Journal Article•
A simple rapid micromethod for detecting chronic granulomatous disease of childhood.
About: This article is published in Journal of Laboratory and Clinical Medicine.The article was published on 1970-03-01 and is currently open access. It has received 123 citations till now. The article focuses on the topics: Chronic granulomatous disease.
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TL;DR: A mouse model of CGD is created by targeted disruption of p47phox, one of the genes in which mutations cause human CGD, and a critical role for the phagocyte NADPH oxidase in mammalian host defense is confirmed.
Abstract: Chronic granulomatous disease (CGD) is caused by a congenital defect in phagocyte reduced nicotinamide dinucleotide phosphate (NADPH) oxidase production of superoxide and related species. It is characterized by recurrent life-threatening bacterial and fungal infections and tissue granuloma formation. We have created a mouse model of CGD by targeted disruption of p47phox, one of the genes in which mutations cause human CGD. Identical to the case in human CGD, leukocytes from p47phox-/- mice produced no superoxide and killed staphylococci ineffectively. p47phox-/- mice developed lethal infections and granulomatous inflammation similar to those encountered in human CGD patients. This model mirrors human CGD and confirms a critical role for the phagocyte NADPH oxidase in mammalian host defense.
481 citations
TL;DR: Treatment with rhG-CSF can lead to a large increase in the numbers of functional neutrophils in patients with congenital agranulocytosis, and the number of new infectious episodes and the requirement for intravenous antibiotics have decreased.
Abstract: Congenital agranulocytosis is a disorder characterized by severe neutropenia and a profound deficiency of identifiable neutrophil progenitors in bone marrow. In an attempt to stimulate neutrophil production and thereby reduce the morbidity and mortality associated with this disease, we administered recombinant human granulocyte colony-stimulating factor (rhG-CSF) in doses of 3 to 60 μg per kilogram of body weight per day to five patients with congenital agranulocytosis. In all five patients, an increase in the number of neutrophils was noted eight to nine days after the initiation of the effective dosage (the dose at which the neutrophil count reached 1000 cells per microliter or more and the bone marrow showed granulocyte maturation beyond the myelocyte stage). The absolute neutrophil counts rose from less than 100 to between 1300 and 9500 cells per microliter. Marrow aspirates obtained after 14 days at the effective dosage showed maturation to the mature neutrophil stage. The side effects that ...
444 citations
TL;DR: A potential role for rhG-CSF is indicated in the treatment of patients with myelodysplastic syndrome, congenital agranulocytosis, radiation-induced myelosuppression, and bone marrow transplantation and the increase in absolute numbers of functionally active neutrophils may have a profound effect in the rate and severity of neutropenia-related sepsis.
Abstract: We examined the in vivo effects of recombinant human granulocyte colony-stimulating factor (rhG-CSF) in primates (cynomolgus monkeys) treated with subcutaneous doses of rhG-CSF for 14-28 d. A dose-dependent increase in the peripheral white blood cells (WBC) was seen, reaching a plateau after 1 wk of rhG-CSF treatment. The elevation of WBC was due to an increase in the absolute neutrophil count. These results demonstrate that rhG-CSF is a potent granulopoietic growth and differentiation factor in vivo. In cyclophosphamide (CY)-induced myelosuppression, rhG-CSF was able to shorten the time period of WBC recovery in two treated monkeys to 1 wk, as compared to more than 4 wk for the control monkey. Its ability to significantly shorten the period of chemotherapy-induced bone marrow hypoplasia may allow clinicians to increase the frequency or dosage of chemotherapeutic agents. In addition, the increase in absolute numbers of functionally active neutrophils may have a profound effect in the rate and severity of neutropenia-related sepsis. Furthermore, the activities reported here indicate a potential role for rhG-CSF in the treatment of patients with myelodysplastic syndrome, congenital agranulocytosis, radiation-induced myelosuppression, and bone marrow transplantation.
418 citations
TL;DR: Reconciliation of neutrophil precursors in the bone marrow and a circulating neutrophIL count of less than 1500 cells per microliter in patients with idiopathic neutropenia is confirmed.
Abstract: CHRONIC idiopathic neutropenia is characterized by maturational arrest of neutrophil precursors in the bone marrow and a circulating neutrophil count of less than 1500 cells per microliter. Other h...
157 citations
TL;DR: The mother of two girls with CGD had a normal NBT slide test, suggesting an autosomal recessive inheritance, and two brothers with a CGD variant showed 10 to 30 per cent NBT-positive cells, when endotoxin-coated slides were used.
153 citations