Targeted therapy in antiphospholipid syndrome

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

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.

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.