Safety and effectiveness of clofazimine in nontuberculous mycobacterial lung disease

RATIONALE: Clofazimine has been reported to be effective in the treatment of nontuberculous mycobacterial pulmonary disease (NTM-PD); however, there are scarce data regarding its tolerability and efficacy. OBJECTIVES: We sought to review the safety and efficacy of clofazimine in patients with NTM-PD in our clinic. METHODS: We retrospectively reviewed all patients with NTM PD who received a regimen containing clofazimine. Demographics, clinical and radiological data, indication and dose of clofazimine, accompanying drugs, need for dose adjustments and adverse drug effects were recorded. Clinical and microbiological outcomes were assessed in those patients who received clofazimine for 6 months or longer. MEASUREMENTS AND MAIN RESULTS: Forty-seven patients received a regimen containing clofazimine for a mean (SD) of 26.3 (26.3) months. Thirty-six (76.6%) patients developed at least one adverse effect while receiving clofazimine and 12 (25.5%) had to stop clofazimine due to an adverse effect. Ten of 17 patients (58.8%) tested had a prolonged QTc while receiving clofazimine. Out of 35 patients who were on clofazimine for more than 6 months, 15 (42.9%) experienced an improvement in symptoms and 7/27 (25.9%) converted their sputum to culture negative within a year following the initiation of clofazimine. CONCLUSIONS: Adverse effects were common in our series, but attribution to any specific drug was difficult. A significant minority required clofazimine cessation due to intolerance. QTc interval should be monitored in patients receiving regimens containing macrolides, fluoroquinolones and/or clofazimine. Prospective randomized studies are needed to better assess the efficacy and safety of this drug in the management of NTM lung disease.