Summary
Background: Since the introduction of exogenous factor VIII therapy, several studies
have explored the clinical benefits of prophylactic use of factor VIII. Little research,
though, has focused on the economic aspects of this regimen. We conducted a cost analysis
using data from the Orthopedic Outcomes Study, a prospective, cross-national study
of the clinical outcomes associated with different patterns of factor VIII utilization
to examine the health care costs incurred and expenditures averted in patients receiving
on-demand versus prophylactic use of factor VIII in hemophilia. Methods and Analysis:
831 patients with severe hemophilia aged 1 to 31 years, from 19 centers around the
world were included in the cost analysis. Patients were categorized into three groups
according to the number of weeks during the study years in which they received prophylactic
regimens of factor VIII. For each subject, we estimated the costs of hospitalization,
surgery, days lost from school or work, and factor VIII utilization. Costs were then
stratified by age and by joint score to assess confounding, and a multivariate model
developed to determine the relationship between use of factor VIII prophylaxis and
total costs, while controlling for potential confounders.
Results: Patients who received factor VIII episodically incurred substantially greater
disability-related costs (days lost from school or work, days hospitalized due to
hemophilia, surgery) than patients who received factor VIII prophylactically for some
or all of the study period. For all treatment regimens, most disability-related costs
were accounted for by hospitalization for hemophilia-related conditions. The cost
of factor VIII itself was substantial in all treatment categories but was highest
among patients who received year-round prophylaxis, exceeding the savings resulting
from reduced disability and other health care expenditures.
Conclusions: Reductions in non-factor health care costs and disability associated
with prophylactic use of factor VIII in hemophilia were substantial and helped somewhat
to offset the much higher costs of this regimen. For certain subgroups, frequent episodic
treatment may be more expensive than full-time prophylaxis. However, because of the
very high cost of year-round prophylactic use of factor VIII, total health care expenditures
were highest among patients receiving this therapeutic regimen. However, because prophylaxis
clearly offers important clinical benefits, this approach may be warranted on medical
rather than economic grounds.
