Related Experiment Video

Updated: Jun 6, 2025

High-speed Video Microscopy Analysis for First-line Diagnosis of Primary Ciliary Dyskinesia
05:32

High-speed Video Microscopy Analysis for First-line Diagnosis of Primary Ciliary Dyskinesia

Published on: January 19, 2022

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Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study

Robert A Gardner1, Thomas W Ferkol2,3, Stephanie D Davis2

  • 1Department of Pediatrics, Levine Children's Hospital, Wake Forest University School of Medicine, Charlotte, North Carolina, USA.

Pediatric Pulmonology
|November 22, 2024
PubMed

Insights

Management of primary ciliary dyskinesia (PCD) involves diverse therapies, with older patients and those with specific genetic defects using more treatments. Current PCD treatment lacks standardized evaluation, highlighting the need for evidence-based guidelines.

Area of Science:

  • Pulmonology
  • Genetics
  • Pediatrics

Background:

  • Primary ciliary dyskinesia (PCD) management is largely empirical, lacking systematic evaluation.
  • Understanding current therapeutic approaches is crucial for improving patient care.

Purpose of the Study:

  • To describe the range and trends of therapies used in pediatric patients with PCD.
  • To identify factors associated with the use of specific treatments, including antibiotics.

Main Methods:

  • A prospective, longitudinal, multicenter observational study enrolled pediatric participants with PCD.
  • Therapies were recorded annually, and age-related trends were analyzed.
  • Generalized estimating equations identified covariates influencing therapy prevalence and oral antibiotic use.

Main Results:

  • 137 participants completed 897 visits over 13 years; nearly all received antibiotics.
  • Cephalosporins and amoxicillin-clavulanate were most common; 31% used chronic azithromycin.
  • Older patients and those with inner dynein arm/microtubular defects used more amoxicillin-clavulanate, chronic azithromycin, inhaled steroids, and hypertonic saline.

Conclusions:

  • Therapy use in PCD increases with age and disease severity, suggesting progression and more severe phenotypes.
  • A wide array of therapies are employed, but evidence-based studies defining benefits and risks are lacking.
Abstract

Keywords:
natural historyprimary ciliary dyskinesiatherapy

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