• Sandra E Juul, Bryan A Comstock, Rajan Wadhawan, Dennis E Mayock, Sherry E Courtney, Tonya Robinson, Kaashif A Ahmad, Ellen Bendel-Stenzel, Mariana Baserga, Edmund F LaGamma, L Corbin Downey, Raghavendra Rao, Nancy Fahim, Andrea Lampland, Ivan D Frantz, Janine Y Khan, Michael Weiss, Maureen M Gilmore, Robin K Ohls, Nishant Srinivasan, Jorge E Perez, Victor McKay, Phuong T Vu, Jean Lowe, Karl Kuban, T Michael O'Shea, Adam L Hartman, Patrick J Heagerty, and PENUT Trial Consortium.
• From the University of Washington, Seattle (S.E.J., B.A.C., D.E.M., P.T.V., P.J.H.); Florida Hospital Orlando, Orlando (R.W.), the University of Florida, Gainesville (M.W.), South Miami Hospital, South Miami (J.E.P.), and Johns Hopkins All Children's Hospital, St. Petersburg (V.M.) - all in Florida; the University of Arkansas for Medical Sciences, Little Rock (S.E.C.); the University of Louisville, Louisville, KY (T.R.); Methodist Children's Hospital, San Antonio, TX (K.A.A.); Children's Hospital and Clinics of Minnesota (E.B.-S.) and University of Minnesota Masonic Children's Hospital (R.R., N.F.), Minneapolis, and Children's Minnesota, St. Paul (A.L.) - all in Minnesota; the University of Utah, Salt Lake City (M.B.); Maria Fareri Children's Hospital at Westchester Medical Center, Valhalla, NY (E.F.L.); Wake Forest School of Medicine, Winston-Salem (L.C.D.), and the University of North Carolina, Chapel Hill (T.M.O.) - both in North Carolina; Beth Israel Deaconess Medical Center (I.D.F.) and Boston University (K.K.) - both in Boston; Prentice Women's Hospital (J.Y.K.) and Children's Hospital of the University of Illinois (N.S.) - both in Chicago; Johns Hopkins University, Baltimore (M.M.G.), and the National Institute of Neurological Disorders and Stroke, Bethesda (A.L.H.) - both in Maryland; and the University of New Mexico, Albuquerque (R.K.O., J.L.).
• N. Engl. J. Med. 2020 Jan 16; 382 (3): 233243233-243.

BackgroundHigh-dose erythropoietin has been shown to have a neuroprotective effect in preclinical models of neonatal brain injury, and phase 2 trials have suggested possible efficacy; however, the benefits and safety of this therapy in extremely preterm infants have not been established.MethodsIn this multicenter, randomized, double-blind trial of high-dose erythropoietin, we assigned 941 infants who were born at 24 weeks 0 days to 27 weeks 6 days of gestation to receive erythropoietin or placebo within 24 hours after birth. Erythropoietin was administered intravenously at a dose of 1000 U per kilogram of body weight every 48 hours for a total of six doses, followed by a maintenance dose of 400 U per kilogram three times per week by subcutaneous injection through 32 completed weeks of postmenstrual age. Placebo was administered as intravenous saline followed by sham injections. The primary outcome was death or severe neurodevelopmental impairment at 22 to 26 months of postmenstrual age. Severe neurodevelopmental impairment was defined as severe cerebral palsy or a composite motor or composite cognitive score of less than 70 (which corresponds to 2 SD below the mean, with higher scores indicating better performance) on the Bayley Scales of Infant and Toddler Development, third edition.ResultsA total of 741 infants were included in the per-protocol efficacy analysis: 376 received erythropoietin and 365 received placebo. There was no significant difference between the erythropoietin group and the placebo group in the incidence of death or severe neurodevelopmental impairment at 2 years of age (97 children [26%] vs. 94 children [26%]; relative risk, 1.03; 95% confidence interval, 0.81 to 1.32; P = 0.80). There were no significant differences between the groups in the rates of retinopathy of prematurity, intracranial hemorrhage, sepsis, necrotizing enterocolitis, bronchopulmonary dysplasia, or death or in the frequency of serious adverse events.ConclusionsHigh-dose erythropoietin treatment administered to extremely preterm infants from 24 hours after birth through 32 weeks of postmenstrual age did not result in a lower risk of severe neurodevelopmental impairment or death at 2 years of age. (Funded by the National Institute of Neurological Disorders and Stroke; PENUT ClinicalTrials.gov number, NCT01378273.).Copyright © 2020 Massachusetts Medical Society.

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