title: Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura.
abstract: We assessed safety and efficacy of rituximab in a prospective study of 36 patients, age 2.6 to 18.3 years, with severe chronic immune thrombocytopenic purpura (ITP). The primary outcome of sustained platelets above 50 x 10(9)/L (50,000/mm3) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [CI], 16% to 48%). Median response time was 1 week (range, 1 to 7 weeks). Attainment of the primary outcome was not associated with age, prior pharmacologic responses, prior splenectomy, ITP duration, screening platelet count, refractoriness, or IgM reduction. First-dose, infusion-related toxicity was common (47%) despite premedication. Significant drug-related toxicities included third-dose hypotension (n = 1) and serum sickness (n = 2). Peripheral B cells were depleted in all subjects. IgM decreased 3.4% per week, but IgG did not significantly decrease. Rituximab was well tolerated, with manageable infusion-related side effects, but 6% of subjects developed serum sickness. Rituximab is beneficial for some pediatric patients with severe, chronic ITP.
authors.affiliation: Division of Hematology/Oncology, Children's Hospital Boston, Boston, MA 02115, USA.
authors.forename: Carolyn M
authors.initials: CM
authors.lastname: Bennett
authors.forename: Zora R
authors.initials: ZR
authors.lastname: Rogers
authors.forename: Daniel D
authors.initials: DD
authors.lastname: Kinnamon
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
authors.forename: Donald H
authors.initials: DH
authors.lastname: Mahoney
authors.forename: Thomas C
authors.initials: TC
authors.lastname: Abshire
authors.forename: Hadi
authors.initials: H
authors.lastname: Sawaf
authors.forename: Theodore B
authors.initials: TB
authors.lastname: Moore
authors.forename: Mignon L
authors.initials: ML
authors.lastname: Loh
authors.forename: Bertil E
authors.initials: BE
authors.lastname: Glader
authors.forename: Maggie C
authors.initials: MC
authors.lastname: McCarthy
authors.forename: Brigitta U
authors.initials: BU
authors.lastname: Mueller
authors.forename: Thomas A
authors.initials: TA
authors.lastname: Olson
authors.forename: Adonis N
authors.initials: AN
authors.lastname: Lorenzana
authors.forename: William C
authors.initials: WC
authors.lastname: Mentzer
authors.forename: George R
authors.initials: GR
authors.lastname: Buchanan
authors.forename: Henry A
authors.initials: HA
authors.lastname: Feldman
authors.forename: Ellis J
authors.initials: EJ
authors.lastname: Neufeld
mesh_terms: D000293:Adolescent; D000911:Antibodies, Monoclonal / Q000627:therapeutic use* / Q000633:toxicity*; D058846:Antibodies, Monoclonal, Murine-Derived; D002648:Child; D002675:Child, Preschool; D006801:Humans; D007022:Hypotension / Q000139:chemically induced; D007155:Immunologic Factors / Q000627:therapeutic use* / Q000633:toxicity*; D018579:Patient Selection; D011446:Prospective Studies; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D000069283:Rituximab; D012713:Serum Sickness / Q000139:chemically induced; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D000069283:Rituximab