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 language | English |
|---|---|
| Pages (from-to) | 1932-1937 |
| Number of pages | 6 |
| Journal | Blood |
| Volume | 106 |
| Issue number | 6 |
| DOIs | |
| Publication status | Published - Sep 15 2005 |
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ASJC Scopus subject areas
- Hematology
Cite this
Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura : A study of 11 cases. / Fakhouri, Fadi; Vernant, Jean Paul; Veyradier, Agnès; Wolf, Martine; Kaplanski, Gilles; Binaut, Raynald; Rieger, Manfred; Scheiflinger, Friedrich; Poullin, Pascale; Deroure, Benjamin; Delarue, Richard; Lesavre, Philippe; Vanhille, Philippe; Hermine, Olivier; Remuzzi, Giuseppe; Grünfeld, Jean Pierre.
In: Blood, Vol. 106, No. 6, 15.09.2005, p. 1932-1937.Research output: Contribution to journal › Article
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TY - JOUR
T1 - Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura
T2 - A study of 11 cases
AU - Fakhouri, Fadi
AU - Vernant, Jean Paul
AU - Veyradier, Agnès
AU - Wolf, Martine
AU - Kaplanski, Gilles
AU - Binaut, Raynald
AU - Rieger, Manfred
AU - Scheiflinger, Friedrich
AU - Poullin, Pascale
AU - Deroure, Benjamin
AU - Delarue, Richard
AU - Lesavre, Philippe
AU - Vanhille, Philippe
AU - Hermine, Olivier
AU - Remuzzi, Giuseppe
AU - Grünfeld, Jean Pierre
PY - 2005/9/15
Y1 - 2005/9/15
N2 - 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.
AB - 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.
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U2 - 10.1182/blood-2005-03-0848
DO - 10.1182/blood-2005-03-0848
M3 - Article
VL - 106
SP - 1932
EP - 1937
JO - Blood
JF - Blood
SN - 0006-4971
IS - 6
ER -