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Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children


This is a prospective, open label, randomized, two-arm, multi-center Phase 3 trial. Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard therapies. The primary objective is to determine if the proportion of patients with platelet response is significantly greater in patients treated with eltrombopag compared to those treated with standard therapies.

Eligibility Criteria

  • Ages eligible for study: 1-18 years
  • Sexes eligible for study: all
  • Platelets <30 x 10^9/L at screening
  • Requires pharmacologic treatment from the perspective of the treating clinician.
  • Newly Diagnosed ITP (<3 months from diagnosis)

Detailed inclusion and exclusion criteria


Jenny Despotovic, DO
Texas Children’s Hematology Center