1. Relapse Rate in Survivors of Acute Autoimmune Thrombotic Thrombocytopenic Purpura Treated with or without Rituximab.
- Author
-
Falter T, Herold S, Weyer-Elberich V, Scheiner C, Schmitt V, von Auer C, Messmer X, Wild P, Lackner KJ, Lämmle B, and Scharrer I
- Subjects
- Adolescent, Adult, Antigens, CD20 immunology, Autoantibodies blood, Child, Female, Follow-Up Studies, Humans, Male, Middle Aged, Recurrence, Retrospective Studies, Sex Factors, Treatment Outcome, Young Adult, ADAMTS13 Protein deficiency, Autoimmune Diseases drug therapy, Immunologic Factors therapeutic use, Purpura, Thrombotic Thrombocytopenic drug therapy, Rituximab therapeutic use
- Abstract
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., Competing Interests: The authors declare that they have no conflicts of interest relevant to the manuscript. I. Scharrer is a member of the Data Safety Monitoring Board in the BAX 930 study (investigating recombinant ADAMTS13 infusion in hereditary TTP). She received travel and accommodation support for participating at scientific congresses or meetings from Bayer and NovoNordisk. B. Lämmle is chairman of the Data Safety Monitoring Board in the BAX 930 study (investigating recombinant ADAMTS13 infusion in hereditary TTP). He is on the Advisory Board of Ablynx for the development of caplacizumab. He holds a patent on ADAMTS13 and received travel and accommodation support for participating at scientific congresses or meetings from Baxalta, Siemens, Alexion, Ablynx and Bayer and speaker's fees from Siemens, Bayer and Alexion., (Georg Thieme Verlag KG Stuttgart · New York.)
- Published
- 2018
- Full Text
- View/download PDF