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抗磷脂综合征的靶向治疗

Targeted therapy in antiphospholipid syndrome.

作者信息

Sciascia Savino, Khamashta Munther A, D'Cruz David P

机构信息

aGraham Hughes Lupus Research Laboratory, Lupus Research Unit, Division of Women's Health, The Rayne Institute, King's College, London, UK bCentro di Ricerche di Immunologia Clinica ed Immunopatologia e Documentazione su Malattie Rare (CMID), Università di Torino, Italy cLouise Coote Lupus Unit, Guy's and St Thomas' NHS Foundation Trust, St Thomas' Hospital, London, UK.

出版信息

Curr Opin Rheumatol. 2014 May;26(3):269-75. doi: 10.1097/BOR.0000000000000051.

DOI:10.1097/BOR.0000000000000051
PMID:24646948
Abstract

PURPOSE OF REVIEW

To review novel therapeutic targets that are currently under investigation to develop safer, targeted therapies for antiphsopholipid antibody (aPL)-mediated clinical manifestations.

RECENT FINDINGS

Novel therapeutic options potentially available include anti-CD20 monoclonal antibodies and new-generation anticoagulants (such as direct thrombin and anti-Xa inhibitors). Research focusing on interfering with aPL-mediated cell activation, targeting complement components and the innovative concept of blocking the pathogenic subpopulation of aPL with tailored peptides are currently being explored.

SUMMARY

Antiphospholipid syndrome is an autoimmune disease characterized by thrombosis and pregnancy morbidity occurring in patients persistently positive for aPL. Current therapeutic options remain confined to long-term anticoagulation with vitamin K antagonists. The future holds much promise with the identification of novel potential targets, many of which are currently under investigation. The challenge will be to design prospective randomized controlled clinical trials to provide the evidence necessary to support integration of these therapies into clinical practice.

摘要

综述目的

回顾目前正在研究的新型治疗靶点,以开发更安全的针对抗磷脂抗体(aPL)介导的临床表现的靶向治疗方法。

最新发现

潜在可用的新型治疗选择包括抗CD20单克隆抗体和新一代抗凝剂(如直接凝血酶抑制剂和抗Xa抑制剂)。目前正在探索针对干扰aPL介导的细胞活化、靶向补体成分以及用定制肽阻断aPL致病亚群的创新概念的研究。

总结

抗磷脂综合征是一种自身免疫性疾病,其特征为aPL持续阳性的患者出现血栓形成和妊娠并发症。目前的治疗选择仍局限于使用维生素K拮抗剂进行长期抗凝。随着新型潜在靶点的发现,未来充满希望,其中许多靶点目前正在研究中。挑战将是设计前瞻性随机对照临床试验,以提供必要的证据来支持将这些疗法纳入临床实践。

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