Rationale: Antibiotic therapy for early Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF) is effective, but the optimal therapeutic regimen and duration for early treatment remains unclear. The EarLy Inhaled Tobramycin for Eradication (ELITE) study was designed to assess the efficacy and safety of two regimens (28 and 56 days) of tobramycin inhalation solution (TIS) 300 mg/5 mL (TOBI®) twice daily for the treatment of early onset P. aeruginosa infection in CF patients.
Methods: In this open-label, randomised, multicentre study, CF patients (aged ≥6 months) with early P. aeruginosa infection were treated for 28 days with TIS twice daily administered by the PARI LC PLUS™ jet nebuliser. After 28 days, patients were randomised 1:1 to either stop TIS (n=45) or to receive a further 28 days of TIS (n=43).
Measurements: The primary endpoint was the median time to recurrence of P. aeruginosa (any strain). Secondary endpoints included the proportion of patients free of P. aeruginosa infection one month after cessation of therapy and safety assessments.
Main results: The median time to recurrence of P. aeruginosa (any strain) was similar between the two groups. In total, 93% and 92% of the patients were free of P. aeruginosa infection one month after the end of treatment and 66% and 69% remained free after 27 months in the 28-day and 56-day groups, respectively. TIS was well tolerated.
Conclusions: Treatment with TIS for 28 days is an effective and well tolerated therapy for early P. aeruginosa infection in CF patients.