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Recombinant human DNase is an expensive mucolytic which does not benefit all patients with cystic fibrosis. Company sponsored trials in unselected cystic fibrosis patients have documented wide variability in spirometric responses to the drug, but the data are presented in a way which prevents the clinician from assessing which patients are likely to benefit.
We therefore read with interest the editorial by Dr Innes regarding the assessment of response to DNase in cystic fibrosis.1However, whilst we agree that it is necessary to target DNase, we have reservations regarding the use of “n-of-1 trials” for this therapy. Dr Innes states that this approach has been used in Scotland and quotes a study unpublished at the time of writing in support of it. However, this study has already …