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Chronic cold agglutinin disease (CAD) is a clonal B-cell lymphoproliferative disease caused by IgM autoantibodies directed against red cells. Rituximab is the standard treatment for patients with symptomatic anemia, but nearly half prove refractory to this agent.
Now, investigators have conducted a prospective, multicenter, open-label, phase II study (GIMEMA) to test whether a short course of intravenous bortezomib (1.3 mg/m2 on days 1, 4, 8, and 11) might be safe and effective for 19 anemic CAD patients refractory to prior treatment.
The primary endpoint of transfusion independence or a hemoglobin increase of >2 g/dL was achieved in six patients (32%); responses were complete in three and partial in three. Response was maintained in four pat…