Ambroxol (2-amino-3,5-dibromo-N-[trans-4-hydroxycyclohexyl]benzylamine), an over-the-counter mucoactive agent has been effectively used to treat both acute and chronic respiratory diseases since 1978. The current review is aimed to establish clinical evidences on its use in children with acute and chronic respiratory diseases. The authors utilized the data from both published and unpublished clinical studies, and real-world evidence studies. These studies, consisting of almost 1,300 pediatric patients, suggested strong clinical outcomes subsequent the use of ambroxol in these patients. These findings were consistent regardless of age, including for patients as young as 1 month old. Moreover, approximately all studies revealed that ambroxol was a well-tolerated agent in children. Therefore, these clinical evidences of efficacy for ambroxol established it as a potent agent offering significant benefits to pediatric patients for its licensed use as a secretolytic therapy in acute and chronic bronchopulmonary disorders, owing to its potential of improving both abnormal mucus secretion and its impaired transport. 

The review included active-controlled studies which demonstrated that the efficacy of ambroxol is non-inferior to that of N-acetylcysteine, carbocysteine/S-carboxymethyl cysteine and sobrerol. It was more potent at relieving symptoms and signs of acute and chronic respiratory disease with a more rapid onset of action. In addition, evidences have suggested that ambroxol can act synergistically with antibiotics, causing faster resolution of symptom in contrast to treatment with antibiotics alone. 

The real world evidence data showed that favourable efficacy and safety of ambroxol in a total of 3,629 patients (including over 244 children) as a non-prescription medication in a heterogeneous population within a real-life, day-to-day setting. In a study conducted by Kardos et al. revealed that ambroxol rapidly reduced Bronchitis Severity Scale scores and ameliorated bronchitis-associated impairments. Furthermore, very few adverse events were seen and almost all patients (97%) rated the global tolerability of ambroxol as ‘good’ or ‘very good’.

In another review done by Zhang et al., no adverse effects on fetuses or neonates were found following treatment with ambroxol. Moreover, high-dose ambroxol treatment did not lead to any adverse events in children aged 1 month-12 years with acute respiratory distress syndrome (ARDS).

Source: Kantar A, Klimek L, Cazan D, Sperl A, Sent U, Mesquita M. An overview of efficacy and safety of ambroxol for the treatment of acute and chronic respiratory diseases with a special regard to children. Multidiscip Respir Med. 2020;15(1):511. Published 2020 Mar 3.