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Wednesday 01 May 2002

Rituximab therapy for refractory thrombotic thrombocytopenic purpura.

By: Gutterman LA, Kloster B, Tsai HM.

Blood Cells Mol Dis 2002 May-Jun;28(3):385-91

While most patients of thrombotic thrombocytopenic purpura (TTP) respond to plasma exchange and achieve remission, a subset of the patients experience multiple relapses or develop a persistent disease that may be debilitating and life threatening. We report the experience of rituximab treatment in three consecutive patients who had required periodic plasma exchange for greater than 50-180 days after failing other modalities of treatments. Two patients each received eight doses of rituximab infusion and had clinical remissions that have lasted for 23 months and ongoing in one patient and for 17 months before relapse in the other. The third patient received four doses of rituximab infusion and had improvement of disease for 60 days until her death from unrelated causes. Analysis of the von Willebrand factor-cleaving metalloprotease activity (ADAMTS13) and its inhibitor in two patients showed that rituximab treatment was associated with a rise of the protease level and a decrease of the inhibitor titers in one patient who achieved clinical remission and a decrease of the inhibitor titer but no increase in protease level in the other patient who had a partial response. Based on the responses to rituximab and its mild toxicity in these three patients, and the lack of effective alternative treatments, additional exploration of the role of rituximab in the treatment of refractory TTP is warranted.

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