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• W2914170487 abstract "Chronic granulomatous disease (CGD) is a rare immunodeficiency disorder of phagocytic cells resulting in failure to kill a characteristic spectrum of bacteria and fungi and to resolve inflammation. The last few years have witnessed major advances in pathogenesis and clinical management of the disease: Better understanding of 3 physiologic anti-inflammatory functions of NADPH oxidase-derived reactive oxygen species: Promotion of the clearance of dying host cells, suppression of inflammasomes, and regulation of type I interferon signalling. This insight is opening new avenues for targeted drug interventions. Advances in reduced intensity conditioning (RIC) for allogeneic hematopoietic stem cell transplantation (HSCT) make it a promising and safe procedure even for fragile patients with ongoing severe infection or hyperinflammation. Encouraging early data of a multicenter trial of gene-replacement therapy using a self-inactivated lentiviral vector. Combining targeted anti-infectious/anti-inflammatory measures and considering extended indications for curative HSCT are key to improving patient outcome further. Gene therapy will likely become a viable option for disease correction, but long-term assessment is not yet possible. Statement of novelty: We discuss important advances in pathogenesis and treatment of CGD that will change our approach to clinical management." @default.
• W2914170487 created "2019-02-21" @default.
• W2914170487 creator A5081973009 @default.
• W2914170487 date "2019-03-01" @default.
• W2914170487 modified "2023-10-18" @default.
• W2914170487 title "Chronic granulomatous disease 2018: advances in pathophysiology and clinical management" @default.
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• W2914170487 doi "https://doi.org/10.14785/lymphosign-2018-0012" @default.
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