Chronic airway disease in primary ciliary dyskinesia-spiced with geno-phenotype associations

Kim G Nielsen1,2, Mathias G Holgersen1, Suzanne Crowley3

  • 1Department of Paediatrics and Adolescent Medicine, Danish PCD & chILD Centre, CF Centre Copenhagen, Paediatric Pulmonary Service, ERN Accredited, Copenhagen, Denmark.

Insights

Primary ciliary dyskinesia (PCD) is a genetic disorder causing chronic airway disease from birth. Research focuses on understanding its lifelong impact and developing personalized treatments like mRNA therapy.

Area of Science:

  • Pulmonary Medicine
  • Genetics
  • Immunology

Background:

  • Primary ciliary dyskinesia (PCD) is a genetic, multi-organ ciliopathy characterized by chronic airway disease.
  • The condition affects the upper and lower respiratory tract, sinuses, and middle ear, with lung disease beginning at birth.

Purpose of the Study:

  • To review the current understanding of chronic airway disease in primary ciliary dyskinesia, focusing on the lower airways.
  • To highlight research challenges, knowledge gaps, and future opportunities in PCD lung disease.

Main Methods:

  • Narrative review of existing literature on primary ciliary dyskinesia.
  • Focus on lower airway manifestations and lifelong disease progression.

Main Results:

  • PCD involves severe neutrophil inflammation in the respiratory tract, minimally impacted by current infection-focused treatments.
  • Clinical presentation, lung function, structural damage, infection burden, and treatment needs evolve throughout a patient's life.

Conclusions:

  • A deeper understanding of PCD's multifaceted aspects and genotype-specific courses is crucial.
  • Genetic diagnosis and comprehensive patient characterization are essential for advancing personalized medicine, including potential mRNA therapies.

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