What is the efficacy of pulmozyme in children?

Pulmozyme

is an inhaled medication that thins mucus and is used to manage and treat cystic fibrosis. It has been shown to reduce respiratory infections in selected patients with forced vital capacity (FVC) greater than 40% of predicted value.

Pulmozyme increases the pourability and reduces the viscoelasticity of CF sputum in vitro and increases its mucociliary transportability in animal models.

Short-term (10-day) Phase I and II clinical trials demonstrated that pulmozyme was safe and effective in improving lung function in clinically stable CF patients with mild to moderate lung disease, with FVC ≥40% of predicted value.

About cystic fibrosis

Cystic fibrosis is an autosomal recessive multisystem progressive disease that causes mucus in various organs to become thicker. The disease primarily affects a patient's upper and lower respiratory tract, causing long-term lung infection and limiting the ability to breathe over time.

pulmozyme for children

Study purpose: Airway mucus obstruction is a major challenge for children in the pediatric intensive care unit (PICU). To evaluate the evidence and contemporary use of the mucolytic drug pulmozyme in the treatment of non-cystic fibrosis disease in the PICU.

Methods: For quality assessment and data synthesis, only randomized controlled trials (RCTs) comparing pulmozyme with standard care or placebo in critically ill pediatric patients (<18 years) in the PICU were included.

Study results: pulmozyme resulted in a reduction of approximately 1 day in the duration of mechanical ventilation, a 36% reduction. There is evidence that treating cystic fibrosis patients with pulmozyme improved lung function compared with placebo in trials lasting one month to two years, and reduced lung exacerbations in trials of six months or longer.

Treatment with pulmozyme for more than one month was associated with improvements in lung function in CF patients, and results from a six-month trial showed the same effect. Two years of treatment (based on one trial) significantly improved children's FEV(1) without a significant reduction in the risk of infectious exacerbations.

Voice changes and rash appear to be the only ones reported with increased frequency in randomized controlled trials.

References:

den Hollander B, Linssen RSN, Cortjens B, van Etten-Jamaludin FS, van Woensel JBM, Bem RA; Dutch Collaborative PICU Research Network. Use of dornase alfa in the paediatric intensive care unit: current literature and a national cross-sectional survey. Eur J Hosp Pharm. 2022 May;29(3):123-128. doi: 10.1136/ejhpharm-2020-002507. Epub 2020 Oct 29. PMID: 33122405; PMCID: PMC9047925.

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