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Updated: Oct 2, 2026
Measurement of Factor V Activity in Human Plasma Using a Microplate Coagulation Assay
Published on: September 9, 2012
Response to treatment and adverse events associated with use of recombinant activated factor VII in children: a
James D Cooper1, Arthur K Ritchey2
1Assistant Professor, Department of Pediatrics, Division of Hematology/Oncology/BMT, Children's Hospital of Pittsburgh of UPMC, 4401 Penn Avenue, Pittsburgh, PA 15224, USA.
Insights
Recombinant activated factor VII (rFVIIa) showed mixed results in children, successfully controlling bleeding in many cases but with a concerning 16.7% rate of unexpected thromboses. Further research is needed to clarify its risk-benefit profile in pediatric patients.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Clinical Outcomes Research
Background:
- Recombinant activated factor VII (rFVIIa) is FDA-approved for hemophilia with inhibitors and factor VII deficiency.
- Efficacy and safety data for off-label pediatric use of rFVIIa are less clear than in adults.
- Secondary thromboses have been reported in adults receiving rFVIIa.
Purpose of the Study:
- To analyze the characteristics and outcomes of rFVIIa use in a pediatric population.
- To evaluate the efficacy and adverse event profile of rFVIIa in children.
- To assess the rate of secondary thromboses associated with pediatric rFVIIa administration.
Main Methods:
- Retrospective analysis of electronic medical records at a children's hospital.
- Inclusion of all pediatric patient episodes treated with rFVIIa over 33 months.
- Data collection on patient diagnosis, laboratory values, concurrent treatments, adverse events, and outcomes.
Main Results:
- 66 patient episodes received rFVIIa, with gastrointestinal bleeding being the most common indication (36.4%).
- Only one patient received rFVIIa for an FDA-approved indication.
- Treatment failure occurred in 33.3% of bleeding control courses, associated with diffuse bleeding.
- Unexpected adverse thromboses developed in 16.7% of patients within one week of rFVIIa treatment.
Conclusions:
- rFVIIa was effective in controlling serious bleeding episodes in most pediatric cases reviewed.
- Efficacy was limited in patients with diffuse bleeding at the time of administration.
- The observed thrombosis rate of 16.7% is higher than anticipated, warranting further investigation into causality and the pediatric risk-benefit ratio.
Background:
Recombinant activated factor VII (rFVIIa) is United States (US) Food and Drug Administration (FDA)-approved for patients with hemophilia with inhibitors or congenital factor VII deficiency. Initial reports of off-label use highlighted its efficacy, though newer reports have not repeated these findings. In both types of publication, though, secondary thromboses have been seen in adult patients. The data in children are less clear.
Methods:
This study analyzed all rFVIIa use at a large children's hospital for characteristics and outcomes. Recipients of rFVIIa were identified retrospectively via the electronic medical record. Data on patient diagnosis, lab data, other treatments, adverse events, and outcomes were collected.
Results:
Over 33 months, 66 patient episodes were treated with a total of 606 doses (median = 2). The most common indication (36.4%) was gastrointestinal bleeding (24/66 patients). Only one patient received a dose for an approved labeled indication. For control of bleeding, 33.3% of courses were unsuccessful (19/57). Bleeding from multiple sites was associated with treatment failure. In 16.7% of patients (11/66), unexpected adverse thromboses developed within 1 week of completing a course of rFVIIa. Thromboses in both intra- and extra-corporeal sites were included if they compromised patient care.
Conclusions:
In the majority of cases reviewed, rFVIIa was successful in stopping or slowing serious bleeding episodes. It was least effective when a patient had diffuse bleeding at the time of administration. The thrombosis rate of 16.7% was higher than expected, though causality cannot be declared. Further investigation is needed to determine the risk-benefit ratio in children.
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