High-dose cyclophosphamide without stem cell rescue for the treatment of refractory Behcet's disease

Behcet's disease (BD) is a systemic inflammatory disorder characterized by recurrent mucocutaneous ulcerations, ocular inflammation, and numerous severe systemic inflammatory manifestations. While most patients respond to standard immunosuppressive therapies, a subset will develop refractory disease. In this report, the use of a novel therapy for the treatment of BD is described.

Two patients with severe refractory BD who were treated with nonmyeloablative high-dose cyclophosphamide therapy without stem cell rescue.

After treatment, both patients were completely weaned off immunosuppressive therapy and entered disease-free remissions of 18 and 24 months, respectively.

These data suggest high-dose cyclophosphamide therapy without stem cell rescue as an alternative for the treatment of refractory BD.