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Blood First Edition Paper, prepublished online November 6, 2009; DOI 10.1182/blood-2009-06-222448.
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Submitted June 4, 2009; accepted October 13, 2009.

Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Donald M. Arnold1,1, Ishac Nazi2, Aurelio Santos2, Howard Chan1, Nancy M. Heddle1, Theodore E. Warkentin2 and John G. Kelton1

1 Department of Medicine, Michael G. DeGroote School of Medicine, McMaster University, Hamilton, Ontario, Canada; 2 Department of Pathology and Molecular Medicine, Michael G. DeGroote School of Medicine, McMaster University, Hamilton, Ontario, Canada

* Corresponding author; email: arnold{at}mcmaster.ca

Abstract

Treatment options for patients with chronic refractory immune thrombocytopenic purpura (ITP) are limited. Because combination immunosuppressant therapy had proved effective in ITP and other disorders, we used this approach in patients with particularly severe and refractory ITP. In this retrospective, observational study, we determined the response (platelet count above 30x109/L and doubling of baseline) among 19 refractory ITP patients. Treatment consisted of azathioprine, mycophenolate mofetil and cyclosporine. The patients had failed a median of 6 prior treatments, including splenectomy (in all except one). Of 19 patients, 14 (73.7%) achieved a response lasting a median of 24.0 months after which time 8 (57.1%) relapsed. Of the 8 relapsing patients, 6 responded to additional treatments. Of the 14 patients who achieved an initial response, 2 (14.3%) remained in remission after eventually stopping all medications. Severe adverse events did not occur. Combination immunosuppressant therapy can produce a rise in the platelet count, sometimes sustained in refractory ITP patients.


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