Prognosis and treatment of 46 Chinese pediatric cystic fibrosis patients
Background: Since public awareness of cystic fibrosis (CF) has increased, more children have been diagnosed with CF in China. This study aimed to investigate medical and other challenges faced by pediatric CF patients in China.Method: Treatments and treatment outcomes were retrospectively analyzed for 46 pediatric CF patients diagnosed from August 2009 to June 2019.Results: Of 46 pediatric CF study patients, four died (one of pulmonary infection, one of severe respiratory failure, two of
... causes) and five were lost to follow-up. Thirty-seven patients were monitored for 0.03 to 9.21 years; patients exhibited fewer attacks of respiratory tract infections after diagnosis (4.49 ± 2.13 episodes/year before diagnosis VS 1.97 ± 1.87 times/year after 1-year treatment, p < 0.05), significantly reduced sputum production and experienced 1.62 ± 1.71 exacerbations/year. Patient mean body mass index was 16.87 ± 3.53 and pancreatic malfunction persisted in 15 patients. Of the 28 children older than 6 years of age, 17 received lung function tests during follow-up; no significant differences were observed between lung function at follow-up and at diagnosis (FEV1: 82.45% ± 16.56% vs 75.26% ± 22.34%, FVC: 87.18% ± 13.64% vs 86.99% ± 19.95%, FEF75%: 46.51% ± 28.78% vs 36.63% ± 24.30%, P = 0.27, 0.97, 0.20, respectively). In 27 cases with sputum results at follow-up, Pseudomonas aeruginosa was detected in 17 (62.97%), persisted in 14, newly emerged in 3 and was eliminated in 5 cases. Computed tomography lung scans of 22 cases revealed increased bronchiectasis in 6 cases after 2-60 months of treatment, decreased bronchiectasis in 3 cases after 2-96 months of treatment and unchanged bronchiectasis in 13 cases treated for 5-60 months. Twenty-four patients complied with aerosolized therapy, azithromycin, pancreatic enzymes and nutritional intake-based treatments, while 9 patients once stopped aerosolized therapy or azithromycin and 4 patients stopped all medications. Overall, azithromycin and tobramycin treatments were administered for 0.5-62 months and 0.5-48 months, respectively, and triggered no obvious adverse reactions. Conclusion: No obvious deterioration of clinical presentation or lung function were found in Chinese pediatric CF patients after receiving standard therapeutic and active treatments, although malnutrition and low compliance were persistent challenges.