title: Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia.
abstract: Treatments for immune thrombocytopenic purpura (ITP) providing durable platelet responses without continued dosing are limited. Whereas complete responses (CRs) to B-cell depletion in ITP usually last for 1 year in adults, partial responses (PRs) are less durable. Comparable data do not exist for children and 5-year outcomes are unavailable. Patients with ITP treated with rituximab who achieved CRs and PRs (platelets > 150 × 10(9)/L or 50-150 × 10(9)/L, respectively) were selected to be assessed for duration of their response; 72 adults whose response lasted at least 1 year and 66 children with response of any duration were included. Patients had baseline platelet counts < 30 × 10(9)/L; 95% had ITP of > 6 months in duration. Adults and children each had initial overall response rates of 57% and similar 5-year estimates of persisting response (21% and 26%, respectively). Children did not relapse after 2 years from initial treatment whereas adults did. Initial CR and prolonged B-cell depletion predicted sustained responses whereas prior splenectomy, age, sex, and duration of ITP did not. No novel or substantial long-term clinical toxicity was observed. In summary, 21% to 26% of adults and children with chronic ITP treated with standard-dose rituximab maintained a treatment-free response for at least 5 years without major toxicity. These results can inform clinical decision-making.
authors.affiliation: Department of Pediatrics, Division of Hematology/Oncology, Platelet Disorders Research and Treatment Program, Weill Medical College of Cornell University, 525 E 68th St, New York, NY 10065, USA. vivek.patel@med.einstein.yu.edu
authors.forename: Vivek L
authors.initials: VL
authors.lastname: Patel
authors.forename: Matthieu
authors.initials: M
authors.lastname: Mahévas
authors.forename: Soo Y
authors.initials: SY
authors.lastname: Lee
authors.forename: Roberto
authors.initials: R
authors.lastname: Stasi
authors.forename: Susanna
authors.initials: S
authors.lastname: Cunningham-Rundles
authors.forename: Bertrand
authors.initials: B
authors.lastname: Godeau
authors.forename: Julie
authors.initials: J
authors.lastname: Kanter
authors.forename: Ellis
authors.initials: E
authors.lastname: Neufeld
authors.forename: Tillmann
authors.initials: T
authors.lastname: Taube
authors.forename: Ugo
authors.initials: U
authors.lastname: Ramenghi
authors.forename: Shalini
authors.initials: S
authors.lastname: Shenoy
authors.forename: Mary J
authors.initials: MJ
authors.lastname: Ward
authors.forename: Nino
authors.initials: N
authors.lastname: Mihatov
authors.forename: Vinay L
authors.initials: VL
authors.lastname: Patel
authors.forename: Philippe
authors.initials: P
authors.lastname: Bierling
authors.forename: Martin
authors.initials: M
authors.lastname: Lesser
authors.forename: Nichola
authors.initials: N
authors.lastname: Cooper
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use; D007167:Immunotherapy / Q000379:methods; D008297:Male; D008875:Middle Aged; D008954:Models, Biological; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000628:therapy*; D000069283:Rituximab; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D000069283:Rituximab