title: Ketoconazole is effective against the chronic mucocutaneous candidosis of autoimmune polyendocrinopathy-candidosis-ectodermal dystrophy (APECED).
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 group (p = 0.001). Oral candidosis cleared up in all patients, but more rapidly (less than 2 weeks) in those aged less than 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.
mesh_terms: D000293:Adolescent; D000309:Adrenal Insufficiency / Q000188:drug therapy*; D000328:Adult; D001327:Autoimmune Diseases / Q000188:drug therapy*; D002177:Candidiasis / Q000188:drug therapy*; D002178:Candidiasis, Chronic Mucocutaneous / Q000188:drug therapy*; D002648:Child; D002986:Clinical Trials as Topic; D004311:Double-Blind Method; D004476:Ectodermal Dysplasia / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007654:Ketoconazole / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D011897:Random Allocation; D013577:Syndrome
chemical_list: D007654:Ketoconazole