Eculizumab in refractory catastrophic antiphospholipid syndrome: a case report and systematic review of the literature

Catastrophic antiphospholipid syndrome (CAPS) is a rare disorder, characterized by the development of multiple vascular thrombosis over a short period of time, in patients with persistently detectable antiphospholipid antibodies (aPLs). The vascular occlusions predominantly affect small vessels. The overall mortality is 36.9%, despite the recent progress in the therapeutic approach. It has been shown that aPLs are able to induce a hypercoagulability state through different mechanisms of action, including complement activation, which in turn plays a key role in the pathogenesis of some thrombotic microangiopathies. Consequently, complement inhibition may be proposed as a targeted intervention to effectively prevent the progression of the microthrombotic storm. The employment of the complement inhibitor eculizumab has been proposed in CAPS on the basis of occasional reports and expert opinion. We report the case of a 54-year-old woman with a CAPS refractory to conventional therapies, who was successfully treated with eculizumab. The administration of this anti-C5 monoclonal antibody aborted the acute progressive thrombotic events and prevented further clinical episodes of thrombosis in the following year. We also faced our case to a systematic literature review, by analyzing all reported cases of CAPS in which eculizumab was added to conventional therapy. Even if further investigation is needed, our results suggest that the inhibition of one mechanism of aPL-induced organ damage may be an add-on treatment for this condition.