PROPHYLACTIC USE OF FACTOR-VIII - AN ECONOMIC-EVALUATION

Background: Since the introduction of exogenous factor VIII therapy, s everal studies have explored the clinical benefits of prophylactic use of factor VLII. 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 VII I utilization to examine the health care costs incurred and expen ditu res averted in patients receiving on-demand versus prophylactic use of factor Vm in hemophilia. Methods and Analysis: 831 patients with seve re hemophilia aged I to 31 years, from 19 centers around the world wer e included in the cost analysis. Patients were categorized into three groups according to the number of weeks during the study years in whic h they received prophylactic regimens of factor VIII. For each subject , we estimated the casts of hospitalization, surgery, days lost from s chool or work, and factor VIII utilization. Costs were then stratified by age and by joint score to assess confounding, and a multivariate m odel developed to determine the relationship between use of factor Vm prophylaxis and total costs, while controlling for potential confounde rs. Results: Patients who received factor VIII episodically incurred s ubstantially greater disability-related tests (days lost from school o r work, days hospitalized due to hemophilia, surgery) than patients wh o received factor VIII prophylactically for some or all of die study p eriod. For all treatment regimens, most disability-related costs were account ed for by hospitalization for hemophilia-related conditions. T he cost of factor VIII itself was substantial in all treatment categor ies but was highest among patients who received year-round prophylaxis , exceeding the savings resulting from reduced disability and other he alth care expenditures. Conclusions: Reductions in non-factor health c are costs and disability associated with prophylactic use of factor VI II in hemophilia were substantial and helped somewhat to offset the mu ch higher costs of this regimen. For certain subgroups. frequent episo dic treatment may be more expensive than full-time prophylaxis. Howeve r, because of the very high cost of year-round prophylactic use of fac tor VIII, total health care expenditures were highest among patients r eceiving this therapeutic regimen. However, because prophylaxis clearl y offers important clinical benefits. this approach may be warranted o n medical rather than economic grounds.