title: 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.affiliation: Médecine Interne, Hôpital Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris 12, Créteil, France.
authors.forename: Bertrand
authors.initials: B
authors.lastname: Godeau
authors.forename: Raphael
authors.initials: R
authors.lastname: Porcher
authors.forename: Olivier
authors.initials: O
authors.lastname: Fain
authors.forename: François
authors.initials: F
authors.lastname: Lefrère
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: Stéphane
authors.initials: S
authors.lastname: Cheze
authors.forename: Anne
authors.initials: A
authors.lastname: Vekhoff
authors.forename: Marie-Paule
authors.initials: MP
authors.lastname: Chauveheid
authors.forename: Jerôme
authors.initials: J
authors.lastname: Stirnemann
authors.forename: Lionel
authors.initials: L
authors.lastname: Galicier
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Bourgeois
authors.forename: Stéphanie
authors.initials: S
authors.lastname: Haiat
authors.forename: Bruno
authors.initials: B
authors.lastname: Varet
authors.forename: Michel
authors.initials: M
authors.lastname: Leporrier
authors.forename: Thomas
authors.initials: T
authors.lastname: Papo
authors.forename: Mehdi
authors.initials: M
authors.lastname: Khellaf
authors.forename: Marc
authors.initials: M
authors.lastname: Michel
authors.forename: Philippe
authors.initials: P
authors.lastname: Bierling
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000633:toxicity; D058846:Antibodies, Monoclonal, Murine-Derived; D002908:Chronic Disease; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D012074:Remission Induction; D000069283:Rituximab; D013156:Splenectomy; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000069283:Rituximab