Successful Treatment of Catastrophic Antiphospholipid Syndrome with Mycophenolate Mofetil in a Teenager Without Underlying Disease

Catastrophic antiphospholipid syndrome (CAPS) is a severe form of the antiphospholipid syndrome, a rare autoimmune disease characterized by the presence of antiphospholipid antibodies (APAs) directed against endothelial and plasma proteins in association with recurrent venous and/or arterial thromboembolic events and/or foetal loss. CAPS is marked by small vessel thrombosis, multiorgan failure and a mortality rate of up to 50%. The currently preferred treatment regimen for CAPS includes: anticoagulants, glucocorticosteroids, plasmapheresis with or without intravenous immunoglobulins. Our case of a paediatric CAPS patient is unusual for two reasons: 1) the patient did not suffer from SLE or any other risk factor correlated with increased possibility of thrombosis or CAPS and 2) it is probably the first published case demonstrating successful a remission of CAPS with the immunosuppressant mycophenolate mofetil (MMF) and anti-coagulants. Thus, our case highlights the need for further clinical trials of the effectiveness of new immunosuppressants in the treatment of CAPS and APS.