Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: A study of 11 cases

Fadi Fakhouri, Jean Paul Vernant, Agnès Veyradier, Martine Wolf, Gilles Kaplanski, Raynald Binaut, Manfred Rieger, Friedrich Scheiflinger, Pascale Poullin, Benjamin Deroure, Richard Delarue, Philippe Lesavre, Philippe Vanhille, Olivier Hermine, Giuseppe Remuzzi, Jean Pierre Grünfeld

Research output: Contribution to journalArticlepeer-review

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease that occurs mainly in young adults. Acquired cases are usually a result of antibodies directed against ADAMTS13 (a disintegrin-like and metalloprotease [reprolysin type] with thrombospondin type 1 motif 13), a protease that cleaves the von Willebrand factor multimers. Prognosis has been improved by plasma therapy, but some acute severe forms are refractory to this treatment and achieving a sustained remission is still a challenge in chronic relapsing forms. We therefore conducted a multicentric open-label prospective trial to test the efficacy of rituximab, an anti-B-cell monoclonal antibody, as a curative and prophylactic treatment in patients with TTP as a result of anti-ADAMTS13 antibodies. Six patients were included during an acute refractory TTP episode. Five patients with severe relapsing TTP and persistent anti-ADAMTS13 antibodies were prophylactically treated during remission. All patients received 4 weekly infusions of rituximab. The target of treatment was to restore a significant ADAMTS13 plasma activity (> 10%). Treatment with rituximab led to clinical remission in all cases of acute refractory TTP. In all patients, anti-ADAMTS13 antibodies disappeared, and a significant (18%-75%) plasma ADAMTS13 activity was detected following treatment. Tolerance of rituximab was good. Rituximab is a promising first-line immunosuppressive treatment in patients with acute refractory and severe relapsing TTP related to anti-ADAMTS13 antibodies.

Original languageEnglish
Pages (from-to)1932-1937
Number of pages6
JournalBlood
Volume106
Issue number6
DOIs
Publication statusPublished - Sep 15 2005

ASJC Scopus subject areas

  • Hematology

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