Nystatin prophylaxis and treatment in severely immunodepressed patients



Nystatin is sometimes used prophylactically in patients with severe immunodeficiency or in the treatment of fungal infection in such patients, although its effect seems to be equivocal.


To study whether nystatin decreases morbidity and mortality when given prophylactically or therapeutically to patients with severe immunodeficiency.

Search methods

We searched PubMed from 1966 to 7 July 2014 and the reference lists of identified articles.

Selection criteria

Randomised clinical trials comparing nystatin with placebo, an untreated control group, fluconazole or amphotericin B.

Data collection and analysis

Data on mortality, invasive fungal infection and colonisation were independently extracted by both authors. A random‐effects model was used unless the P value was greater than 0.10 for the test of heterogeneity.

Main results

We included 14 trials (1569 patients). The drugs were given prophylactically in 12 trials and as treatment in two. Eleven trials were in acute leukaemia, solid cancer, or bone marrow recipients; one in liver transplant patients; one in critically ill surgical and trauma patients; and one in AIDS patients. Nystatin was compared with placebo in three trials, with fluconazole in 10, and amphotericin B in one; the dose varied from 0.8 MIE to 72 MIE daily and was 2 mg/kg/d in a liposomal formulation. The effect of nystatin was similar to that of placebo on fungal colonisation (relative risk (RR) 0.85, 95% confidence interval (CI) 0.65 to 1.13). There was no statistically significant difference between fluconazole and nystatin on mortality (RR 0.75, 95% CI 0.54 to 1.03) whereas fluconazole was more effective in preventing invasive fungal infection (RR 0.40, 95% CI 0.17 to 0.93) and colonisation (RR 0.50, 95% CI 0.36 to 0.68). There were no proven fungal infections in a small trial that compared amphotericin B with liposomal nystatin. The results were very similar if the three studies that were not performed in cancer patients were excluded. For the 2011 and 2014 updates no additional trials were identified for inclusion.

Authors' conclusions

Nystatin cannot be recommended for prophylaxis or the treatment of Candida infections in immunodepressed patients.


Peter C Gøtzsche, Helle Krogh Johansen


Plain language summary

Prevention and treatment of fungal infections with nystatin in severely immunodepressed patients

People on chemotherapy for cancer, receiving a transplant or with AIDS are at risk of fungal infections. These infections can be life‐threatening, especially when they spread throughout the body. Nystatin is sometimes given as a routine preventive measure or as treatment in these patients. The review found that nystatin was no better than placebo (no treatment).


Peter C Gøtzsche, Helle Krogh Johansen

Reviewer's Conclusions

Authors' conclusions 

Implications for practice 

The effect of nystatin given orally to immunodepressed patients was no better than that of placebo, whereas it was inferior to the effect of fluconazole. Nystatin cannot be recommended for prophylaxis or treatment of Candida infections in immunodepressed patients.

Implications for research 

There seems to be little scope for further trials of nystatin given orally to immunodepressed patients since more effective antifungal agents exist, and since the effect of nystatin was at placebo level.

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