title: Gender and duration of disease differentiate responses to rituximab-dexamethasone therapy in adults with immune thrombocytopenia.
abstract: Adults often develop chronic immune thrombocytopenia (ITP) for which treatment order is uncertain. Rituximab and three cycles of dexamethasone (4R + 3Dex) improve treatment responses and short-term disease control but long-term outcome is not known. In adults with ITP treated with 4R + 3D, we sought long-term outcome and associated prognostic variables. Forty-nine adults treated at Weill-Cornell received 4R + 3Dex. Their clinical characteristics were reviewed. Duration was median time to treatment failure; Kaplan-Meier estimates were developed. Vbeta Tcell receptor (VBTCR) repertoire was obtained after treatment in 36 patients. Patients were adults with ITP 18-64 years old, median age 37. The 27 females were twice as likely to have an ongoing response to 4R + 3Dex (44.1%) as males (19.6%; P = 0.009). For ITP duration <12 months, 52.7% of patients had continuing responses to 4R + 3Dex compared to 15.3% of patients with diagnosis >12 months (P = 0.02). Females with ITP duration of <12 months had continuing responses in 78.6%, compared to males with <12 months duration of ITP (21.2%). For patients with disease duration <12 months, 67% of females had continuing responses, compared to 31% of males (P = 0.004). Post-treatment polyclonal VBTCR was seen in 9/10 continuing responders (six female, three male) but only 13/26 relapsers/nonresponders (P = 0.068). Durable remissions after treatment with 4R + 3Dex were more frequent in female patients with <12 months of ITP duration and those with polyclonal VBTCR after treatment, emphasizing the roles of duration of disease, gender and T cells in chronic ITP. Differences in pathophysiology of ITP by gender and by duration of ITP require further study. Am. J. Hematol. 91:907-911, 2016. © 2016 Wiley Periodicals, Inc.
authors.affiliation: Division of Hematology-Oncology, Department of Medicine, New York Presbyterian Hospital, Weill Cornell Medicine, New York, New York.
authors.forename: John
authors.initials: J
authors.lastname: Chapin
authors.affiliation: Division of Hematology-Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, New York.
authors.forename: Christina S
authors.initials: CS
authors.lastname: Lee
authors.affiliation: Department of Pathology, Stanford University School of Medicine, Palo Alto, California.
authors.forename: Haiyu
authors.initials: H
authors.lastname: Zhang
authors.affiliation: Department of Pathology, Stanford University School of Medicine, Palo Alto, California.
authors.forename: James L
authors.initials: JL
authors.lastname: Zehnder
authors.affiliation: Division of Hematology-Oncology, Department of Medicine, New York Presbyterian Hospital, Weill Cornell Medicine, New York, New York.|Division of Hematology-Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, New York.
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
mesh_terms: D000293:Adolescent; D000328:Adult; D003907:Dexamethasone / Q000008:administration & dosage*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D016693:Receptors, Antigen, T-Cell, alpha-beta; D012074:Remission Induction / Q000379:methods; D000069283:Rituximab / Q000008:administration & dosage*; D012737:Sex Factors; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016693:Receptors, Antigen, T-Cell, alpha-beta; D000069283:Rituximab; D003907:Dexamethasone