The effect of dornase alfa on ventilation Inhomogeneity in patients with Cystic Fibrosis.

• Outcome measures to assess therapeutic interventions in cystic fibrosis (CF) patients with mild lung disease are lacking. Our aim was to determine if the Lung Clearance Index (LCI) can detect a treatment response to dornase alfa in paediatric CF patients with normal spirometry. CF patients between 6 and 18 years of age with FEV1% ≥80% predicted were eligible. In a crossover design, 17 patients received 4 weeks of dornase alfa and placebo in a randomized sequence separated by a 4-weekwashout period. The primary endpoint was the change in LCI from dornase alfa versus placebo. A mixed model approach incorporating period-dependent baselines was used. This trial was registered with ClinicalTrials.gov, number NCT00557089. The mean age±SD was 10.32±3.35 years. Dornase alfa improved LCI versus placebo (0.90±1.44, p=0.022). Forced expiratory flow at 25-75% expired volume (FEF25-75) measured by percent predicted and z-scores also improved in subjects on dornase alfa (6.1%±10.34, p=0.03; and 0.28 z-score±0.46, p=0.03). Dornase alfa significantly improved LCI. Therefore the LCI may be a suitable tool to assess early intervention strategies in this patient population.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Pediatrik (hsv//swe)

MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Pediatrics (hsv//eng)

Nyckelord

Children

cystic fibrosis

dornase alfa

lung clearance index

spirometry

ventilation inhomogeneity

Publikations- och innehållstyp

ref (ämneskategori)

art (ämneskategori)