A Multicenter, Open-Label Study of Posaconazole Oral Suspension in Treatment of Patients with Invasive Fungal Infection Refractory to or Intolerant of First-Line Therapy,

Invasive fungal infection represents one major health threat for patients with a compromised immune system.

We performed a multicenter, open-label clinical study of posaconazole oral suspension in the treatment of 63 patients with invasive fungal infection that was refractory to or intolerant of first-line therapy. The primary efficacy end point was the cumulative response rate at the end of posaconazole treatment. The safety end points were adverse events (AEs), laboratory test results, and vital sign measurements, as well as patients' rate of withdrawal because of AEs or laboratory abnormalities.

Of the 63 patients enrolled in the study, 62 (98.4%) were treated with study drug and included in the safety set. From those 62 patients, 59 patients entered the full analysis set for efficacy data assessment. A total of 47 patients were included in the per-protocol set. The cumulative clinical response rates at weeks 4, 8, and 12 of treatment were 69.49% (41/59, 95% confidence interval [CI]: 56.13–80.81), 62.71% (37/59, 95% CI: 49.15–74.96), and 64.41% (38/59, 95% CI: 50.87–76.45), respectively. Cumulative fungal pathogens eradication rates at week 4, week 8, and the end of treatment (week 12) were 47.06% (95% CI: 22.98–72.19), 47.06% (95% CI: 22.98–72.19), and 52.94% (95% CI: 27.81–77.02), respectively. At the end of treatment (week 12), 2 patients had died; the cumulative survival rate was 96.61% (95% CI: 88.29–99.59). The incidences of AEs, adverse reactions, and SAEs were 79.0%, 32.3%, and 8.1%, respectively. None of the serious adverse reactions were related to the study drug.

Posaconazole is effective in treating patients with refractory invasive fungal infection. As a novel broad-spectrum triazole antifungal with a good long-term safety profile, posaconazole can be used to treat patients with fungal infection relapse after antifungal treatment, patients with fungal infection resistant to routine antifungal treatment, patients with fungal infection intolerant of routine antifungal treatment due to side effects, and patients with organ dysfunction (e.g., renal dysfunction). (ClinicalTrials.gov number, NCT00811642)

No relevant conflicts of interest to declare.