Journal Article

Long-Term Interferon-γ Therapy for Patients with Chronic Granulomatous Disease

Beatriz E. Marciano, Robert Wesley, Ellen S. De Carlo, Victoria L. Anderson, Lisa A. Barnhart, Dirk Darnell, Harry L. Malech, John I. Gallin and Steven M. Holland

in Clinical Infectious Diseases

Published on behalf of Infectious Diseases Society of America

Volume 39, issue 5, pages 692-699
Published in print September 2004 | ISSN: 1058-4838
Published online September 2004 | e-ISSN: 1537-6591 | DOI: http://dx.doi.org/10.1086/422993
Long-Term Interferon-γ Therapy for Patients with Chronic Granulomatous Disease

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Background. Chronic granulomatous disease (CGD) is a rare disorder of phagocytes in which absent production of superoxide and hydrogen peroxide in phagocytes predisposes patients to bacterial and fungal infections. Infections are dramatically reduced by prophylaxis with antibiotics, antifungals, and interferon-γ (IFN-γ).

Methods. Seventy-six patients with CGD were enrolled in an uncontrolled, open-label follow-up study to assess the long-term clinical safety and efficacy of IFN-γ therapy. Patients received IFN-γ subcutaneously 3 times per week.

Results. We observed patients for up to 9 years, for a total observation period of 328.4 patient-years. The incidence of serious infections was 0.30 infections per patient-year; for serious bacterial infections, the incidence was 0.18 cases per patient-year, and for serious fungal infections, it was 0.12 cases per patient-year. Thirty-seven percent of patients reported an adverse event, the most common of which was fever. Twenty-six patients withdrew from the study (3 because of adverse events, 15 because of patient preference, and 8 because of transfer to another trial). There were no life-threatening IFN-γ—related adverse events and no discernible effects on growth. The overall mortality rate was 1.5% per patient-year.

Conclusion. IFN-γ prophylaxis for CGD appears to be effective and well tolerated over a prolonged period of time.

Journal Article.  4234 words.  Illustrated.

Subjects: Infectious Diseases ; Immunology ; Public Health and Epidemiology ; Microbiology

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