Montelukast moderately decreased asthma symptoms in children with persistent asthma

Question: In children with persistent asthma, is montelukast a well-tolerated and effective therapeutic option?

Design: Randomized {allocation concealed*}†, blinded (patients, clinicians, and {data collectors}†),* placebo-controlled trial with 12-week follow-up.

Setting: 93 centers in Africa, Australia, Europe, North America, and South America.

Patients: 689 children who were 2 to 5 years of age (mean age 4 y, 59% boys, 56% white), had ≥ 3 episodes of asthma symptoms in the previous year, had a total asthma symptom score ≥ 1 (of 24) for ≥ 8 days during the 2-week placebo baseline period, and used β-agonists for ≥ 8 days during the placebo baseline period. Exclusion criteria included asthma intubation and emergency department treatment or hospitalization for asthma within 1 month before the study. Follow-up ranged from 98% to 100% for safety and from 81% to 96% for other outcomes.

Intervention: Patients were allocated to montelukast, 4-mg chewable tablet (n = 461), or to placebo tablet (n = 228). Tablets were given once daily at bedtime for 12 weeks.

Main outcome measures: Safety, asthma symptoms, and days with β-agonist use and without asthma.

Main results: Analysis was by intention to treat for {safety}† and effectiveness end points. The montelukast and placebo groups did not differ for frequency of overall adverse effects or individual adverse effects except for asthma, which occurred more frequently in the placebo group (30% vs 38%, difference 8.0%, 95% CI 0.18 to 16). The groups did not differ for discontinuation of treatment because of adverse effects (3.5% vs 3.1% {P > 0.999}‡). Groups did not differ for ≥ 1 laboratory adverse effect (3.6% vs 5.4%, {P = 0.31}‡). Montelukast decreased asthma symptom scores (Table) and days with β-agonist use (49% vs 55%, P = 0.001) and increased days without asthma (34% vs 28%, P = 0.002) more than did placebo.

Conclusion: In children with persistent asthma, montelukast was well tolerated, decreased asthma symptom scores and β-agonist use, and increased days without asthma.