Nitazoxanide in the treatment of acquired immune deficiency syndrome-related cryptosporidiosis: Results of the United States compassionate use program in 365 patients

Abstract

Background: Cryptosporidiosis in patients with acquired immune deficiency syndrome is a serious, life-threatening disease. Aim: A large compassionate use clinical trial was conducted in the USA to make nitazoxanide available to patients with acquired immune deficiency syndrome-related cryptosporidiosis and to collect data related to safety and effectiveness of the drug in this population. Methods: Patients at least 3 years of age with acquired immune deficiency syndrome, diarrhoea (≥4 stools/day for >2 weeks) and Cryptosporidium-positive stools received 500-1500 mg of nitazoxanide twice daily. Patients were evaluated at weeks 1, 2, 4 and monthly thereafter for drug safety and effectiveness including the stool examinations, review of symptoms and patient diaries. Data analysis for clinical and parasitological response was intention-to-treat. Results: Three hundred and sixty-five patients were enrolled at 165 study centres throughout the USA. The duration of treatment ranged from 1 to 1528 days (median 62 days). Among the 357 patients included in the intent-to-treat analysis, 209 (59%) achieved a sustained clinical response while on treatment. Clinical responses were closely associated with Cryptosporidium-negative stools (P < 0.0001). No safety issues were identified at doses up to 3000 mg/day or for long durations of treatment. Conclusions: Nitazoxanide can be considered useful therapy for treatment of with acquired immune deficiency syndrome-related cryptosporidiosis. © 2006 The Author.