Cost-effectiveness analysis of antifungal prophylaxis in patients undergoing hematopoietic stem cell transplantation

Background: Micafungin sodium is indicated for the prophylaxis of Candida infections in patients undergoing hematopoietic stem cell transplantation (HSCT). One Phase III, multi-institutional, randomized, doubleblind comparative trial involving 882 adult and pediatric patients found that micafungin was more effective, in terms of significantly lower rates of systemic fungal infections and empiric antifungal therapy (AFT), than fluconazole as antifungal prophylaxis during the neutropenic phase following HSCT. Thus, despite the higher cost of micafungin versus fluconazole, micafungin prophylaxis may be associated with reduced costs. Objective: The aim of this analysis was to determine the cost-effectiveness of micafungin prophylaxis compared with fluconazole prophylaxis in patients undergoing HSCT. Methods: Efficacy data were taken from the clinical study. The economic analysis was conducted from the hospital perspective, using costs incurred from admission through discharge. Each of the patients was assigned costs and effectiveness based on outcomes data from the clinical study. Published literature was used to estimate hospital costs associated with HSCT and prophylaxis, empiric AFT, and treatment of a probable or proven Candida or Aspergillus infection. Mean costs and effectiveness were calculated in each treatment group. To test the variability of the results using repeated sampling, a bootstrapping analysis was also conducted, with 1000 simulations of random samples of 100 patients from each treatment group. If appropriate to describe the results, incremental costeffectiveness ratios were calculated, and sensitivity analyses were conducted by varying components of cost. Results: This analysis included data from 882 patients (527 males, 355 females; micafungin, 425 patients, mean age, 43.2 years [range, 0.6–73.0 years]; fluconazole, 457 patients, mean age, 41.9 years [range, 0.6–71.0 years]). Total hospital costs per patient were $121,098 and $124,957 in micafungin and fluconazole recipients, respectively—a difference of $3859. The bootstrapping analysis found that micafungin prophylaxis was cost-saving in 72.4% of the samples compared with 9.2% with fluconazole prophylaxis. Sensitivity analyses on estimated hospital costs found that micafungin was a cost-effective therapy. Conclusion: In this analysis of data from a clinical study in adults and children undergoing HSCT, micafungin prophylaxis was associated with reduced hospital costs, and resultant total patient costs, compared with fluconazole prophylaxis. (Clin Ther. 2008;30: 964–973) © 2008 Excerpta Medica Inc.