Actually, there is at least one small study showing the effectiveness of Pulmozyme in children under the age of five (published in <u>Pediatric Pulmonology</u>, volume 32, pp 377-382, 2001.) Researchers from the University of Michigan treated patients ranging from 1 to 5 years old with either Pulmozyme or placebo for 100 days in a randomized double-blind clinical trial. The kids who got the Pulmozyme showed improvements on their X-ray and high-resolution CT scans, while the scores for those on placebo got worse. Also, parents of the kids on Pulmozyme reported decreased cough, increased activity level, improved appetite, and improved sleep.
As others have pointed out, Pulmozyme is used primarily to prevent lung damage from occurring, and there is plenty of evidence that it is important to start using it even before there is evidence of <u>any</u> lung deterioration. One study, also from <u>Pediatric Pulmonology</u> (volume 34, pp 237-241, 2002), found a 34% reduction in pulmonary exacerbations requiring iv antibiotics and improvements in both FEV1 and FEF25-75 over a 2-year treatment period in kids with <u>almost normal lung function</u> (FEV1 approx 95% and FEF25-75 approx 85% before treatment).
While Pulmozyme, like all drugs, is unlikely to work for everyone, there is no way that that it could produce these kinds of results in a long series of different studies (I'll get more citations if anyone wants) unless it was helpful for most cf patients. We started our son on it almost eleven years ago, when he was 6-1/2, and we consider it "liquid gold", well worth even its exhorbitant price. In fact, we consider it so important for preserving his health that he has <u>never</u> skipped a dose -- not even during the difficult days after our house was destroyed by fire. He says he can feel the difference even if he delays treatment for a few hours.
So I would say to the parents of young children with cf, please try the Pulmozyme before your child starts showing problems. By all means reconsider if you see significant side effects; but if not, even if you can't tell whether the treatments are working, you are very likely making an important investment in your child's long-term health.
Bambi, mom of Jordan, 17 w cf