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Off-label recombinant factor VIIa use and thrombosis in children: a multi-center cohort study.
J Pediatr. 2011 May; 158(5):820-825.e1.JPed

Abstract

OBJECTIVE

To describe the off-label use of recombinant factor VIIa (rFVIIa) in tertiary care pediatric hospitals across the United States and to assess thrombotic events.

STUDY DESIGN

A retrospective multi-center cohort study using the Pediatric Health Information System administrative database. Children 18 years of age or younger who received rFVIIa between 2000 and 2007 were included. A label admission was defined as an admission with an International Classification of Diseases diagnostic code for hemophilia or factor VII deficiency; admissions without these codes were classified as off-label.

RESULTS

There were 4942 rFVIIa admissions, representing 3764 individual subjects; 74% (3655) of the admissions were off-label. There was a 10-fold increase in the annual rate of off-label admissions from 2000 to 2007 (from 2 to 20.8 per 10 000 hospital admissions, P < .001). The mortality rate in the off-label group was 34% (1258/3655). Thrombotic events occurred in 10.9% (399/3655) of the off-label admissions.

CONCLUSIONS

The off-label use of rFVIIa in hospitalized children is increasing rapidly despite the absence of adequate clinical trials demonstrating safety and efficacy. Thrombotic events are common and mortality is high among patients receiving off-label rFVIIa. Further studies are warranted to determine whether these adverse events are attributable to rFVIIa.

Authors+Show Affiliations

Division of Hematology, The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. witmer@email.chop.eduNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Journal Article
Multicenter Study

Language

eng

PubMed ID

21146180

Citation

Witmer, Char M., et al. "Off-label Recombinant Factor VIIa Use and Thrombosis in Children: a Multi-center Cohort Study." The Journal of Pediatrics, vol. 158, no. 5, 2011, pp. 820-825.e1.
Witmer CM, Huang YS, Lynch K, et al. Off-label recombinant factor VIIa use and thrombosis in children: a multi-center cohort study. J Pediatr. 2011;158(5):820-825.e1.
Witmer, C. M., Huang, Y. S., Lynch, K., Raffini, L. J., & Shah, S. S. (2011). Off-label recombinant factor VIIa use and thrombosis in children: a multi-center cohort study. The Journal of Pediatrics, 158(5), 820-e1. https://doi.org/10.1016/j.jpeds.2010.10.038
Witmer CM, et al. Off-label Recombinant Factor VIIa Use and Thrombosis in Children: a Multi-center Cohort Study. J Pediatr. 2011;158(5):820-825.e1. PubMed PMID: 21146180.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Off-label recombinant factor VIIa use and thrombosis in children: a multi-center cohort study. AU - Witmer,Char M, AU - Huang,Yuan-Shung, AU - Lynch,Kevin, AU - Raffini,Leslie J, AU - Shah,Samir S, Y1 - 2010/12/10/ PY - 2010/03/19/received PY - 2010/09/09/revised PY - 2010/10/26/accepted PY - 2010/12/15/entrez PY - 2010/12/15/pubmed PY - 2011/6/17/medline SP - 820 EP - 825.e1 JF - The Journal of pediatrics JO - J Pediatr VL - 158 IS - 5 N2 - OBJECTIVE: To describe the off-label use of recombinant factor VIIa (rFVIIa) in tertiary care pediatric hospitals across the United States and to assess thrombotic events. STUDY DESIGN: A retrospective multi-center cohort study using the Pediatric Health Information System administrative database. Children 18 years of age or younger who received rFVIIa between 2000 and 2007 were included. A label admission was defined as an admission with an International Classification of Diseases diagnostic code for hemophilia or factor VII deficiency; admissions without these codes were classified as off-label. RESULTS: There were 4942 rFVIIa admissions, representing 3764 individual subjects; 74% (3655) of the admissions were off-label. There was a 10-fold increase in the annual rate of off-label admissions from 2000 to 2007 (from 2 to 20.8 per 10 000 hospital admissions, P < .001). The mortality rate in the off-label group was 34% (1258/3655). Thrombotic events occurred in 10.9% (399/3655) of the off-label admissions. CONCLUSIONS: The off-label use of rFVIIa in hospitalized children is increasing rapidly despite the absence of adequate clinical trials demonstrating safety and efficacy. Thrombotic events are common and mortality is high among patients receiving off-label rFVIIa. Further studies are warranted to determine whether these adverse events are attributable to rFVIIa. SN - 1097-6833 UR - https://www.unboundmedicine.com/medline/citation/21146180/Off_label_recombinant_factor_VIIa_use_and_thrombosis_in_children:_a_multi_center_cohort_study_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0022-3476(10)00948-0 DB - PRIME DP - Unbound Medicine ER -