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自身免疫性肝炎未来试验中未满足的需求和新模型。

Unmet needs and new models for future trials in autoimmune hepatitis.

机构信息

Institute of Cellular Medicine, Newcastle University, Newcastle, UK.

Department of Gastroenterology, Hepatology, and Endocrinology, Hannover Medical School, Hannover, Germany.

出版信息

Lancet Gastroenterol Hepatol. 2018 May;3(5):363-370. doi: 10.1016/S2468-1253(18)30043-8. Epub 2018 Apr 6.

Abstract

Despite advances in understanding and treatment of autoimmune hepatitis, important unmet clinical needs remain in both adult and paediatric patient populations. The evolving research landscape and assembly of large patient cohorts are creating unique opportunities to translate science into new therapies and care pathways, with the potential to substantially improve the lives of patients with autoimmune hepatitis. However, these areas of unmet need represent real challenges that need to be addressed if this vision is to be realised. In this Viewpoint, we outline the challenges in adult autoimmune hepatitis, particularly in relation to disease-modifying therapy, and trial design and delivery. Paediatric autoimmune hepatitis presents its own set of challenging problems.

摘要

尽管人们在自身免疫性肝炎的理解和治疗方面取得了进展,但在成年和儿科患者群体中仍存在重要的未满足的临床需求。不断发展的研究领域和大量患者队列的组建为将科学转化为新的治疗方法和护理途径创造了独特的机会,有可能极大地改善自身免疫性肝炎患者的生活。然而,如果要实现这一愿景,这些未满足的需求领域确实代表了需要解决的真正挑战。在本观点中,我们概述了成人自身免疫性肝炎的挑战,特别是在疾病修饰疗法和试验设计和实施方面。儿科自身免疫性肝炎也提出了一系列具有挑战性的问题。

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