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Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone

      Objectives

      To assess initial and long-term outcome of children with persistent/chronic idiopathic thrombocytopenic purpura (ITP) treated with 4 infusions of rituximab and three 4-day cycles of dexamethasone (4R+3Dex) including cohorts with most benefit and/or treatment associated toxicity.

      Study design

      All pediatric patients with ITP at Weill-Cornell who received 4R+3Dex were included in this retrospective study. Duration was median time from first rituximab infusion to treatment failure. Patient cohort included 33 children ages 1-18 years with persistent/chronic ITP; 19 were female, 10 of whom were adolescents. Every patient had failed more than 1 and usually several ITP treatments.

      Results

      Children were treated with rituximab, 375 mg/m2 weekly for 4 weeks and three 4-day courses of dexamethasone 28 mg/m2 (40 mg max). Average age of nonresponders was 7.75 years, and initial responders averaged 12.7 years (P = .0073); 30% maintained continuing response at 60 months or last check-up. Eight of the 10 patients who underwent remission were female with ITP <24 months prior to initiating 4R+3Dex. All responding male patients except 2 relapsed.

      Conclusions

      Durable unmaintained ITP remission after 4R+3Dex was seen almost exclusively in female adolescents with <24 months duration of ITP. This provides a new therapeutic paradigm for a subpopulation with hard-to-treat chronic ITP. The pathophysiology of ITP underlying this distinction requires further elucidation.

      Keywords

      Abbreviations:

      4R+3Dex (4 infusions of rituximab and three 4-day cycles of dexamethasone), CR (Complete response), ITP (Idiopathic thrombocytopenic purpura), PR (Partial response)
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      Linked Article

      • Combination immune-based therapy for chronic ITP
        The Journal of PediatricsVol. 191
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          Idiopathic thrombocytopenia purpura (ITP) is a common pediatric disorder that often dramatically presents with sudden onset bruising, petechiae, and (typically quite severe) thrombocytopenia. While various therapies (glucocorticoids, intravenous gamma globulin, intravenous anti-D) can transiently increase the platelet count, serious bleeding from acute ITP is uncommon, with little evidence that preemptive treatment reduces either the risk of serious bleeding or the already low (10-15%) incidence of developing chronic ITP.
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