title: Safety and efficacy of rituximab in adult immune thrombocytopenia: results from a prospective registry including 248 patients.
abstract: We conducted a prospective multicenter registry of 248 adult patients with immune thrombocytopenia (ITP) treated with rituximab to assess safety. We also assessed response and predictive factors of sustained response. In total, 173 patients received 4 infusions of 375 mg/m(2) and 72 received 2 fixed 1-g infusions 2 weeks apart. The choice of the rituximab regimen was based on the physician's preference and not patient characteristics. Overall, 38 patients showed minor intolerance to rituximab infusions; infusions had to be stopped for only 3 patients. Seven showed infection (n = 11 cases), with an incidence of 2.3 infections/100 patient-years. Three patients died of infection 12 to 14 months after rituximab infusions, but the role of rituximab was questionable. In total, 152 patients (61%) showed an overall initial response (platelet count ≥30 × 10(9)/L and ≥2 baseline value). At a median follow-up of 24 months, 96 patients (39%) showed a lasting response. On multivariate analysis, the probability of sustained response at 1 year was significantly associated with ITP duration <1 year (P = .02) and previous transient complete response to corticosteroids (P = .05). The pattern of response was similar with the 2 rituximab regimens. With its benefit/risk ratio, rituximab used off-label may remain a valid option for treating persistent or chronic ITP in adults. This trial was registered at www.clinicaltrials.gov as #NC1101295.
authors.affiliation: Service de Médecine Interne, Centre de Référence Labellisé des Cytopénies Auto-Immunes de l'Adulte and.
authors.forename: Mehdi
authors.initials: M
authors.lastname: Khellaf
authors.affiliation: Unité de Recherche Clinique, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France;
authors.forename: Anaïs
authors.initials: A
authors.lastname: Charles-Nelson
authors.affiliation: Service de Médecine Interne, Hôpital Jean-Verdier, Assistance Publique-Hôpitaux de Paris, Université Paris XIII, Bondy, France;
authors.forename: Olivier
authors.initials: O
authors.lastname: Fain
authors.affiliation: Service de Médecine Interne, Centre Hospitalier Régional Universitaire de Lille, Lille, France;
authors.forename: Louis
authors.initials: L
authors.lastname: Terriou
authors.affiliation: Département de Médecine Interne et Maladies Infectieuses, Centre Hospitalier Universitaire Haut Lévêque, Université de Bordeaux, Pessac, France;
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Viallard
authors.affiliation: Service d'Hématologie Clinique, Centre Hospitalier Universitaire de la Côte de Nacre, Caen, France;
authors.forename: Stéphane
authors.initials: S
authors.lastname: Cheze
authors.affiliation: Service de Médecine Interne, Centre Hospitalier Universitaire de Nantes, Nantes, France;
authors.forename: Julie
authors.initials: J
authors.lastname: Graveleau
authors.affiliation: Service d'Hématologie, Centre Hospitalier d'Avignon, Avignon, France;
authors.forename: Borhane
authors.initials: B
authors.lastname: Slama
authors.affiliation: Service de Médecine Interne, Hôpital Le Bocage, Dijon, France;
authors.forename: Sylvain
authors.initials: S
authors.lastname: Audia
authors.affiliation: Service de Médecine Interne, Hôpital de La Conception, Assistance Publique-Hôpitaux de Marseille, Université Aix-Marseille, Marseille, France;
authors.forename: Mikael
authors.initials: M
authors.lastname: Ebbo
authors.affiliation: Service de Médecine Interne, Centre Hospitalier Universitaire Estaing, Clermont Ferrand, France;
authors.forename: Guillaume
authors.initials: G
authors.lastname: Le Guenno
authors.affiliation: Département d'Hématologie, Groupe Hospitalier de l'Institut Catholique de Lille, Lille, France;
authors.forename: Manuel
authors.initials: M
authors.lastname: Cliquennois
authors.affiliation: Service d'Hématologie, Hospices Civils de Lyon, Centre Hospitalier Lyon Sud, Pierre-Bénite, University Claude Bernard Lyon 1, Lyon, France;
authors.forename: Gilles
authors.initials: G
authors.lastname: Salles
authors.affiliation: Service d'Hématologie, Centre Hospitalier Universitaire de Nancy, Nancy, France;
authors.forename: Caroline
authors.initials: C
authors.lastname: Bonmati
authors.affiliation: Département d'Immuno-Hématologie, Centre Hospitalier Universitaire Louis Mourrier, Assistance Publique-Hôpitaux de Paris, Colombes, France;
authors.forename: France
authors.initials: F
authors.lastname: Teillet
authors.affiliation: Service d'Immuno-Pathologie, Centre Hospitalier Universitaire Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France;
authors.forename: Lionel
authors.initials: L
authors.lastname: Galicier
authors.affiliation: Service de Médecine Interne, Groupement Hospitalier Edouard Herriot, Lyon, France;
authors.forename: Arnaud
authors.initials: A
authors.lastname: Hot
authors.affiliation: Service de Médecine Interne, Centre Hospitalier Universitaire Bicêtre, Assistance Publique-Hôpitaux de Paris, Paris, France;
authors.forename: Olivier
authors.initials: O
authors.lastname: Lambotte
authors.affiliation: Service d'Hématologie, Centre Hospitalier Universitaire Necker, Assistance Publique-Hôpitaux de Paris, Paris, France;
authors.forename: François
authors.initials: F
authors.lastname: Lefrère
authors.affiliation: Unité de Recherche Clinique, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France;
authors.forename: Salimatou
authors.initials: S
authors.lastname: Sacko
authors.affiliation: Unité de Recherche Clinique, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France;
authors.forename: Dieudonné Kilendo
authors.initials: DK
authors.lastname: Kengue
authors.affiliation: Service de Médecine Interne, Centre de Référence Labellisé des Cytopénies Auto-Immunes de l'Adulte and Etablissement Français du Sang, Henri-Mondor, Creteil, France; and.
authors.forename: Philippe
authors.initials: P
authors.lastname: Bierling
authors.affiliation: Service de Santé Publique, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France.
authors.forename: Françoise
authors.initials: F
authors.lastname: Roudot-Thoraval
authors.affiliation: Service de Médecine Interne, Centre de Référence Labellisé des Cytopénies Auto-Immunes de l'Adulte and.
authors.forename: Marc
authors.initials: M
authors.lastname: Michel
authors.affiliation: Service de Médecine Interne, Centre de Référence Labellisé des Cytopénies Auto-Immunes de l'Adulte and.
authors.forename: Bertrand
authors.initials: B
authors.lastname: Godeau
mesh_terms: D000328:Adult; D000368:Aged; D058846:Antibodies, Monoclonal, Murine-Derived / Q000009:adverse effects / Q000627:therapeutic use*; D002423:Cause of Death; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000453:epidemiology; D012008:Recurrence; D012042:Registries; D000069283:Rituximab; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D000069283:Rituximab