早期和未分化疾病的分子、细胞和临床信号,以便确定新的药物靶点。对于那些治疗难治性疾病患者,仍然迫切需要开发治疗方法和治疗策略。越来越明显的是,我们需要更好地了解这些疾病的自然病史,包括它们的“疾病前期”状态,并识别分子特征,包括在组织水平上的特征,这有助于疾病的诊断和治疗。由于风湿病领域的这些未满足需求是基于该领域临床专家和科学家的共识确定的,本文档可为个别研究人员、机构和行业提供帮助,以确定其科学活动的优先级。

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