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

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. Pietrogrande; Antonino Trizzino; Paolo Di Bartolomeo; Silvana Martino; Luigi Carpino; Fausto Cossu; Franco Locatelli; Rita Maccario; Paolo Pierani; Maria C. Putti; Achille Stabile; Luigi D. Notarangelo; Alberto G. Ugazio; Alessandro Plebani; Domenico De Mattia

doi:10.1016/j.clim.2007.09.008

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

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. Pietrogrande, Antonino Trizzino, Paolo Di Bartolomeo, Silvana Martino, Luigi Carpino, Fausto Cossu, Franco Locatelli, Rita Maccario, Paolo PieraniMaria C. Putti, Achille Stabile, Luigi D. Notarangelo, Alberto G. Ugazio, Alessandro Plebani, Domenico De Mattia

Research output: Contribution to journal › Article › peer-review

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.

Original language	English
Pages (from-to)	155-164
Number of pages	10
Journal	Clinical Immunology
Volume	126
Issue number	2
DOIs	https://doi.org/10.1016/j.clim.2007.09.008
Publication status	Published - Feb 2008

Keywords

Chronic Granulomatous Disease (CGD)
Cotrimoxazole (CTX)
Follow up
Infections
Interferon gamma (IFNγ)
Itraconazole (ITRA)

ASJC Scopus subject areas

Immunology
Immunology and Allergy

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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, R. M., Pietrogrande, M. C., Trizzino, A., Di Bartolomeo, P., Martino, S., Carpino, L., Cossu, F., Locatelli, F., Maccario, R., ... De Mattia, D. (2008). Clinical features, long-term follow-up and outcome of a large cohort of patients with Chronic Granulomatous Disease: An Italian multicenter study. Clinical Immunology, 126(2), 155-164. https://doi.org/10.1016/j.clim.2007.09.008

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 & De Mattia, D 2008, 'Clinical features, long-term follow-up and outcome of a large cohort of patients with Chronic Granulomatous Disease: An Italian multicenter study', Clinical Immunology, vol. 126, no. 2, pp. 155-164. https://doi.org/10.1016/j.clim.2007.09.008

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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.",

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AU - Martire, Baldassarre

AU - Rondelli, Roberto

AU - Soresina, Annarosa

AU - Pignata, Claudio

AU - Broccoletti, Teresa

AU - Finocchi, Andrea

AU - Rossi, Paolo

AU - Gattorno, Marco

AU - Rabusin, Marco

AU - Azzari, Chiara

AU - Dellepiane, Rosa M.

AU - Pietrogrande, Maria C.

AU - Trizzino, Antonino

AU - Di Bartolomeo, Paolo

AU - Martino, Silvana

AU - Carpino, Luigi

AU - Cossu, Fausto

AU - Locatelli, Franco

AU - Maccario, Rita

AU - Pierani, Paolo

AU - Putti, Maria C.

AU - Stabile, Achille

AU - Notarangelo, Luigi D.

AU - Ugazio, Alberto G.

AU - Plebani, Alessandro

AU - De Mattia, Domenico

PY - 2008/2

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KW - Follow up

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

Abstract

Keywords

ASJC Scopus subject areas

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