Autologous hematopoietic stem cell transplantation for refractory antiphospholipid syndrome causing myocardial necrosis

Autologous hematopoietic stem cell transplantation (HSCT) is currently being evaluated as a treatment for autoimmune diseases, including systemic lupus erythematosus (SLE), that are associated with a very severe prognosis. We describe a 27-year-old woman with SLE with a 10-year history of refractory antiphospholipid syndrome (APS). She developed progressive myocardial necrosis despite treatment with corticosteroids, cyclophosphamide (CYC), cyclosporine, and immunopheresis. After conditioning with CYC, fludarabine, and antithymocyte globulin, autologous HSCT using CD34(+) selection was performed. After transplantation, the clinical symptoms caused by APS remitted, and the serum anticardiolipin antibody level decreased. Remission has persisted for 21 months after transplantation. Although a longer follow-up is required for the assessment of efficacy, autologous HSCT may cure patients with refractory APS.