High-dose erythropoietin fails in extremely preterm infants

  • N Engl J Med

  • de Emily Willingham, PhD
  • Clinical Essentials
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  • High-dose erythropoietin (EPO) in extremely preterm infants failed to yield improvements in risks for neurodevelopmental impairment or death.
  • Disappointing results from this placebo-controlled, randomized trial of 32 weeks of treatment.

Why this matters

  • Preclinical data suggested some protective potential of EPO, as have results of phase 2 trials.

Key results

  • 91% infants received all 6 planned EPO doses.
  • The EPO group did not differ from the placebo group for death or severe neurodevelopmental impairment:
    • 26% with EPO vs 26% with placebo.
    • Relative risk: 1.03 (95% CI, 0.81-1.32).
  • Mortality rate was 13% with EPO vs 11% with placebo.
    • Relative risk: 1.27 (95% CI, 0.91-1.79).
  • Severe neurodevelopmental impairment rate was 11% with EPO vs 14% with placebo.
    • Relative risk: 0.79 (95% CI, 0.51-1.22).
  • Other analyses showed no differences between groups for endpoints including retinopathy of prematurity, sepsis, or serious adverse events.

Study design

  • Multicenter, randomized, double-blind trial, 941 infants (477 EPO; 464 placebo) born at 24 weeks, 0 days to 27 weeks, 6 days of gestation.
  • EPO given intravenously at 1000 U/kg body weight every 48 hours, total 6 doses.
  • Maintenance dose, 400 U/kg body weight 3x/week, subcutaneous injection to 32 weeks, 6 days postmenstrual age.
  • Funding: National Institute of Neurological Disorders and Stroke.


  • Neurodevelopmental testing done at age 2 years, rather than more reliable testing at age 3 years.