In a new study, researchers at Children’s Hospital of Philadelphia found that hydrocortisone treatment did not improve or harm functional outcomes at school age in preterm infants at high risk for bronchopulmonary dysplasia (BPD). While more research is needed, the results, reported today in the Journal of the American Medical Association Pediatrics, suggest that when hydrocortisone is started 2-4 weeks after birth in intubated preterm infants, it does not provide long-term benefits or cause harm for this population. However, the authors said that it is still possible that administering the same medication at an alternative time or with varying dosages might offer benefits to patients.
Infant chronic lung disease or BPD is a prevalent and serious complication in premature infants. Approximately 50% of extremely preterm infants develop BPD during their hospital stay, leading to substantial long-term health issues affecting the lungs, heart, growth, and neurodevelopment. Although prevention has been extensively studied, there is limited data on effective care strategies for infants who are developing BPD or are already diagnosed with BPD. Researchers continue to improve treatments to help these children, and their families have a better quality of life as a result.
In this long-term study, researchers examined the effects of neonatal hydrocortisone treatment versus a placebo on preterm infants at high risk for BPD. The study focused on determining if hydrocortisone could improve outcomes related to motor skills, cognition, academics, and lung development. It involved 800 infants born before 30 weeks of gestation who required mechanical ventilation. These infants were randomly assigned to receive either a 10-day course of hydrocortisone or a placebo, with follow-up visits conducted from 2017 to 2024, when the children were 5-7 years old.
The researchers found no significant difference in overall functional impairment between the hydrocortisone and placebo groups (71.3% vs. 73.3%). The components of functional impairment – cognitive, motor, academic delay, and exercise capacity – showed no significant differences between the two groups. Motor delay was the most common impairment observed. The only notable difference was lower body weight in the hydrocortisone group.
“Our findings provide valuable data on the long-term safety and efficacy of hydrocortisone in very preterm infants, contributing to the broader understanding of postnatal steroid therapies and their impact on developmental outcomes,” said Sara B. DeMauro, MD, MSCE, a senior study author and an attending neonatologist with the Division of Neonatology and Program Director of the Neonatal Follow-up Program at Children's Hospital of Philadelphia. “Further research may explore alternative treatments or interventions to support this vulnerable population.”
The research was supported by the National Institutes of Health and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (U10 HD21373, UG1 HD21364, UG1 HD21385, UG1 HD27851, UG1 HD27853, UG1 HD27856, UG1 HD27880,UG1 HD27904, UG1 HD34216, UG1HD36790, UG1 HD40492, UG1 HD40689, UG1 HD53089, UG1 HD53109, UG1 HD68244, UG1HD68270, UG1 HD68278, UG1 HD68263, UG1 HD68284; UG1 HD87226, UG1 HD87229) and the National Center for Advancing Translational Sciences (NCATS) (UL1 TR6, UL1 TR41, UL1 TR42, UL1 TR77, UL1 TR93, UL1 TR105, UL1 TR442, UL1 TR454, UL1 TR1117), provided grant support for the Neonatal Research Network, including for the Hydrocortisone for BPD trial. The National Heart, Lung, and Blood Institute (NHLBI) provided grant support for the HYBRiD Outcomes Study (UH3HL137872, U24HL137729).
DeMauro et al. “Early School Age Functional Outcomes of Preterm-Born Children Treated with Hydrocortisone vs. Placebo.” JAMA. Online December 8, 2025. DOI: 10.1001/jamapediatrics.2025.4801.
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In a new study, researchers at Children’s Hospital of Philadelphia found that hydrocortisone treatment did not improve or harm functional outcomes at school age in preterm infants at high risk for bronchopulmonary dysplasia (BPD). While more research is needed, the results, reported today in the Journal of the American Medical Association Pediatrics, suggest that when hydrocortisone is started 2-4 weeks after birth in intubated preterm infants, it does not provide long-term benefits or cause harm for this population. However, the authors said that it is still possible that administering the same medication at an alternative time or with varying dosages might offer benefits to patients.
Infant chronic lung disease or BPD is a prevalent and serious complication in premature infants. Approximately 50% of extremely preterm infants develop BPD during their hospital stay, leading to substantial long-term health issues affecting the lungs, heart, growth, and neurodevelopment. Although prevention has been extensively studied, there is limited data on effective care strategies for infants who are developing BPD or are already diagnosed with BPD. Researchers continue to improve treatments to help these children, and their families have a better quality of life as a result.
In this long-term study, researchers examined the effects of neonatal hydrocortisone treatment versus a placebo on preterm infants at high risk for BPD. The study focused on determining if hydrocortisone could improve outcomes related to motor skills, cognition, academics, and lung development. It involved 800 infants born before 30 weeks of gestation who required mechanical ventilation. These infants were randomly assigned to receive either a 10-day course of hydrocortisone or a placebo, with follow-up visits conducted from 2017 to 2024, when the children were 5-7 years old.
The researchers found no significant difference in overall functional impairment between the hydrocortisone and placebo groups (71.3% vs. 73.3%). The components of functional impairment – cognitive, motor, academic delay, and exercise capacity – showed no significant differences between the two groups. Motor delay was the most common impairment observed. The only notable difference was lower body weight in the hydrocortisone group.
“Our findings provide valuable data on the long-term safety and efficacy of hydrocortisone in very preterm infants, contributing to the broader understanding of postnatal steroid therapies and their impact on developmental outcomes,” said Sara B. DeMauro, MD, MSCE, a senior study author and an attending neonatologist with the Division of Neonatology and Program Director of the Neonatal Follow-up Program at Children's Hospital of Philadelphia. “Further research may explore alternative treatments or interventions to support this vulnerable population.”
The research was supported by the National Institutes of Health and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (U10 HD21373, UG1 HD21364, UG1 HD21385, UG1 HD27851, UG1 HD27853, UG1 HD27856, UG1 HD27880,UG1 HD27904, UG1 HD34216, UG1HD36790, UG1 HD40492, UG1 HD40689, UG1 HD53089, UG1 HD53109, UG1 HD68244, UG1HD68270, UG1 HD68278, UG1 HD68263, UG1 HD68284; UG1 HD87226, UG1 HD87229) and the National Center for Advancing Translational Sciences (NCATS) (UL1 TR6, UL1 TR41, UL1 TR42, UL1 TR77, UL1 TR93, UL1 TR105, UL1 TR442, UL1 TR454, UL1 TR1117), provided grant support for the Neonatal Research Network, including for the Hydrocortisone for BPD trial. The National Heart, Lung, and Blood Institute (NHLBI) provided grant support for the HYBRiD Outcomes Study (UH3HL137872, U24HL137729).
DeMauro et al. “Early School Age Functional Outcomes of Preterm-Born Children Treated with Hydrocortisone vs. Placebo.” JAMA. Online December 8, 2025. DOI: 10.1001/jamapediatrics.2025.4801.
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