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Drugs to Prevent Bronchopulmonary Dysplasia: Effect of Baseline Risk on the Number Needed to Treat

Erik A Jensen1, Robin S Roberts2, Barbara Schmidt3

  • 1Division of Neonatology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

The Journal of Pediatrics
|March 8, 2020
PubMed

Insights

This study presents a visual tool to help clinicians understand the benefits of treatments like caffeine, vitamin A, and hydrocortisone for preventing bronchopulmonary dysplasia (BPD) in very preterm infants.

Area of Science:

  • Neonatal Medicine
  • Pediatric Pulmonology
  • Clinical Pharmacology

Background:

  • Bronchopulmonary dysplasia (BPD) is a significant risk for infants born very preterm.
  • The baseline risk of BPD varies considerably among these vulnerable infants.
  • Evidence-based drug therapies exist to potentially prevent BPD.

Purpose of the Study:

  • To develop a visual aid for understanding the efficacy of BPD preventative therapies.
  • To illustrate the "number needed to treat" (NNT) for key drugs across varying baseline BPD risks.
  • To aid clinical decision-making regarding BPD prevention strategies.

Main Methods:

  • Designed a visual aid tool.
  • Incorporated data on "number needed to treat" (NNT) with confidence intervals (CIs).
  • Displayed NNT for caffeine, vitamin A, and hydrocortisone.

Main Results:

  • The visual aid effectively demonstrates how NNT for BPD prevention changes with baseline risk.
  • Caffeine, vitamin A, and hydrocortisone show varying treatment efficacies depending on the infant's initial risk.
  • Confidence intervals highlight the precision of these treatment effect estimates.

Conclusions:

  • The developed visual aid facilitates personalized risk-benefit assessments for BPD prevention.
  • Clinicians can better utilize evidence-based therapies by considering individual infant baseline risk.
  • This tool supports informed choices in managing very preterm infants at risk for BPD.

Keywords:
NNTbaseline riskcaffeinechronic lung diseaseevidence-based medicinehydrocortisonevitamin A

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