PURPOSE OF THE STUDY.
To investigate the efficacy of montelukast in the treatment of young pediatric patients with episodic asthma.
Children aged 6 months to 5 years with a history of asthma symptoms during the past year and symptom-free periods between episodes were included in this multicenter (111 sites), randomized, double-blind, double-dummy trial that took place from November 2006 to August 2009.
Patients were randomized to 1 of 3 groups: daily montelukast plus intermittent, episode-driven placebo; daily placebo with montelukast given during episodes; or daily placebo with placebo given during episodes. Doses were given at night. Episode-driven medications were given for 12 days from the start of the episode. All patients could use short-acting β-agonists as needed for symptom relief. Action plans were given to the families. On a daily basis, symptom calendars were completed by parents, which included respiratory symptoms and use of medication for the treatment. During episodes of asthma, questions included day and night symptoms, use of asthma medications, limitation of activity, and use of health care resources. The primary endpoint was the number of episodes leading to an asthma attack that led to utilization of health care resources. Other endpoints were measured, including symptoms during the 3 days preceding an attack and during the 12-day treatment period, percentage of asthma-free days, and use of β-agonist.
Almost 600 patients were randomized to each treatment arm. Almost 75% of patients had a least 1 episode of asthma, with almost half of patients culminating in an exacerbation. No significant difference was seen between groups in the number of episodes leading to asthma exacerbations. Daily montelukast reduced symptoms over the 12-day treatment period of asthma episodes compared with placebo (P = .045). β-Agonist use was reduced with both daily (P = .048) and intermittent (P = .028) montelukast compared with placebo. However, because of prespecified rules for multiplicity adjustments (requiring a positive primary endpoint), statistical significance for secondary endpoints could not be concluded.
The number of asthma episodes leading to asthma exacerbations over a yearlong period in these young pediatric patients was not reduced by montelukast use, although improvements occurred in some endpoints.
The treatment of pediatric patients with severe intermittent asthma is challenging for clinicians. The optimal treatment for these patients is unknown; studies do not show benefits of preventive medications as in other forms of persistent asthma. This study did not show improvement in the number of asthma exacerbations requiring utilization of health care resources with the use of montelukast, although some secondary endpoints were suggestive. The need exists for further studies to improve the prevention of the morbidity seen during the asthma exacerbations of these patients.
- Copyright © 2012 by the American Academy of Pediatrics