Authors' objectives:

To analyse the current state of scientific knowledge regarding the safety, efficacy, and cost of recombinant human dornase in the treatment of cystic fibrosis patients. Methods: Literature review and information synthesis

Authors' results and conclusions: Recombinant human dornase is proposed as a possible new therapeutic alternative for the treatment of selected patients with cystic fibrosis. Various studies show that recombinant human dornase is both safe and efficacious in cystic fibrosis patients between the ages of 5 and 55 and whose forced vital capacity is greater than 40 percent of predicted value. Efficacy is principally defined in terms of the drug's ability to reduce the number of respiratory exacerbations suffered. Two different doses (a daily dose of 2.5 mg and a twice daily dose) of recombinant human dornase were compared with placebo in a phase III randomised controlled clinical trial. Using the fixed effects model to analyse data from that study, we established that recombinant human dornase, independent of dose, can lead to a statistically significant reduction in the number of respiratory exacerbations when compared to placebo. More published evidence is however required before definitive conclusions can be reached about the efficacy of recombinant human dornase given that there is only one single phase III clinical study that has been published in a medical journal. None of the articles revised specifically address issues of quality of life or cost.

Authors' recommendations: There exists insufficient scientific evidence to recommend the unlimited administration of recombinant human dornase to patients with cystic fibrosis. As this drug is currently under investigation, further trials incorporating different dosages, follow-up periods and issues such as quality of life and cost are desirable. Ideally, a comparison of the cost-effectiveness of recombinant human dornase and other available cystic fibrosis treatment alternatives should be carried out.

Project Status: Completed

URL for project: http://www.gencat.net/salut/depsan/units/aatrm/html/en/dir393/doc7921.ht ml

Year Published: 1995

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Spain

Organisation Name: Agencia de Qualitat i Avaluacio Sanitries de Catalunya

Contact Address: Antoni Parada, CAHTA, Roc Boronat, 81-95 (2nd floor), 08005 Barcelona, Spain, Tel. +34 935 513 928, Fax: +34 935 517 510

Contact Name: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net

Contact Email: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net

Copyright: Catalan Agency for Health Technology Assessment and Research