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Blood, 1 August 2005, Vol. 106, No. 3, pp. 925-928. Prepublished online as a Blood First Edition Paper on April 12, 2005; DOI 10.1182/blood-2004-12-4885.
Submitted December 27, 2004
Mario Negri Institute for Pharmacological Research, Clinical Research Center for Rare Diseases, Aldo e Cele Dacco, Ranica, Bergamo, Italy * Corresponding author; email: gremuzzi{at}marionegri.it.
Thrombotic thrombocytopenic purpura (TTP) is a rare disorder of small vessels that is associated with deficiency of the von Willebrand factor-cleaving protease, ADAMTS-13. The presence of anti-ADAMTS-13 autoantibodies is considered a factor predisposing to relapses. Despite close monitoring and intensive plasma treatment, in these patients acute episodes are still associated with substantial morbidity and mortality rates and the optimal therapeutic option should rather be prevention of relapses. This study was conducted in a patient with recurrent TTP due to high titers of ADAMTS-13 inhibitors, who used to have 2 relapses of TTP a year and compared the standard treatment plasma exchange with rituximab. Results documented that plasma exchange had only a small transient effect on ADAMTS-13 activity and inhibitors, on the contrary prophylaxis with rituximab was associated with disappearance of anti-ADAMTS-13 antibodies, a progressive recovery of protease activity, and allowed the patient to maintain a disease-free state for more than 2 year follow-up.
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