Granzotto Marilena, Leone Valentina, Lepore Loredana, Zerial Marlenka, Tommasini Alberto, Ciambra Roberta, Gombac Francesca, Ventura Alessandro
Laboratory of Immunology, IRCCS Burlo Garofolo, Trieste, Italy.
Pediatr Hematol Oncol. 2005 Mar;22(2):147-51. doi: 10.1080/08880010590907267.
Chronic granulomatous 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.
慢性肉芽肿病（CGD）是一种罕见的遗传性免疫缺陷病。CGD患者的中性粒细胞对吞噬的真菌和细菌的杀伤存在缺陷，这不仅是由于氧化爆发受损，还因为吞噬后吞噬泡内缺乏正常的pH值。由于金刚烷胺等弱碱可能会逆转这些pH异常，作者使用该药物治疗了2例CGD患者。与健康对照相比，他们观察到患者中性粒细胞的吞噬体pH值和杀伤活性均有改变。自使用该药物以来，患者未出现新的感染，这表明金刚烷胺可作为CGD预防方案的一部分发挥作用。这些结果表明有必要在更大规模的研究中对该药物进行测试。
