10 THERAPY New therapy for cystic fibrosis A short term, preliminary study has shown that ' ... treatment with aerosolized rhDNase {recombinant human deoxyribonuclease II is an effective method of improving lung function in patients with cystic fibrosis'. However, because of its mechanism of action, this therapy is likely to be effective only in patients with purulent sputum. An initial dose titration study in 16 patients with cystic fibrosis, using aerosolised rhDNase 0.1 to 20 mg/day (n = 3). 20 mg/day, 10mg tid or 20mg bid. showed a consistent elevation of forced vital capacity (FVC) and FEV I with increasing dose. FVC and FEV I significantly increased from baseline with the latter 3 dose schedules. and II patients noticed an improvement in their breathing ability. Cleaved DNA was noted in sputum after treatment. demonstrating biological activity by rhDNase. A placebo-controlled study was then initiated in 16 patients who received rhDNase IOmg bid and placebo in a crossover fashion; only II patients completed both treatments. Sputum volume increased similarly with both treatments; however. aerosolised rhDNase significantly improved FVC and FEV I in the morning and the early evening. compared with baseline and placebo therapy. One week after therapy completion, FVC and FEV I had declined towards baseline and analysis after 3 months showed no residual effects of therapy. Hubbard RC. McElvaney NG. Birrer P. Shak S. Robinson WW. et al. ..\ preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. New England Journal of Medicine 326: 812·815. 19 Mar 1992 4 Apr 1992INPHARMA® ISSN 0156-2703/92/0404-0010/$100/0 © Adi51nternational lid

New therapy for cystic fibrosis

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10 THERAPY

New therapy for cystic fibrosis A short term, preliminary study has shown

that ' ... treatment with aerosolized rhDNase {recombinant human deoxyribonuclease II is an effective method of improving lung function in patients with cystic fibrosis'. However, because of its mechanism of action, this therapy is likely to be effective only in patients with purulent sputum.

An initial dose titration study in 16 patients with cystic fibrosis, using aerosolised rhDNase 0.1 to 20 mg/day (n = 3). 20 mg/day, 10mg tid or 20mg bid. showed a consistent elevation of forced vital capacity (FVC) and FEV I with increasing dose. FVC and FEV I significantly increased from baseline with the latter 3 dose schedules. and II patients noticed an improvement in their breathing ability. Cleaved DNA was noted in sputum after treatment. demonstrating biological activity by rhDNase.

A placebo-controlled study was then initiated in 16 patients who received rhDNase IOmg bid and placebo in a crossover fashion; only II patients completed both treatments. Sputum volume increased similarly with both treatments; however. aerosolised rhDNase significantly improved FVC and FEV I in the morning and the early evening. compared with baseline and placebo therapy. One week after therapy completion, FVC and FEV I had declined towards baseline and analysis after 3 months showed no residual effects of therapy. Hubbard RC. McElvaney NG. Birrer P. Shak S. Robinson WW. et al. ..\ preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. New England Journal of Medicine 326: 812·815. 19 Mar 1992

4 Apr 1992INPHARMA® ISSN 0156-2703/92/0404-0010/$100/0 © Adi51nternational lid