CLINICAL BENEFIT FROM NEBULIZED HUMAN RECOMBINANT DNASE IN KARTAGENERS-SYNDROME

Nebulized recombinant human DNase (rhDNase) reduces sputum viscosity, improves pulmonary function, and results in a small reduction in acute respiratory exacerbations requiring intravenous antibiotics in many p atients with cystic fibrosis (CF). rhDNase is now recommended for use in CF patients with moderately severe suppurative lung disease. A 14-y ear-old girl with suppurative lung disease [forced expiratory volume i n 1 second (FEV(1)) 69% and forced vital capacity (FVC) 81% predicted] secondary to Kartagener's syndrome and severe gastroesophageal reflux had worsening spirometry together with intractable gastrointestinal s ymptoms over the previous 18 months despite conventional treatment. Sh e was, therefore, started on 2.5 mg rhDNase once daily. Her cough less ened and the volume of sputum decreased within 72 hours of commencemen t of treatment; this improvement was strongly associated with a dramat ic reduction in gastrointestinal symptoms. Spirometry after 4 weeks of treatment demonstrated a 20% improvement in FEV(1) and a 13% improvem ent in FVC. These improvements have been maintained after 4 months of rhDNase therapy. The use of rhDNase should be considered in patients w ith Kartagener's syndrome and a multicenter trial may be justified. (C ) 1995 Wiley-Liss, Inc.