Long-term follow-up and outcome of 39 patients with chronic granulomatous disease

J Liese, S Kloos, V Jendrossek, T Petropoulou… - The Journal of …, 2000 - Elsevier
J Liese, S Kloos, V Jendrossek, T Petropoulou, U Wintergerst, G Notheis, M Gahr…
The Journal of pediatrics, 2000Elsevier
Objectives: To evaluate the clinical long-term course in patients with chronic granulomatous
disease (CGD) with respect to different CGD subtypes and currently used antimicrobial
prophylactic measures. Study design: The records of 39 patients with CGD who were
monitored during a period of 22 years were reviewed. All infections, infectious
complications, and clinical outcomes were documented for a total observation period of 610
patient-years and were stratified with respect to different CGD subtypes. Results …
Objectives
To evaluate the clinical long-term course in patients with chronic granulomatous disease (CGD) with respect to different CGD subtypes and currently used antimicrobial prophylactic measures.
Study design
The records of 39 patients with CGD who were monitored during a period of 22 years were reviewed. All infections, infectious complications, and clinical outcomes were documented for a total observation period of 610 patient-years and were stratified with respect to different CGD subtypes.
Results
Lymphadenitis, skin abscesses, and pneumonia occurred in 87%, 72%, and 59% of the patients, respectively. In 151 microbiologic isolates Staphylococcus aureus, Aspergillus species, Candida species, Pseudomonas species, and Salmonella species were the most frequently detected microorganisms. There were 167 severe infections requiring hospitalization and intravenous antimicrobial treatment, resulting in an incidence of 3.7 severe infections per 100 patient months (SI/100 PM). Long-term antibiotic prophylaxis significantly reduced the incidence of severe bacterial infections from 4.8 SI/100 PM to 1.6 SI/100 PM (P = .0035). In contrast, fungal infections increased under antibiotic prophylaxis from a mean incidence of 0.2 SI/100 PM to 1.9 SI/100 PM (P = .04). We found a 50% survival rate through the fourth decade of life, with a plateau after the third decade of life. Patients with a complete absence of cytochrome b558 showed an earlier manifestation of their disease and a higher incidence of infections and had significant lower survival than patients with only diminished cytochrome b558 or autosomal recessive CGD.
Conclusions
Infections with Aspergillus species have become the major cause of infectious complications and death in patients with CGD. Prophylactic and therapeutic measures are needed to further increase life expectancy and quality for patients with CGD. (J Pediatr 2000;137:687-93)
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