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Published on: August 25, 2014
Response to Therapeutic Interventions in the NICU: Role of Sex as a Biological Variable
Rose Albert1, Albertina Lee1, Krithika Lingappan1
1Division of Neonatology and Department of Pediatrics; Children's Hospital of Philadelphia and University of Pennsylvania, Philadelphia, PA.
Insights
Biological sex significantly impacts preterm infant health and treatment responses. Analyzing outcomes by sex is crucial for personalized neonatal medicine and improving therapeutic strategies.
Area of Science:
- Neonatal Medicine
- Pediatric Research
- Biological Sex Differences
Background:
- Biological sex is a critical factor in disease pathophysiology and treatment response.
- Sex-specific differences are observed in prematurity-related outcomes and neonatal therapies.
- Limited reporting of neonatal study outcomes stratified by sex hinders understanding.
Purpose of the Study:
- To highlight the importance of sex as a biological variable in neonatal research.
- To emphasize the need for sex-disaggregated data in clinical and translational studies.
- To advocate for reporting outcomes by biological sex in neonates.
Main Methods:
- Review of existing literature on sex differences in neonatal outcomes.
- Analysis of the impact of biological sex on prematurity-related conditions.
- Assessment of therapeutic responses stratified by biological sex.
Main Results:
- Preterm infants show sex-specific differences in disease predisposition and resilience.
- Neonatal therapies (e.g., steroids, indomethacin) elicit different responses based on sex.
- Most neonatal studies collect sex data but do not report outcomes by sex.
Conclusions:
- Sex-specific differences in neonates necessitate tailored medical approaches.
- Disaggregating neonatal study data by sex is essential for precision medicine.
- Future research and meta-analyses should prioritize sex-stratified outcome reporting.
Abstract:
Sex as a biological variable plays a critical role in the pathophysiology of specific diseases and can have a potential impact on the response to therapies and disease outcomes. Sex-specific differences have been reported in prematurity-related outcomes, suggesting that preterm infants exhibit differences in biological predisposition or resilience to disease. Furthermore, striking differences in response to common neonatal therapies such as antenatal and postnatal steroids, indomethacin, and other nonpharmacologic agents raise the critical need to assess therapeutic responses stratified by biological sex. Very few clinical and translational studies in neonates report outcomes by sex, even though most account for biological sex at enrollment. Sex-specific differences in the newborn may arise from baseline or adaptive differences in male and female preterm neonates. In the current era of precision medicine and the increasing interest in tailoring risk-based therapy to patients, data from neonatal clinical studies should be disaggregated by sex and reported for informing studies with a larger sample size or meta-analyses.
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