Introduction: Fungal infection is believed to be an important cause of morbidity and mortality in neutropenic patients. The diagnosis is difficult and the effect of treatment is uncertain.

Objective: To study whether antifungals are effective in patients with haematologic malignancies and neutropenia.

Methods: Randomised trials comparing antifungals vs no prevention or placebo were collected. A modified Cochrane search strategy was used. Data were extracted independently by two observers. Mortality was the primary outcome.

Results: Thirty studies were identified. Data were available in 24; amphotericin B was used in 7, fluconazole in 5, ketoconazole in 8, other antifungals in 4. The randomisation method was not fully described in any study. The median followup period was 56 days (not available in 11 studies). No significant difference was found in mortality (odds ratio 0.91, 95% CI 0.73-1.13, 197/1118 (17-6%) patients died in the treatment groups vs 212/1130 (18.8%) in the untreated groups; number of deaths was not available in 8 studies). Number of systemic fungal infections (odds ratio 0.48, 0.29-0.78), number of colonized patients (odds ratio 0.40, 0.26-0.63), and use of additional antifungal therapy (odds ratio 0-69, 0.52-0.90) were lower in the intervention groups.

Discussion: Antifungals have no effect on mortality, only on surrogate outcomes in patients with haematologic malignancies and neutropenia. Prevention with these drugs seems unwarranted but larger trials are needed.