Antifungal therapy for febrile neutropenia: issues in clinical trial design

Abstract

Amphotericin B therapy for fever during neutropenia became a standard practice approximately 20 years ago. Treatment of febrile neutropenia has since become a common indication sought in the development of new antifungals, with several alternatives approved for 'empirical therapy' based on the results of randomized clinical trials. Recent studies have highlighted problematic issues in trial design and interpretation; inclusion criteria and the definition of success are two examples of controversial issues. Here, issues in trial design will be reviewed to address the question: Can (and should) new antifungals be evaluated for treatment of fever during neutropenia?