Bone marrow transplant has improved to a level where it has become a more standard curative treatment for patients whose CGD has led to severe challenges.
Gene therapy for chronic granulomatous disease is an experimental clinical treatment and is not yet a standard of care. A CGD patient’s blood stem cells are collected and a healthy version of the gp91- phox gene is inserted into the stem cells. The patient with CGD undergoes chemotherapy before the cells are intravenously infused. After recovery from chemotherapy and the corrected cells have engrafted, the stem cells are potentially able to produce new white blood cells that can fight off infection.
No course of action, whether CGD treatment/prophylaxis or curative option, is without risks. It is important to encourage patients with CGD and their families to educate themselves in order to select the path that is right for them
While each patient with CGD is different due to individual levels of NADPH-oxidase activity and form of CGD (as well as other unrelated health or environmental issues) it is recommended that patients be seen and, if necessary, managed proactively by a team of qualified specialists/physicians, who work in concert with a primary care physician. These can include (but are not limited to):
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