Rates of Adverse and Serious Adverse Events in Children with Cystic Fibrosis

Overview

Journal J Cyst Fibros

Publisher Elsevier

Specialty Pulmonary Medicine

Date 2021 Mar 22

PMID 33745860

Citations 1

Authors

Jessica E Pittman

Umer Khan

Theresa A Laguna

Sonya Heltshe

Christopher H Goss

Don B Sanders

Affiliations

Soon will be listed here.

Abstract

Background: 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.

Methods: 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.

Results: 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.

Conclusions: AEs occur commonly in pediatric CF clinical trial participants. Season of enrollment could affect AE rates.

Citing Articles

Potential implicit bias in attribution of adverse events in randomized controlled trials in cystic fibrosis.

Somayaji R, Wessels M, Milinic T, Ramos K, Mayer-Hamblett N, Ramsey B J Cyst Fibros. 2023; 22(4):669-673.

PMID: 37286384 PMC: 10529796. DOI: 10.1016/j.jcf.2023.05.014.

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