Relapse Rate in Survivors of Acute Autoimmune Thrombotic Thrombocytopenic Purpura Treated with or without RituximabFunding This study (BMBF 01EO1503) as well as Tanja Falter (BMBF 01EO1003) were supported by the Federal Ministry of Education and Research.
19 February 2018
11 July 2018
20 September 2018 (eFirst)
Background Autoimmune thrombotic thrombocytopenic purpura (iTTP) is caused by autoantibody-mediated severe a disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13 (ADAMTS13) deficiency leading to micro-angiopathic haemolytic anaemia (MAHA) and thrombocytopenia with organ damage. Patients survive with plasma exchange (PEX), fresh frozen plasma replacement and corticosteroid treatment. Anti-CD20 monoclonal antibody rituximab is increasingly used in patients resistant to conventional PEX or relapsing after an acute bout.
Objective This retrospective observational study focused on the relapse rate and possible influencing factors including treatment with rituximab first introduced in 2003.
Patients and Methods Seventy patients treated between January 2003 and November 2014 were evaluated. Number, duration, clinical manifestations, laboratory data and treatment of acute episodes were documented. Diagnostic criteria of acute iTTP were thrombocytopenia, MAHA, increased lactate dehydrogenase and severe ADAMTS13 deficiency.
Results Fifty-four female and 16 male patients had a total of 224 acute episodes over a median observation period of 8.3 years. The relapse rate was 2.6% per month, for women 2.4% and for men 3.5% per month. Since 2003, 17 patients with a first iTTP episode were treated with rituximab, whereas 28 were not. There was a trend towards lower relapse rates after rituximab treatment over the ensuing years. However, this was statistically not significant.
Conclusion This analysis does not show a significant reduction of acute iTTP relapses by rituximab given during an acute bout. Initial episodes are characterized by more severe clinical signs compared with the less severe relapses. Furthermore, men suffer significantly more frequent and considerably more serious acute relapses.
KeywordsADAMS/ADAMTS13 - thrombotic thrombocytopenic purpura (TTP/HUS) - clinical studies - autoantibodies
T. Falter: Study concept and design, data analysis, writing of the manuscript and approval. S. Herold: Data acquisition and analysis, writing of the manuscript and approval. V. Weyer: Statistical advice, revision of the manuscript and approval. C. Scheiner: Statistical advice, revision of the manuscript and approval. V. Schmitt: Data acquisition, revision of the manuscript and approval. X. Messmer: Data acquisition and approval. C. von Auer: Revision of the manuscript and approval. P. Wild: Revision of the manuscript and approval. K. Lackner: Revision of the manuscript and approval. B. Lämmle: Data compilation, writing of the manuscript, revision of the manuscript and approval. I. Scharrer: Study initiation, revision of the manuscript, writing of the manuscript and approval.
* Tanja Falter and Stephanie Herold are first co-authors of the study.
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