A phase 2, open-label study of the safety and efficacy of intravenous anidulafungin as a treatment for azole-refractory mucosal candidiasis.

BACKGROUND

Azole-refractory mucosal candidiasis is a debilitating disease frequently seen in patients who are immunosuppressed as a result of HIV, malignancy, posttransplant immunosuppressive therapy, persistent neutropenia, steroid use, or diabetes. Anidulafungin has potent activity against a broad spectrum of Candida species, including strains resistant to azoles and amphotericin B. We performed an open-label, noncomparative study to examine efficacy and safety of anidulafungin in patients with azole-refractory oropharyngeal and esophageal candidiasis.

METHODS

Patients enrolled met diagnostic criteria for azole-refractory mucosal candidiasis. They received intravenous anidulafungin 100 mg on day 1 followed by daily 50-mg doses on day 2 through day 14 or for a maximum of 21 days. Primary efficacy variables were clinical response (for oropharyngeal candidiasis) and endoscopic and clinical response (for esophageal candidiasis) at the end of therapy.

RESULTS

Nineteen patients were enrolled; 89% had advanced HIV infection. Clinical success was observed in 95% of patients at end of therapy, and endoscopic success was observed in 92% of patients with esophageal candidiasis. At follow-up, clinical success was maintained in 47% of patients. The most common adverse event, experienced by 4 patients, was nausea and/or vomiting.

CONCLUSIONS

Anidulafungin was well tolerated and efficacious in the treatment of patients with azole-refractory esophageal and oropharyngeal candidiasis.