title: Relapsing or refractory idiopathic thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: the role of rituximab.
abstract: Idiopathic thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) is a rare disease responsive to treatment with plasma exchange (PE) but with a high percentage of relapse or refractory patients. A severe deficiency of ADAMTS-13 (<5% of normal activity), congenital or caused by an autoantibody, may be specific for TTP and it has been proposed that severe ADAMTS-13 deficiency now defines TTP. B cells play a key role in both the development and the perpetuation of autoimmunity, suggesting that B-cell depletion could be a valuable treatment approach for patients with idiopathic TTP-HUS. This review of the literature focuses on the role of rituximab, a chimeric monoclonal antibody directed against CD20 antigen expressed by B lymphocytes, in patients with relapsing or refractory TTP-HUS with or without ADAMTS-13 deficiency, suggesting that rituximab may produce clinical remission in a significant proportion of patients. Rituximab therapy reduces plasma requirement and avoids complications related to salvage-immunosuppressive therapy. In conclusion, rituximab provides an effective, well-tolerated, and safe treatment option for patients with idiopathic TTP-HUS, thus giving an alternative approach to the current treatment based on PE.
authors.affiliation: Cattedra ed U.O. di Ematologia con trapianto, U.O. di Immunoematologia e Servizio Trasfusionale, and U.O. di Neurologia, Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone di Palermo, Palermo, Italy.
authors.forename: Domenica
authors.initials: D
authors.lastname: Caramazza
authors.forename: Gerlando
authors.initials: G
authors.lastname: Quintini
authors.forename: Ignazio
authors.initials: I
authors.lastname: Abbene
authors.forename: Alessandra
authors.initials: A
authors.lastname: Malato
authors.forename: Giorgia
authors.initials: G
authors.lastname: Saccullo
authors.forename: Lucio
authors.initials: L
authors.lastname: Lo Coco
authors.forename: Rosa
authors.initials: R
authors.lastname: Di Trapani
authors.forename: Roberto
authors.initials: R
authors.lastname: Palazzolo
authors.forename: Rita
authors.initials: R
authors.lastname: Barone
authors.forename: Giuseppina
authors.initials: G
authors.lastname: Mazzola
authors.forename: Sergio
authors.initials: S
authors.lastname: Rizzo
authors.forename: Paolo
authors.initials: P
authors.lastname: Ragonese
authors.forename: Paolo
authors.initials: P
authors.lastname: Aridon
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Abbadessa
authors.forename: Sergio
authors.initials: S
authors.lastname: Siragusa
mesh_terms: D051722:ADAM Proteins / Q000037:antagonists & inhibitors / Q000276:immunology; D000071120:ADAMTS13 Protein; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000627:therapeutic use; D058846:Antibodies, Monoclonal, Murine-Derived / Q000008:administration & dosage / Q000502:physiology* / Q000627:therapeutic use*; D004351:Drug Resistance / Q000502:physiology; D006801:Humans; D007155:Immunologic Factors / Q000494:pharmacology / Q000627:therapeutic use; D007167:Immunotherapy / Q000379:methods; D011697:Purpura, Thrombotic Thrombocytopenic / Q000628:therapy*; D012008:Recurrence; D000069283:Rituximab
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D000069283:Rituximab; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human