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Patterns of Off-Label Prescribing in the Pediatric Intensive Care Unit and Prioritizing Future Research
Angela S Czaja1, Pamela D Reiter2, M Lynn Schultz3
1Division of Critical Care, Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colorado ; Critical Care, Children's Hospital Colorado, Aurora, Colorado ; Center for Pharmaceutical Outcomes Research, Aurora, Colorado.
Insights
Most pediatric intensive care unit patients receive off-label medications, increasing their length of stay and mortality. Research should prioritize high-impact drugs like dexmedetomidine and hydromorphone.
Area of Science:
- Pediatric critical care medicine
- Pharmacology
- Drug safety
Background:
- Off-label medication prescribing is common in pediatric intensive care units (PICUs).
- Evidence supporting the safety and efficacy of many off-label drug uses in critically ill children is limited.
- Understanding patterns of off-label use is crucial for improving patient outcomes.
Purpose of the Study:
- To characterize the prevalence of off-label prescribing in US PICUs.
- To identify patient and treatment factors associated with off-label medication use.
- To pinpoint high-priority medications requiring further clinical investigation.
Main Methods:
- Retrospective analysis of medication data from 39 children's hospitals in 2010.
- Identified off-label use as prescribing for patients younger than the FDA-approved age.
- Utilized hierarchical multivariable modeling to determine factors associated with off-label use.
Main Results:
- 85% of 66,896 PICU patients received at least one off-label medication.
- Off-label drug use was associated with longer PICU/hospital stays and increased mortality.
- Factors linked to off-label use included younger age, chronic conditions, organ failure, and mechanical ventilation.
Conclusions:
- The majority of critically ill children are exposed to medications without definitive evidence of efficacy or safety for their condition.
- High-impact medications, including dexmedetomidine, dopamine, and hydromorphone, were identified as priorities for future research.
- Targeted research on high-impact off-label drugs is essential to improve evidence-based care in PICUs.
Objectives:
To characterize off-label prescribing among US pediatric intensive care units (PICUs), determine characteristics associated with off-label use, and identify medications in highest need for additional study.
Methods:
Medications prescribed for ≥1% PICU patients (age < 18 years) in 2010 were identified from 39 children's hospitals. Use in a patient younger than the Food and Drug Administration (FDA)-approved age for any indication was considered off-label. Hierarchical multivariable modeling was used to identify characteristics associated with off-label use, accounting for center effects. Highest-impact drugs were defined by: 1) high off-label use (off-label use in at least 5% of the PICU cohort), 2) high risk medication, and 3) high priority status by the FDA or Best Pharmaceuticals for Children Act (BPCA).
Results:
A total of 66,896 patients received ≥1 medication of interest (n = 162) during their PICU stay. A median of 3 (interquartile range, 2-6) unique drugs per patient were used off-label. Those who received ≥1 drug off-label (85% of the cohort) had longer median PICU (2 days vs 1 day) and hospital (6 days vs 3 days) lengths of stay and higher mortality (3.6% vs 0.7%), p < 0.001. Factors independently associated with off-label drug use included: age 1 to 5 years, chronic conditions, acute organ failures, mechanical ventilation, arterial or venous catheters, dialysis, and blood products. Half of prescribed medications (n = 84) had been used off-label: 26 with significant off-label use, 30 high-risk medications, and 47 with high FDA/BPCA priority. The highest impact medications identified were: dexmedetomidine, dopamine, hydromorphone, ketamine, lorazepam, methadone, milrinone, and oxycodone.
Conclusions:
Most PICU patients are exposed to off-label medication use, with uncertain evidence. Future medication research in this population should focus on medications with high impact potential.
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