Immunoablative high-dose cyclophosphamide without stem cell rescue in paraneoplastic pemphigus: Report of a case and review of this new therapy for severe autoimmune disease

Paraneoplastic pemphigus (PNP) is a refractory and life-threatening autoimm une mucocutaneous disease. We have recently reported the effectiveness and safety of ablative intravenous cyclophosphamide (200 mg/kg daily over 4 day s) without stem cell rescue in patients with refractory autoimmune diseases including systemic lupus erythematosus, autoimmune cytopenias, chronic inf lammatory demyelinating polyneuropathy, and aplastic anemia. We report chro nic lymphocytic leukemia-associated PNP in a patient who presented with ext ensive and debilitating painful oral ulcerations and received ablative ther apy. The patient tolerated the regimen well and showed a slow but sustained improvement despite persistence of the underlying neoplasm. Eighteen month s after therapy, the oral ulcerations were almost completely healed and the circulating autoantibodies became negative. Currently, the patient remains on cyclosporine and a low dose of prednisone. This provides further eviden ce for the efficacy and safety of this regimen in the management of severe autoimmune diseases including PNP.