rhDNase in cystic fibrosis

Cystic fibrosis is a disease that is relatively rare but expensive for patients, families, and carers. The introduction of rhDNase has been associated with controversy as to its benefits and costs. Milla describes the experience in a centre that prescribed this drug to patients, 60% of whom had an FEV₁ of more than 80% predicted at the time of prescription.1 Overall, the group had an accelerated decline in lung function following its introduction. This study illustrates the importance of patient selection and follow up in the prescription of rhDNase.

Paediatricians and chest physicians from the South & West Region of the UK have audited their use of DNase as part of their contribution to the South & West Cystic Fibrosis database. In 1995 78 (12%) of the 664 patients receiving care within the region had been prescribed DNase. This had risen to 143 (22%) in 1996. We subsequently defined criteria for its use: patients over five years of age, FEV₁ <70% predicted, and more than one course of intravenous antibiotics during the previous year. In 1995 12 (17%) of patients receiving DNase did not appear to meet these clinical criteria. A further 36 patients who were eligible under these criteria were not receiving the drug.

Innes2 rightly emphasises the responsibility of carers to target this treatment effectively—it is also important that treatment is seen to be equitable and not dependent on postcode. Our experience illustrates that a regional cystic fibrosis database can be a clinically relevant and cost effective device for targeting appropriate treatment. The annual cost of DNase for two patients would be sufficient to fund a regional audit to monitor and influence this and other expensive treatments in patients with cystic fibrosis.