1. Clinical features, long-term follow-up and outcome of a large cohort of patients with Chronic Granulomatous Disease: an Italian multicenter study.
- Author
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Martire B, Rondelli R, Soresina A, Pignata C, Broccoletti T, Finocchi A, Rossi P, Gattorno M, Rabusin M, Azzari C, Dellepiane RM, Pietrogrande MC, Trizzino A, Di Bartolomeo P, Martino S, Carpino L, Cossu F, Locatelli F, Maccario R, Pierani P, Putti MC, Stabile A, Notarangelo LD, Ugazio AG, Plebani A, and De Mattia D
- Subjects
- Anti-Infective Agents therapeutic use, Antifungal Agents therapeutic use, Antiviral Agents therapeutic use, Bacterial Infections prevention & control, Child, Child, Preschool, Cohort Studies, Female, Follow-Up Studies, Humans, Infant, Interferon-gamma therapeutic use, Italy, Itraconazole therapeutic use, Kaplan-Meier Estimate, Male, Retrospective Studies, Treatment Outcome, Trimethoprim, Sulfamethoxazole Drug Combination therapeutic use, Bacterial Infections etiology, Granulomatous Disease, Chronic complications, Granulomatous Disease, Chronic diagnosis, Granulomatous Disease, Chronic mortality
- Abstract
A retrospective clinical and immunological survey was conducted in 60 patients with Chronic Granulomatous Disease. A prospective controlled non-randomized study of the efficacy of long-term IFNgamma treatment was carried out. The mean age at the time of diagnosis was 4.4 years; mean duration of follow-up was 10.4 years. Lung and skin infections were the most frequent manifestations both prior to diagnosis and during follow-up. Aspergillus species was the first cause of infection and of death in our cohort. The mortality rate was 13%. Long term prophylaxis with IFNgamma did not significantly change the rate of total infection per patient-year compared to controls (p=0.07). Our data provide clear evidence that protocols of continuing intensive surveillance and monitoring of compliance with anti-infective regimens may significantly improve the quality of life and long-term survival in patients with CGD. No evidence justifying long-term prophylaxis with IFNgamma was obtained.
- Published
- 2008
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