Clinical features, long-term follow-up and outcome of a large cohort of patients with Chronic Granulomatous Disease: An Italian multicenter study

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• 作者：Baldassarre Martire ; Roberto Rondelli ; Annarosa Soresina ; Claudio Pignata ; Teresa Broccoletti ; Andrea Finocchi ; Paolo Rossi ; Marco Gattorno ; Marco Rabusin ; Chiara Azzari ; Rosa M. Dellepiane ; Maria C.
• 关键词：Chronic Granulomatous Disease (CGD) ; Interferon gamma (IFNγ ; ) ; Cotrimoxazole (CTX) ; Itraconazole (ITRA) ; Infections ; Follow up
• 刊名：Clinical Immunology
• 出版年：2008
• 出版时间：February 2008
• 年：2008
• 卷：126
• 期：2
• 页码：155-164
• 全文大小：273 K

文摘

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.