Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study.

Abstract

Whether rituximab could effectively and safely avoid splenectomy for adults with chronic immune thrombocytopenic purpura (ITP) remains unresolved. A multicenter, prospective, open-label, single-arm, phase 2 trial was conducted to assess rituximab safety and efficacy in adult splenectomy candidates with chronic ITP. Sixty patients with chronic (>or= 6 months) ITP and platelet counts less than 30 x 10(9)/L received a weekly intravenous infusion of rituximab (375 mg/m(2)) for 4 weeks. All other ITP treatments were stopped. A good response was defined as a platelet count 50 x 10(9)/L or more, with at least a doubling of the initial value at 1 and 2 years after the first rituximab infusion. Patients who required another treatment during follow up were considered nonresponders. Sixteen patients experienced transient side effects that necessitated treatment discontinuation for only 1. Good 1-year responses were obtained in 40% of the patients (24/60 [95% confidence interval: 28%-52%]). At 2 years, 33.3% (20/60 patients) had good responses and 6.7% (4/60) had sustained platelet counts of 30 x 10(9)/L or more without treatment. Thirty-six (60%) patients failed to respond; 25 underwent splenectomy. Based on these results, rituximab was an apparently safe and effective splenectomy-avoiding option in some adults with chronic ITP. This trial is registered at http://clinicaltrials.gov as NCT00225875.

• Authors: Bertrand Godeau, Raphael Porcher, Olivier Fain, François Lefrère, Pierre Fenaux, Stéphane Cheze, Anne Vekhoff, Marie-Paule Chauveheid, Jerôme Stirnemann, Lionel Galicier, Emmanuelle Bourgeois, Stéphanie Haiat, Bruno Varet, Michel Leporrier, Thomas Papo, Mehdi Khellaf, Marc Michel, Philippe Bierling
• Journal: Blood (Blood) Vol. 112 Issue 4 Pg. 999-1004 (Aug 15 2008) ISSN: 1528-0020 [Electronic] United States
• PMID: 18463354 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)

Chemical References

• Antibodies, Monoclonal
• Antibodies, Monoclonal, Murine-Derived
• Rituximab

Topics

• Adolescent
• Adult
• Aged
• Aged, 80 and over
• Antibodies, Monoclonal (administration & dosage, toxicity)
• Antibodies, Monoclonal, Murine-Derived
• Chronic Disease
• Drug-Related Side Effects and Adverse Reactions
• Female
• Follow-Up Studies
• Humans
• Male
• Middle Aged
• Platelet Count
• Purpura, Thrombocytopenic, Idiopathic (drug therapy)
• Remission Induction
• Rituximab
• Splenectomy
• Treatment Outcome