Faculty of Health, Southern Cross University, Coolangatta, Queensland, Australia.
International Rice Research Institute, Laguna, Philippines.
Pediatr Pulmonol. 2023 Jul;58(7):1931-1941. doi: 10.1002/ppul.26413. Epub 2023 Apr 25.
The objective of this study was to conduct a web-based questionnaire to investigate self-reported phenotypes and disease burdens of individuals living in Australia and diagnosed with cystic fibrosis (CF) using a case-control study design.
An online questionnaire was distributed to individuals with CF and healthy control subjects. Overall health rating, medications, family history, education, clinical indicators of disease, and symptoms, including their severity and frequency, were evaluated.
There was a total of 119 respondents consisting of 59 people living with CF and 60 controls. The CF cohort had significantly lower tertiary educational levels compared to controls. The analysis specific to the CF cohort depicted a significant correlation between the frequency of hospitalizations and the level of education in the CF cohort. Of the 26 self-reported symptoms of CF that were analyzed, 14 were significantly higher in the people living with CF. The CF cohort reporting symptoms of chronic pain (25%) described an increase in the burden of disease, depicting a 30% longer mean hospitalization, increased consumption of medications and significant relationships with four other symptoms, including muscle aches, digestive issues, pancreatic insufficiency, and abdominal swelling.
The nationwide survey identified a diverse range of clinical manifestations experienced by the Australian CF population. Chronic pain, linked to aging and the changing landscape of disease, was a significant indicator of the burden of disease. A comprehensive understanding of the phenotypic profiles and symptom variability will contribute to future research and provide insights into the impacts of disease and the burden of therapy, particularly in children, at the start of their health journey.
本研究旨在采用病例对照研究设计，通过网络问卷调查，调查居住在澳大利亚并被诊断为囊性纤维化 (CF) 的个体的自我报告表型和疾病负担。
向 CF 患者和健康对照者分发在线问卷。评估总体健康状况评分、药物使用情况、家族史、教育程度、疾病临床指标以及症状，包括其严重程度和频率。
共有 119 名受访者，包括 59 名 CF 患者和 60 名对照者。CF 队列的高等教育程度明显低于对照组。针对 CF 队列的分析表明，住院频率与 CF 队列的教育程度之间存在显著相关性。在分析的 26 项 CF 自我报告症状中，有 14 项在 CF 患者中明显更高。报告慢性疼痛症状（25%）的 CF 患者描述了疾病负担的增加，表明平均住院时间延长了 30%，药物消耗增加，并与包括肌肉疼痛、消化问题、胰腺功能不全和腹部肿胀在内的其他 4 种症状存在显著关系。
全国性调查确定了澳大利亚 CF 人群所经历的各种临床表现。慢性疼痛与年龄增长和疾病变化的环境有关，是疾病负担的一个重要指标。对表型特征和症状变异性的全面了解将有助于未来的研究，并深入了解疾病的影响和治疗负担，特别是在儿童开始其健康之旅时。
