Abstract
BackgroundSince 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.MethodTreatments and treatment outcomes were retrospectively analyzed for 46 pediatric CF patients diagnosed from August 2009 to June 2019. Pre- and post-treatment results were compared using independent samples t-test.ResultsOf 46 pediatric CF study patients, four died 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. For 17 children, no significant differences in lung function were found at follow-up as compared to lung function 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). Pseudomonas aeruginosa (17/27) and bronchiectasis (22/22) were found during follow-up evaluation. Twenty-four patients (64.8%) maintained good adherence to therapies. Overall, azithromycin and tobramycin treatments were administered for 0.5–62 months and 0.5–48 months, respectively, and triggered no obvious adverse reactions.ConclusionNo obvious declines in 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.
Highlights
Cystic fibrosis (CF) is a common life-limiting autosomal recessive genetic disorder, with highest prevalence in Europe, North America and Australia
For 17 children, no significant differences in lung function were found at follow-up as compared to lung function 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)
No obvious declines in clinical presentation or lung function were found in Chinese pediatric cystic fibrosis (CF) patients after receiving standard therapeutic and active treatments, malnutrition and low compliance were persistent challenges
Summary
Cystic fibrosis (CF) is a common life-limiting autosomal recessive genetic disorder, with highest prevalence in Europe, North America and Australia. The median age at CF diagnosis is currently 8.7 years in Chinese patients, according to results of a systematic review report [5]. This median age at diagnosis is significantly delayed as compared to the median age at diagnosis of 0.5 years reported in European patients [6] and of 3 months reported in American patients (according to the 2017 Patient Registry Annual Data Report). Factors underlying CF patient diagnostic delays and obstacles patients face in maintaining treatment compliance and achieving positive outcomes were investigated by retrospectively reviewing patient records in order to better understand challenges faced by children with cystic fibrosis in China. This study aimed to investigate medical and other challenges faced by pediatric CF patients in China
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