Safety and Effectiveness of Long-Term Interferon Gamma Therapy in Patients with Chronic Granulomatous Disease

In chronic granulomatous disease (CGD), diminished or absent neutrophil NADPH oxidase function leads to recurrent pyogenic infections and granuloma formation. In a recent randomized, placebo-controlled trial, short-term prophylactic use of recombinant human interferon gamma (rIFN-γ 1b) reduced the risk of serious infection in CGD patients by 67%. The current study evaluated the safety and effectiveness of long-term rIFN-γ therapy in CGD patients. Patients were treated three times weekly with rIFN-γ and evaluated semiannually. Serious infections (requiring hospitalization and parenteral antibiotic therapy), adverse clinical events, and measures of growth and development were noted. Thirty patients were evaluated for 12 months. The total average duration of rIFN-γ therapy was 2.5 years. Three patients developed a total of four serious infections (0.13 infections per patient year). This rate compares favorably with rates of 1.10 and 0.38 infections per patient year found in the placebo and rIFN-γ groups, respectively, during a previous study. Common adverse events were fever (23%), diarrhea (13%), and flu-like illness (13%). No serious adverse event was attributable to rIFN-γ therapy and no obvious effects on growth and development were observed. rIFN-γ is a safe and effective adjunctive therapy for reducing the frequency and severity of serious infections in CGD patients.