Ketoconazole is Effective against the Chronic Mucocutaneous Candidosis of Autoimmune Polyendocrinopathy‐Candidosis‐Ectodermal Dystrophy (APECED)

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Abstract
Ketoconazole was administered as a single daily oral dose of 200 mg to 12 patients with chronic mucocutaneous candidosis (CMC) of autoimmune polyendocrinopathy-candidosis-ectodermal dystrophy (APECED). The study was double-blind and placebo-controlled, with 4-month therapy periods and crossover, and transfer to open-label ketoconazole therapy in cases of failure. During the double-blind trial, all six initially ketoconazole-treated patients showed a clear clinical and mycological improvement. In contrast there was no change or worsening in the initially placebo-treated groups (p = 0.001). Oral candidosis cleared up in all patients, but more rapidly (< 2 weeks) in those aged < 25 years than in older patients (4-10 weeks; P = 0.001). Similarly, nail candidosis improved more rapidly in the younger group. All patients had a recurrence of the candidosis during 36-48 months of post-therapy follow-up. The recurrences likewise responded to ketoconazole. In one patient serum transaminase activities were transiently and marginally elevated during 2-6 weeks of therapy.