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 IFNγ 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 IFNγ 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 IFNγ was obtained.
Clinical features, long-term follow-up and outcome of a large cohort ofpatients with Chronic Granulomatous Disease: An Italian multicenter study / Martire, B., Rondelli, R., Soresina, A., Pignata, C., Broccoletti, T., Finocchi, A., Rossi, P., Gattorno, M., Rabusin, M., Azzari, C., Dellepiane, R.M., Pietrogrande, M.C., Trizzino, A., DI BARTOLOMEO, P., Martino, S., Carpino, L., Cossu, F., Locatelli, F., Maccario, R., Pievani, P., et al.. - In: CLINICAL IMMUNOLOGY. - ISSN 1521-6616. - STAMPA. - 126:(2007), pp. 155-164. [10.1016/j.clim.2007.09.008]
Clinical features, long-term follow-up and outcome of a large cohort ofpatients with Chronic Granulomatous Disease: An Italian multicenter study.
PIGNATA, CLAUDIO;BROCCOLETTI, TERESA;
2007
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 IFNγ 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 IFNγ 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 IFNγ was obtained.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
