Background: Cold agglutinin disease (CAD) is an acquired autoimmune hemolytic anemia mediated by cold-reactive autoantibodies that bind erythrocyte carbohydrate antigens, causing hemagglutination, complement-mediated hemolysis and C3d positive direct antiglobulin test. Conventional therapies for CAD are largely ineffective, but remissions after treatment with the anti-CD20 monoclonal antibody rituximab are increasingly being reported.

Patients and Methods: A total of 16 CAD patients (10 women, 6 men) with a median age of 48 years (range, 20–86 years), were treated in our center between May 2002 and January 2006. CAD was idiopathic in 7 cases and associated with other conditions in 9 (systemic lupus erythematous in 4, chronic lymphoproliferative disorder in 3, and unrelated donor cord blood transplant in 2). Hemoglobin concentration before treatment ranged from 4.9 to 10 g/dL. Median IgM anti-I titer was 512 (range, 128–100,000). Rituximab was given as single agent in doses of 375 mg/m2, at days 1, 8, 15 and 22.

Results: The overall response rate was 62.5%, with 9 patients achieving complete remission (56%). Median duration of response was 24 months (range, 5–48 months). Of the 6 non-responders, 5 died from disease progression and 1 remains alive with transfusion dependence. No serious infusion-related adverse events occurred with rituximab.

Conclusion: Rituximab is a safe and effective therapeutic option and should be considered as first-line treatment for patients with CAD.

Disclosure: No relevant conflicts of interest to declare.

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