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

J Pediatr. 2017 Dec:191:225-231. doi: 10.1016/j.jpeds.2017.08.036.

Abstract

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: autoimmune disease; immune therapy; platelets; thrombocytopenia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Anti-Inflammatory Agents / administration & dosage*
  • Anti-Inflammatory Agents / therapeutic use
  • Child
  • Child, Preschool
  • Chronic Disease
  • Dexamethasone / administration & dosage*
  • Dexamethasone / therapeutic use
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Humans
  • Immunologic Factors / administration & dosage*
  • Immunologic Factors / therapeutic use
  • Infant
  • Infusions, Intravenous
  • Male
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Retrospective Studies
  • Rituximab / administration & dosage*
  • Rituximab / therapeutic use
  • Treatment Outcome

Substances

  • Anti-Inflammatory Agents
  • Immunologic Factors
  • Rituximab
  • Dexamethasone