Synopsis

Source of validity: Scientific
Rating (out of 5): 4

Mannitol is scientifically validated for use in Cystic Fibrosis (CF), primarily as an inhaled osmotic agent. CF is characterized by abnormal chloride and sodium transport in epithelial cells, leading to thick, viscous mucus in the lungs. This mucus obstructs airways and promotes recurrent infections. Mannitol, when inhaled as a dry powder, works by drawing water into the airways through osmotic action, hydrating and thinning the mucus. This facilitates mucociliary clearance, improves lung function, and reduces the risk of infections.

Multiple randomized controlled trials have demonstrated the efficacy and safety of inhaled mannitol in CF patients. For example, the CF301 and CF302 phase III trials showed that regular use of inhaled mannitol significantly improved lung function (measured by FEV₁) compared to control groups. These trials also reported improvements in quality of life and acceptable safety profiles, with cough being the most common side effect. As a result, inhaled mannitol (e.g., Bronchitol) has been approved for clinical use in adults (and in some regions, children) with CF who can benefit from enhanced airway clearance.

In summary, the use of mannitol in cystic fibrosis is supported by a robust body of clinical research and is incorporated into treatment guidelines in several countries. Its mechanism of action and clinical benefits are well understood, and it provides an alternative to other mucolytic therapies such as hypertonic saline and dornase alfa.

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