Authors' objectives:

Neutropenic patients with fever that persists despite antibacterial treatment are suspected of having a fungal infection. Conventional amphotericin B may be used as empiric antifungal treatment of children with persistent febrile neutropenia, however there are concerns with its safety profile. Other antifungals are believed to have an improved safety profile, such as caspofungin and liposomal amphotericin B, however due to a higher cost, their use is often limited to circumstances where toxicity with conventional amphotericin B is a concern. There is currently a paucity of comparative clinical and economic evidence between caspofungin and other antifungals in children. Our objectives were to evaluate the efficacy, safety, and cost of caspofungin compared to conventional amphotericin B and liposomal amphotericin B in the empiric treatment of persistent febrile neutropenia in children.

Authors' results and conclusions: One pediatric RCT presented at a conference and one published adult RCT comparing caspofungin and liposomal amphotericin B in febrile neutropenia were identified. In addition, 8 non-comparative studies but no systematic review, meta-analysis, or economic analysis with caspofungin in pediatric patients were identified. The pediatric RCT included 82 patients, 56 and 26 in the caspofungin and liposomal amphotericin B groups respectively. The authors concluded that the two drugs had a similar rate of overall treatment response. There was a trend towards a lower rate of some adverse events when caspofungin was compared to liposomal amphotericin B (nephrotoxicity, 6% vs. 8%, and hypokalemia, 4% vs. 11%, respectively, among others). A trend towards a higher frequency of rash (9% vs. 0%) and headache (9% vs. 0) was observed for caspofungin compared to liposomal amphotericin B, respectively. The differences were not statistically significant. The costs of empiric antifungal treatment were estimated as CAN$2,503, $3,129 and CAN$1,470 for caspofungin, liposomal amphotericin B and conventional amphotericin B, respectively (14 days, 20 kg/0.0.79 m2 child). The probabilistic sensitivity analysis demonstrated a trend towards a mean cost saving of CAN$667 per patient for caspofungin compared to liposomal amphotericin B (95% confidence interval (CI) -CAN$3,221, + CAN$1,802) with a 68% probability that caspofungin is less costly than liposomal amphotericin B.

Authors' recommendations: Our analyses showed that there was a trend towards lower treatment costs with caspofungin compared to liposomal amphotericin B. Both caspofungin and liposomal amphotericin B present relatively high acquisition costs that may affect the hospital pharmacy budgets, especially if a large number of patients receive these drugs annually in a given institution. Conventional amphotericin B had lower drug acquisition costs however the monitoring, prevention, and treatment of amphotericin B-related complications may be more time and resource consuming compared to caspofungin and liposomal amphotericin B. Due to a lack of comparative data with caspofungin in pediatric patients, conventional amphotericin B could not be incorporated into the comparative analyses.

Authors' methods: Review

Project Status: Completed

Year Published: 2008

URL for published report: https://lab.research.sickkids.ca/task/wp-content/uploads/sites/66/2018/06/2008-01_Caspofungin-FULL-REPORT.pdf

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Canada

Organisation Name: Technology Assessment at SickKids

Contact Address: Program of Child Health Evaluative Sciences, The Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8 tel: (416) 813-8519 fax: (416) 813-5979

Contact Name: wendy.ungar@sickkids.ca

Contact Email: wendy.ungar@sickkids.ca

Copyright: Technology Assessment Unit of the Hospital for Sick Children (TASK)