The Efficacy & Safety of Once Daily Pulmicort Respules™ (Budesonide Nebulizing Suspension; BNS) and Placebo (PBO) in Infants and Young Children with Persistant Asthma † 1948

Introduction: The current inhalation devices used to deliver inhaled glucocorticosteroids (iGCS) are not optimal for use by infants or young children with asthma. BNS (Pulmicort Respules™) is a nebulizable iGCS for use in this patient population. A randomized, double-blind, PBO-controlled, multicenter, 12-week study assessed the efficacy and safety of once daily (QD) BNS in infants and young children with persistent asthma not well-controlled on non-iGCS therapies.

Methods: After a 2-week baseline, 359 patients (5 months - 8 yrs) were randomized to BNS (0.25, 0.5 or 1.0 mg QD) or PBO for 12 weeks. Efficacy variables were patient diary recordings of nighttime and daytime asthma symptom scores (0-3 scale) and number of days use of bronchodilator medication. For patients able to perform the maneuvers, a.m. and p.m. peak expiratory flows (PEF) and spirometry were also assessed. Safety variables were adverse events (AEs), changes in physical exams, clinical laboratory tests, oropharyngeal/nasal fungal cultures, and pre- and post-ACTH-effects on a.m. plasma cortisol levels.

Results: All three BNS treatment groups showed clinically and statistically significant improvements in nighttime and daytime asthma symptom scores, and in the number of days use of bronchodilator medication compared to PBO (p≤0.050). All three BNS treatment groups also showed clinical improvements in FEV₁, with the 0.5 and 1.0 mg BNS QD treatment groups achieving statistical significance versus PBO (p≤0.050). Furthermore, there were no clinically relevant differences between BNS and PBO in the type, incidence or severity of AEs, or in physical exams, clinical laboratory tests, or oropharyngeal/nasal fungal cultures. Finally, analyses of pre- and post-ACTH effects on a.m. plasma cortisol levels reveal no evidence of HPA-axis suppression by BNS after 12 weeks.

Conclusions: These data show that BNS administered once daily is safe and efficacious compared to PBO in the treatment of persistent asthma in non-iGCS-dependent infants and young children. Once daily BNS therapy may be of particular importance with respect to compliance and convenience in this difficult to treat population.