ABSTRACT
In this study, we report the results from the largest cohort to date of newly diagnosed adult immune thrombocytopenia patients randomized to treatment with dexamethasone alone or in combination with rituximab. Eligible were patients with platelet counts ≤25×10(9)/L or ≤50×10(9)/L with bleeding symptoms. A total of 133 patients were randomly assigned to either dexamethasone 40 mg/day for 4 days (n = 71) or in combination with rituximab 375 mg/m(2) weekly for 4 weeks (n = 62). Patients were allowed supplemental dexamethasone every 1 to 4 weeks for up to 6 cycles. Our primary end point, sustained response (ie, platelets ≥50×10(9)/L) at 6 months follow-up, was reached in 58% of patients in the rituximab + dexamethasone group vs 37% in the dexamethasone group (P = .02). The median follow-up time was 922 days. We found longer time to relapse (P = .03) and longer time to rescue treatment (P = .007) in the rituximab + dexamethasone group. There was an increased incidence of grade 3 to 4 adverse events in the rituximab + dexamethasone group (P = .04). In conclusion, rituximab + dexamethasone induced higher response rates and longer time to relapse than dexamethasone alone. This study is registered at http://clinicaltrials.gov as NCT00909077.
Subject(s)
Antibodies, Monoclonal, Murine-Derived/administration & dosage , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Dexamethasone/administration & dosage , Dexamethasone/therapeutic use , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Adult , Age of Onset , Aged , Algorithms , Antibodies, Monoclonal, Murine-Derived/adverse effects , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Dexamethasone/adverse effects , Female , Follow-Up Studies , Humans , Male , Middle Aged , Purpura, Thrombocytopenic, Idiopathic/diagnosis , Purpura, Thrombocytopenic, Idiopathic/epidemiology , Recurrence , Rituximab , Treatment OutcomeABSTRACT
Primary immune thrombocytopenia (ITP)--formerly known as idiopathic thrombocytopenic purpura--is an autoimmune disorder characterized by immune mediated thrombocytopenia. The aetiology of ITP remains unknown, but studies have shown that multiple immunological mechanisms are involved in the pathogenesis of ITP. This article aims to provide an overview of current treatment options, with particular emphasis on new biological therapies: rituximab, a monoclonal anti-CD20 antibody, and the thrombopoietin receptor agonists romiplostim and eltrombopag.
Subject(s)
Purpura, Thrombocytopenic, Idiopathic/drug therapy , Antibodies, Monoclonal, Murine-Derived/therapeutic use , Benzoates/therapeutic use , Dexamethasone/therapeutic use , Glucocorticoids/therapeutic use , Humans , Hydrazines/therapeutic use , Immunoglobulins, Intravenous/therapeutic use , Immunosuppressive Agents/therapeutic use , Prednisolone/therapeutic use , Pyrazoles/therapeutic use , Receptors, Fc/therapeutic use , Recombinant Fusion Proteins/therapeutic use , Rituximab , Splenectomy , Thrombopoietin/therapeutic useABSTRACT
Primary immune thrombocytopenia (ITP)--formerly known as idiopathic thrombocytopenic purpura--is an autoimmune disorder characterized by immune-mediated thrombocytopenia. The aetiology of ITP remains unknown, but studies have shown that multiple immunological mechanisms are involved in the pathogenesis of ITP.This article aims to provide an overview of our knowledge of the pathogenesis of ITP.
