Division of Allergy and Pulmonary Medicine, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States.
Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle WA, United States.
J Cyst Fibros. 2021 Nov;20(6):972-977. doi: 10.1016/j.jcf.2021.02.013. Epub 2021 Mar 18.
Cystic fibrosis (CF) is an autosomal recessive disease characterized by chronic sinopulmonary symptoms and chronic gastrointestinal symptoms that begins in infancy. Children with CF are increasingly being included in clinical trials. In order to fully evaluate the impact of new therapies in future clinical trials, an understanding of baseline adverse event (AE) rates in children with CF is needed. To address this, we determined the rates of common AEs in pediatric patients with CF who participated in two clinical trials.
We reviewed AEs for placebo recipients in the AZ0004 study and inhaled tobramycin recipients in the Early Pseudomonas Infection Control (EPIC) clinical trial. AEs were categorized based on Medical Dictionary for Regulatory Activities (MedDRA) coding classifications and pooled into common, batched AE descriptors. AE rates were estimated from negative binomial models according to age groups, severity of lung disease, and season.
A total of 433 children had 8,266 total AEs reported, or 18.1 (95% CI 17.0, 19.2) AEs per person per year. Respiratory AEs were the most commonly reported AEs, with a rate of 7.6 events per person-year. The total SAE rate was 0.33 per person per-year. Cough was the most commonly reported respiratory AE, with 61% of subjects reporting at least one episode of cough within 4 months. The rate ratio of any AE was higher in Spring, Fall, and Winter, compared with Summer.
AEs occur commonly in pediatric CF clinical trial participants. Season of enrollment could affect AE rates.
囊性纤维化（CF）是一种常染色体隐性遗传病，其特征为慢性鼻-肺症状和慢性胃肠道症状，这些症状始于婴儿期。越来越多的 CF 患儿被纳入临床试验。为了在未来的临床试验中充分评估新疗法的影响，需要了解 CF 患儿的基线不良事件（AE）发生率。为此，我们确定了两项临床试验中接受安慰剂和吸入妥布霉素的 CF 儿科患者的常见 AE 发生率。
我们回顾了 AZ0004 研究中安慰剂组和早期铜绿假单胞菌感染控制（EPIC）临床试验中吸入妥布霉素组的 AE。根据监管活动医学词典（MedDRA）编码分类，AE 被归类为常见、批量 AE 描述符。根据年龄组、肺部疾病严重程度和季节，使用负二项式模型估计 AE 发生率。
共有 433 名儿童报告了 8266 例总 AE，或每人每年 18.1 例（95%CI 17.0，19.2）。呼吸道 AE 是最常见的 AE，发生率为每人每年 7.6 例。总 SAE 发生率为 0.33 例/人/年。咳嗽是最常见的呼吸道 AE，4 个月内有 61%的患者至少报告了一次咳嗽。与夏季相比，春季、秋季和冬季的任何 AE 发生率均较高。
AE 在儿科 CF 临床试验参与者中很常见。入组季节可能会影响 AE 发生率。
