Treatments and prognosis of 82 children with chronic granulomatous disease in a single center / 中华实用儿科临床杂志

ABSTRACT

Objective:To summarize the treatments and outcomes of patients with chronic granulomatous disease (CGD), so as to improve the management of the disease and improve the quality of life of the patients.Methods:Clinical data of 82 children with CGD hospitalized at Children′s Hospital of Chongqing Medical University and born between January 1999 and December 2016 were collected.Patients were divided into the survival group and the death group.The general information, infectious events and inflammatory complications, as well as treatments and survival situations were analyzed.Results:Among the 82 cases, 55 cases (67.1%) were in the survival group and 27 cases (32.9%) were in the death group.Forty-nine (59.8%) cases developed manifestations in the neonatal period.Specifically, there was a statistically significant difference ( P<0.05) in the proportion of patients who deve-loped manifestations in the neonatal period between the death group (81.5%) and the survival group (49.1%). Fifty-eight (70.7%) cases started to receive Compound Sulfamethoxazole since diagnosis, with an average duration of 36.8 months.Fifty-two (63.4%) cases received Voriconazole or Itraconazole to prevent fungal infection, with an average duration of 34.3 months.There were 288 hospitalizations due to infections in total.The detection rate of bacteria, fungi and mycobacterium tuberculosis was 41.3%, and the rate in the death group (51.8%) was higher than that in the survival group (36.9%)( P<0.05). Klebsiella pneumoniae, Staphylococcus aureus, Burkholderia onion and Escherichia coli were the most frequent bacteria detected, among which Klebsiella pneumoniae and Escherichia coli were all exten-ded-spectrum β-lactamases positive.The use of advanced-antibiotics in the death group (70.4%) was higher than that in the survival group (40.0%) ( P<0.05). Seventeen (20.7%) cases developed inflammatory complications, mainly including interstitial lung disease or pulmonary fibrosis, inflammatory bowel disease and incomplete Kawasaki disease.Thirty-two (39.0%) cases received hematopoietic stem cell transplantation (HSCT), of which 25 patients (78.1%) were transplanted in Children′s Hospital of Chongqing Medical University, with a success rate of 96.0% (24/25 cases). The Kaplan-Meier survival curves showed a worse prognosis in those who developed manifestations in neonates, rely on advanced-antibiotics, and did not receive transplantations. Conclusions:The earlier the clinical manifestations appear, and the higher the utilization rate of advanced-antibiotics used, the more serious the condition is.Conservative treatment with drugs is still insufficient, and the emergence of drug-resistant bacteria increases the difficulty of managements.The recognition and treatments of inflammatory complications needs more explorations.HSCT is so far the only curative therapy for CGD in China.