Study of the Effectiveness of Rituximab in Adults With Chronic and Severe Immune Thrombocytopenic Purpura and Candidate for a Splenectomy

Autoimmune Thrombocytopenic Purpura Clinical Trial

Official title: Evaluation De l’Efficacité Du Rituximab (Mabthéra) Chez l'Adulte Atteint d'Un Purpura thrombopénique Auto-Immun Chronique Et sévère Et Candidat à La splénectomie

Status Terminated

Clinical Trial Summary

The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets <= 30x109/L) and candidate to a splenectomy. The objective is to obtain after a treatment by the rituximab a satisfactory response to one year, defined by a number of platelets higher than 50x109/L and at least 2 times superior with the persistent initial figure without treatment during one year after the end of the treatment.

Clinical Trial Description

Adults immune thrombocytopenic purpura has an evolution which is generally chronic defined by the persistence of the thrombocytopenia 6 months after the diagnosis. The treatment is then based on the splenectomy which is proposed by the majority of the teams when the platelets are lower than 30x109/L. The splenectomy is effective at 70 to 80 % of the patients whereas no medicamentous treatment makes it possible to obtain a comparable result. Nevertheless, it exposes to immediate post-operative complications and to a risk of mortal fulminant infections by encapsulated germs, in particular the pneumococcus. However, its long-term effectiveness is discussed with a risk of relapse which would reach 50 % for certain teams.

The rituximab could be an alternative to the splenectomy because of its great frequency of effectiveness and its good tolerance in the short and medium term. None the medicamentous treatments usually suggested in alternative to the splenectomy (disulone, danazol, immunosuppressors) indeed makes it possible to obtain an answer prolonged after the stop of therapeutic in a significant number of cases. Moreover, the use of the immunosuppressors such as the cyclophosphamide, the azathioprine or the ciclosporine appears contestable at this stage of the disease because of potential severity their side effects.The primary endpoint is satisfactory response to one year, defined by a figure of plates >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment by rituximab. Secondary objectives are incomplete response to one year, defined by a figure of platelets >= 30x109/L and < 50x109/L and at least twice the figure initial or > 50x109/L but lower than twice the persistent initial figure without treatment during one year after the end of the treatment by rituximab. Splenectomy at one year satisfactory Response to 2 years incomplete Response to 2 years Splenectomies at 2 years Tolerance of the treatment. ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autoimmune Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

• NCT number: NCT00225875
• Study type: Interventional
• Source: Assistance Publique - Hôpitaux de Paris
• Status: Terminated
• Phase: Phase 2
• Start date: September 2003
• Completion date: July 2007