Bortezomib for Refractory Autoimmune Disease

Bortezomib is a proteasome inhibitor that induces apoptosis in antibody-producing cells. It is approved for the treatment of multiple myeloma but has also shown activity in a variety of autoantibody disorders such as thrombotic thrombocytopenic purpura (TTP), immune thrombocytopenia (ITP), and autoimmune hemolytic anemia (AIHA).

To examine the evidence in support of using bortezomib as salvage therapy for autoimmune disorders, investigators tracked all episodes of off-label use of the drug in Australia state-funded institutions. Bortezomib (1.3 mg/m² on days 1, 4, 8, and 11 of 21-day cycles) was given to 10 patients, of whom 6 had AIHA, 2 had TTP, 1 had ITP, and 1 had acquired hemophilia. All patients were refractory to other agents includin…