Amantadine in chronic granulomatous disease

Marilena Granzotto, Valentina Leone, Loredana Lepore, Marlenka Zerial, Alberto Tommasini, Roberta Ciambra, Francesca Gombac, Alessandro Ventura

Research output: Contribution to journalArticle


Chronic granulomalous disease (CGD) is a rare genetically determined immunodeficiency. Neutrophils from CGD patients show a defective killing of phagocytosed fungi and bacteria, due. not only to an impairment in oxidative burst, but also to absence of normal pH value within phagocytic vacuole following phagocytosis. Because a weak base such as amantadine could potentially reverse these pH abnormalities, the authors used this drug to treat 2 CGD patients. They observed modifications of both phagosomal pH and killing activity on. their neutrophils compared to those of healthy controls. Since the drug has been employed, the patients have not developed new infections, suggesting a role of amantadine as a part of CGD prophylactic regimen. These results suggest the opportunity of testing the drug in larger studies.

Original languageEnglish
Pages (from-to)147-151
Number of pages5
JournalPediatric Hematology and Oncology
Issue number2
Publication statusPublished - Mar 2005


  • Amatadine
  • Chronic granulomatous disease
  • Lysosomal killing

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology
  • Cancer Research

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  • Cite this

    Granzotto, M., Leone, V., Lepore, L., Zerial, M., Tommasini, A., Ciambra, R., Gombac, F., & Ventura, A. (2005). Amantadine in chronic granulomatous disease. Pediatric Hematology and Oncology, 22(2), 147-151.