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Antithrombin concentrate use in children: a multicenter cohort study
Trisha E Wong1, Yuan-Shung Huang, Jason Weiser
1Puget Sound Blood Center, Seattle, WA; Division of Hematology/Oncology, Department of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA.
Insights
Off-label use of antithrombin concentrate in children has rapidly increased, with 97% of patients receiving it for unapproved indications. This trend is particularly noted in neonates and critically ill children undergoing extracorporeal membrane oxygenation.
Area of Science:
- Pediatric pharmacology
- Critical care medicine
- Hematology
Background:
- Antithrombin concentrate is approved for specific thrombotic conditions.
- Its use in pediatric populations, especially off-label, requires further investigation.
- Tertiary care pediatric hospitals are key settings for complex pediatric cases.
Purpose of the Study:
- To describe the patterns of off-label antithrombin concentrate use in US pediatric hospitals.
- To identify patient demographics and clinical characteristics associated with off-label antithrombin concentrate use.
- To analyze trends in off-label antithrombin concentrate utilization over a decade.
Main Methods:
- Retrospective, multicenter cohort study.
- Utilized the Pediatric Health Information System database (2002-2011).
- Included 4210 admissions of children (<18 years) receiving antithrombin concentrate; off-label use defined by absence of ICD codes for primary hypercoagulable states.
Main Results:
- Off-label antithrombin concentrate use increased fivefold over the study period.
- 97% of patients received antithrombin concentrate off-label.
- Largest age group for use was neonates (45.7%); 87% had complex chronic conditions; extracorporeal membrane oxygenation (43.7%) was the most common associated procedure.
Conclusions:
- Off-label antithrombin concentrate use is rapidly increasing in critically ill children, especially those on extracorporeal membrane oxygenation.
- Limited data exist on the safety and efficacy of off-label antithrombin concentrate use.
- Judicious use and adherence to hospital guidelines are recommended pending further research.
Objective:
To describe the off-label use of antithrombin concentrate in tertiary care pediatric hospitals across the US.
Study Design:
This is a retrospective, multicenter, cohort study of 4210 admissions of children younger than 18 years of age who received antithrombin concentrate between 2002 and 2011 within the Pediatric Health Information System administrative database. An on-label admission was defined as an admission with an International Classification of Diseases diagnostic code for a primary hypercoagulable state; admissions without this code were classified as off-label.
Results:
During the 10-year study period, off-label use of antithrombin concentrate increased 5-fold. Overall, 97% of study subjects received antithrombin off-label. Neonates younger than 30 days of age comprised the largest age group (45.7%) of use; 87% of patients had at least one complex chronic condition, with congenital heart/lung defects being the most prevalent primary diagnosis (36.3%). Extracorporeal membrane oxygenation was the most common procedure associated with antithrombin use (43.7%).
Conclusions:
The off-label use of antithrombin concentrate is increasing rapidly, particularly in critically ill children receiving extracorporeal membrane oxygenation, with few parallel studies to substantiate its safety or efficacy. Further preclinical and controlled clinical studies are critical to expanding our knowledge of this drug. In the meantime, antithrombin concentrate should be used judiciously by clinicians and following guidelines instated by hospitals.
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