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  • 1
    Electronic Resource
    Electronic Resource
    Springer
    European journal of pediatrics 154 (1995), S. 295-298 
    ISSN: 1432-1076
    Keywords: Chronic granulomatous disease ; Interferon-gamma ; Infections
    Source: Springer Online Journal Archives 1860-2000
    Topics: Medicine
    Notes: Abstract This was an uncontrolled, open-label follow up study of a previous 12-month, randomized, double-blind, placebo-controlled trial performed to assess the long-term efficacy and safety of Recombinant Human Interferon Gamma (rIFN-γ) in patients with chronic granulomatous disease (CGD). In two centres, 28 patients (24 male, 4 female) with a mean age of 16 years (range 3–37) entered the open-label phase. The patients were treated for a mean of 880 days (range 97–1375 days). Visits were scheduled every 180 days and patients completed one to six visits. rIFN-γ was administered subcutaneously three times weekly at a dose of 0.05 mg per m2. During the open-label phase of the study 12 patients experienced a serious infection requiring hospitalization within 880 days. The median infection-free time was 993 days. No obvious increase of infections over time was seen. Phagocyte superoxide anion production and phagocyte staphylococcal killing were not influenced by therapy. Seven patients were withdrawn from the study, one because of an adverse reaction, three on their own wish and the other three because they changed to another trial. No patient died during the study. Conclusion Treatment of patients with CGD with intracellular active antibiotics and additional interferon gamma as infection prophylaxis is safe and justified.
    Type of Medium: Electronic Resource
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  • 2
    ISSN: 1432-1076
    Keywords: C2 deficiency ; Alternative pathway of complement ; Bacterial infections
    Source: Springer Online Journal Archives 1860-2000
    Topics: Medicine
    Notes: Abstract Selective homozygous deficiency of the second component of complement, C2, with increased susceptibility to infection was detected in five children of two unrelated families. Because the haemolytic activity of the alternative complement pathway (AP) was in the low normal range, we evaluated the AP activation pattern. Serum levels of factor B measured immunochemically and the haemolytic function of factor B were low normal. Levels of C3d were not increased. Activation products of factor B were undetectable indicating the absence of in vivo activation of AP. Activation of C3 in vitro by activators of the AP (zymosan A and lipopolysaccaride) was profoundly deficient in homozygous C2 deficiency while heterozygous carriers exhibited intermediate values. There was no correlation between serum levels of factor B and in vitro C3 activation. We conclude that defective AP activation may contribute to increased susceptibility to bacterial infections in some patients with homozygous C2 deficiency.
    Type of Medium: Electronic Resource
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  • 3
    ISSN: 1432-1076
    Keywords: Chronic granulomatous disease ; Recombinant human granulocyte-macrophage colony stimulating factor
    Source: Springer Online Journal Archives 1860-2000
    Topics: Medicine
    Notes: Abstract Variant X-linked chronic granulomatous disease (CGD) is characterised by a decreased but still measurable respiratory burst and cytochrome b content of phagocytes resulting in a clinically milder form of the disease. We examined the in vivo effect of recombinant human granulocyte-macrophage colony stimulating factor (rh-GM-CSF) on the neutrophil functions of a patient treated for liver abscess. The number of white blood cells was markedly increased at the highest dose of GM-CSF injected (30 μg/kg per day). This was mainly due to a large increase in eosinophils and to a lesser extent in neutrophils. No change in the deficient neutrophil respiratory burst nitroblue tetrazolium (NBT)-reduction, superoxide (O 2 − )-production and cytochrome b content was observed during 6 weeks of therapy with increasing doses of GM-CSF. No significant clinical improvement of the liver abscess was observed during treatment with GM-CSF.
    Type of Medium: Electronic Resource
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