New therapeutic targets for the antiphospholipid syndrome

The antiphospholipid syndrome (APS) is an autoimmune condition whereby pathogenic antiphospholipid antibodies (aPL) cause vascular thrombosis and/or recurrent miscarriage, and carries a high burden of morbidity and mortality. Currently the only proven treatment is long-term anticoagulation, which is not effective in all patients and carries risk of haemorrhage.

Novel therapeutic targets that are currently being explored for APS in order to address the unmet needs of better, safer and ideally targeted therapy. These include B cell depletion, new-generation anticoagulants, interfering with aPL cell-mediated activation of endothelial cells and platelets both at the cell surface level and intracellularly, targeting components of the complement system and the novel concept of using decoy peptides to target only the pathogenic sub-population of aPL.

An overview of the potential targets and rationale underpinning them.

Though current options remain limited for the treatment of APS, the future holds much promise with the identification of multiple targets, many of which are currently being explored. The challenge will be to undertake carefully designed prospective multi-centre trials to generate the evidence necessary to support integration of such candidates into clinical practice.