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DNase

Question
I've just come back from a routine clinic and the consultant wants to put my son (aged 6) on DNase daily for evermore. Do you have any feedback about this? Or still have the feature ask the expert? Apparently they use it in the USA and Europe from 6 or younger, and they feel that lung function is improved for the long-term. Obviously this is a huge decision for me, and I don't want to make a snap decision. My son's lung function is generally good, usually has half dozen courses of antibiotics annually, and has only been admitted for IVs once when he was 2.

I hope you can advise accordingly on how evorn-cf feels about it.
Answer
Dear questioner,
you want have a second opinion about the intention of your CF consultants to put your son on daily inhalation of DNAse.
First of all, it has to be said that it is not possible to make concrete medical treatment decisions via the internet, as we do not know your son, all the details about his illness (lung function values, microbiology results, x-ray etc.) and we do not even know the development of the course his illness. Therefore, the decision for or against the inhalation of DNase (Pulmozyme®) can only be definitely made by the doctors in charge of your son.
However, we will try to give you some background information to make it easier for you to understand this therapeutic regimen.
Pulmozyme® (rh-DNAse) is an inhalative mucolytic drug, that is officially licensed for the treatment of children who are 5 years of age or older. Detailed information about the conducted studies with this drug and the conclusion that can be derived of those can be found in the “Cystic Fibrosis Pulmonary Guidelines” from the American Cystic Fibrosis Foundation (Flume et al in the Am J Respir Crit Care Med 2007, Vol 176:957-969: “Cystic Fibrosis Pulmonary Guidelines. Chronic Medications for Maintenance of Lung Health.” Latest update April 2013).
1. Dornase alpha with mild disease (severity of lung disease is defined by FEV1 % predicted as follows: normal >90% predicted; mildly impaired 70-89% predicted): For individuals with CF, 6 years of age and older, with asymptomatic and mild lung disease, the CF Foundation recommends the chronic use of dornase alpha to improve lung function and reduce exacerbations. Certainty of net benefit is high (so enough or much data available), the estimate of net benefit is moderate and the grade of recommendation: B.
2. Dornase alpha with moderate to severe disease (severity of lung disease is defined by FEV1 % predicted as follows: moderately impaired 40-69% predicted; severely impaired under 40% predicted): For individuals with CF, 6 years of age and older, with moderate to severe lung disease, the CF Foundation strongly recommends the chronic use of dornase alpha to improve lung function, improve the quality of life and reduce exacerbations. Certainty of net benefit is high (so enough or much data available), the estimate of net benefit is substantial and the grade of recommendation: A.
So the chronic use of dornase alpha is recommended to children over 6 with CF, even more strongly, if lung disease is advanced.
Usually, Pulmozyme® is well tolerated, however, side effects like bronchospasm, cough and hoarseness can occur, so sometimes inhalation of a bronchodilator before is necessary.
As the chronic use of inhalative mucolytic drugs is usually necessary in CF, a potential alternative to DNAse as a mucolytic is the inhalation of hypertonic saline (we do not know if your son already inhaled this so far and the physicians in charge want to switch for some reasons). For the inhalation with hypertonic saline there is not so much data available compared to DNAse, it is also recommended by the CFF for CF patients over the age of 6, the certainty of net benefit is moderate, the estimate of net benefit is also moderate and therefore the grade of recommendation: B.
As mentioned above, the decision for Pulmozyme® is dependent on many individual factors of the course of the illness, so that only your team can decide on it. We hope to have given you some background information that makes it easier for you to discuss this with your team.
Yours sincerely,
Dr. Daniela d’Alquen (Coordinator of the central English Archive of ECORN-CF)
10.03.2014