Cyclosporin A for the treatment of cytopenia associated with chronic lymphocytic leukemia

J Cortes, S O'Brien, J Loscertales, H Kantarjian, F Giles, D Thomas, C Koller, M Keating
Cancer 2001 October 15, 92 (8): 2016-22

BACKGROUND: Autoimmune cytopenias are a frequent complication in patients with chronic lymphocytic leukemia (CLL). Anecdotal reports suggest that cyclosporin A (CsA) may be beneficial for patients with CLL-associated pure red cell aplasia. In the current study, the authors investigated the use of CsA in the management of anemia or thrombocytopenia of presumed autoimmune etiology associated with CLL.

METHODS: Thirty-one patients with CLL and anemia or thrombocytopenia of presumed autoimmune etiology were treated with CsA at a dose of 300 mg/day. Sixteen patients (52%) had anemia (hemoglobin <or= 11 g/dL) and 29 patients (94%) had thrombocytopenia (platelet count <or= 100 x 10(9)/L). Seventeen patients (55%) had cytopenia that developed during the course of treatment with fludarabine-based regimens. Nineteen patients (61%) had received prior therapy for this complication using steroids, intravenous immunoglobulin, and/or splenectomy.

RESULTS: Eighteen patients (62%) with thrombocytopenia and 10 patients (63%) with anemia had a major response defined as an increase in the platelet count >or= 50 x 10(9)/L or an increase in hemoglobin >or= 3 g/dL. The median time to initial response was 3 weeks (range, 1-13 weeks) and the median time to best response was 10.5 weeks (range, 1-48 weeks). The median duration of response was 10 months (range, 1+-39+ months). Three patients with fludarabine-associated cytopenias were able to receive further therapy with fludarabine with a lesser decrease in the platelet count. A modest decrease in the tumor burden was observed in six patients. The most common toxicity was <or= Grade 2 (according to the National Cancer Institute's Common Toxicity Criteria) elevation of creatinine, which was observed in 6 patients (19%). Three patients developed opportunistic infections.

CONCLUSIONS: CsA is an effective alternative for the treatment of anemia or thrombocytopenia of suspected autoimmune etiology, including those cases occurring in the course of treatment with fludarabine. A modest antileukemic effect was observed in some patients.

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