Erythropoietin for Neonatal Hypoxic-Ischemic Encephalopathy: A Randomized Clinical Trial - Report - MDSpire
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Erythropoietin Treatment in Neonatal Hypoxic-Ischemic Encephalopathy: Results from a Randomized Clinical Study

  • By

  • Helen G. Liley

  • Rod W. Hunt

  • Rachel L. O’Connell

  • Malcolm R. Battin

  • Iona Novak

  • Yvonne W. Wu

  • Roberta Ballard

  • Lisa Askie

  • Nadia Badawi

  • Alpana Ghadge

  • Susan E. Jacobs

  • Sandra E. Juul

  • Lucille Sebastian

  • Deepika Wagh

  • R. John Simes

  • PAEAN Study Investigators

  • Shannon Clough

  • Jennifer Bowen

  • Paul Colditz

  • Girsih Deshpande

  • Lisa Gold

  • Wendy Hague

  • Kei Lui

  • Dominic Wilkinson

  • Nicola Austin

  • Sarah Bellhouse

  • Max Berry

  • Clare Collins

  • Amanda Dyson

  • Antonio G de Paoli

  • Koert de Waal

  • Bevan Headley

  • Leah Hickey

  • James Holberton

  • David Hou

  • Alison L Kent

  • Vinayak Kodur

  • Richard Mausling

  • Scott Morris

  • Arun Nair

  • David Osborn

  • Victor Samuel Rajadurai

  • Nadia Schmidt

  • Jason Tan

  • Mark B Tracy

  • Flora Y Wong

  • Sarah Finlayson

  • Shannon Hunt

  • Carbo Yeung

  • Laurence Ralston

  • October 1, 2026

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Clinical Report: Erythropoietin Treatment in Neonatal Hypoxic-Ischemic Encephalopathy

Overview

The PAEAN trial evaluated the efficacy of high-dose erythropoietin (EPO) in reducing death and disability in infants with hypoxic-ischemic encephalopathy (HIE) receiving therapeutic hypothermia. Results indicated that EPO did not significantly improve neurodevelopmental outcomes at 2 years compared to therapeutic hypothermia alone.

Background

Neonatal hypoxic-ischemic encephalopathy (HIE) is a significant cause of neurological impairment in newborns, with moderate to severe cases occurring in 1.05 per 1000 term infants in Australia and New Zealand. Current management primarily involves therapeutic hypothermia, yet many infants still experience mortality or neurodevelopmental issues.

Data Highlights

No significant numerical data was provided in the source material.

Key Findings

  • The PAEAN trial was a phase 3, double-blinded, placebo-controlled study involving 313 infants across Australia, New Zealand, and Singapore.
  • Eligibility included infants born at 35+0 weeks’ gestation or later with indicators of birth depression and moderate or severe encephalopathy.
  • High-dose EPO was administered during the first week of life to assess its impact on neurodevelopmental outcomes at 2 years.
  • The trial found no significant reduction in death or moderate/severe disability at 2 years compared to therapeutic hypothermia alone.
  • No definite safety signal was reported in the EPO group.
  • Results align with findings from the HEAL trial, which also did not demonstrate efficacy of EPO in this context.

Clinical Implications

The findings indicate that high-dose erythropoietin does not provide additional benefits to therapeutic hypothermia in improving outcomes for infants with HIE.

Conclusion

The PAEAN trial found that erythropoietin does not enhance neurodevelopmental outcomes in infants with HIE receiving therapeutic hypothermia.

Related Resources & Content

  1. JAMA Pediatrics, 2026 -- Erythropoietin for Neonatal Hypoxic-Ischemic Encephalopathy: A Randomized Clinical Trial
  2. NEJM, 2022 -- Trial of Erythropoietin for Hypoxic–Ischemic Encephalopathy in Newborns
  3. AAP, 2025 -- Part 5: Neonatal Resuscitation: 2025 American Heart Association and American Academy of Pediatrics Guidelines for Cardiopulmonary Resuscitation and Emergency Cardiovascular Care
  4. ASCO Publications — Randomized phase III trial evaluating the role of erythropoietin in the prevention of chemotherapy-induced anemia.
  5. ASCO Publications — Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human Erythropoietin, Epoetin Beta, in Hematologic Malignancies
  6. ASCO Publications — Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human Erythropoietin, Epoetin Beta, in Hematologic Malignancies
  7. ASCO Publications — Erythropoietin use in medically underserved patients (pts)
  8. Part 5: Neonatal Resuscitation: 2025 American Heart Association and American Academy of Pediatrics Guidelines for Cardiopulmonary Resuscitation and Emergency Cardiovascular Care
  9. Trial of Erythropoietin for Hypoxic–Ischemic Encephalopathy in Newborns | New England Journal of Medicine
  10. Erythropoietin for Neonatal Hypoxic-Ischemic Encephalopathy: A Randomized Clinical Trial | Neurology | JAMA Pediatrics | JAMA Network
  11. Evaluating the Safety and Efficacy of Erythropoietin Therapy for Neonatal Hypoxic-Ischemic Encephalopathy: A Systematic Review and Meta-Analysis.

Original Source(s)

Erythropoietin for Neonatal Hypoxic-Ischemic Encephalopathy: A Randomized Clinical Trial

  • by Helen G. Liley, Rod W. Hunt, Rachel L. O’Connell, Malcolm R. Battin, Iona Novak, Yvonne W. Wu, Roberta Ballard, Lisa Askie, Nadia Badawi, Alpana Ghadge, Susan E. Jacobs, Sandra E. Juul, Lucille Sebastian, Deepika Wagh, R. John Simes, PAEAN Study Investigators, Shannon Clough, Jennifer Bowen, Paul Colditz, Girsih Deshpande, Lisa Gold, Wendy Hague, Kei Lui, Dominic Wilkinson, Nicola Austin, Sarah Bellhouse, Max Berry, Clare Collins, Amanda Dyson, Antonio G de Paoli, Koert de Waal, Bevan Headley, Leah Hickey, James Holberton, David Hou, Alison L Kent, Vinayak Kodur, Richard Mausling, Scott Morris, Arun Nair, David Osborn, Victor Samuel Rajadurai, Nadia Schmidt, Jason Tan, Mark B Tracy, Flora Y Wong, Sarah Finlayson, Shannon Hunt, Carbo Yeung, Laurence Ralston

  • October 1, 2026

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