Topics

Off-label use of Kalydeco
Dear physicians, I am a mother with a 3.5-year-old son suffering from CF, living in Germany. My son has been diagnosed at the age of 2.5 years. Overall, his health condition is good. However, the illness affects markedly the gastro-intestinal tract. He has had a long-lasting colonization with germs and has even been underweight after this for a short time. He has the very rare mutation G1244E and the more common one R553X. For the Mutation G1244E there is in my opinion the possibility, to treat him with Kalydeco. Until now, the drug is only licensed for children over the age of 6 in Germany, however on the 18th March 2015, the drug has been licensed for children under 6 years of age in the USA. We as worried parents would like to treat him with the drug off-label, however our Center is denying the prescription off-label with the rationale, that there was no data about the dosage. They do not want to take resposibility for this. I am very despaired and want to do everything that my son can take the drug. I do not understand, why the physicians are refusing so vehemently to prescribe the drug, if even on the homepage of Kalydeco the dosage is written (50 or 75 g granulate). Until now, we did not contact the health insurance however we talked about it in detail with Vertex and with a pharmacy in our home town. Both told us, that the health insurance could cover the costs for the drug, if a CF physician would prescribe it. We know another case of a child in Germany, in this case the father requested the off-label use for the drug and the health insurance agreed. The father posted a contribution on Ecorn. I was not successful in getting in contact with the parents of this child. Could you help us further? I would be very thankful for an answer.
28.06.2015
Kalydeco off label
Hello, my daughter has the mutations delF508 and D1152H. How great is the chance to get Kalydeco off-label? There are obviously in-vitro studies that show, that the CFTR channel is functioning nearly completely in case of intake? There is already a study from Denver about this? In the USA human beings with these mutations profit already from Kalydeco. Many thanks for your efforts.
18.06.2015
Forest manager
Can my son make a training to become a forest manager without worries or is there a big danger to get a germ or fungus?
18.06.2015
Germs in the pool
Hello, I would like to put a pool in the yard for our child with CF. What should I pay attention to in order to limit the risk of colonization with Pseudomonas or other germs? Should the pool have a certain size? Does chlorine kill these germs? If yes, which concentration is needed? It is obviously not possible to fill a bigger pool with fresh water each day! Thank you for answering.
18.06.2015
Port explantation
Dear experts, After 16 years my port was explanted for I did not need it for IV’s since 5 years any more. I decided to do this because I have optical problems and could not rinse it myself anymore as I had done it all the years before. This would have to be done in the hospital from time to time. Unfortunately, the explantation did not work well. The port tube tore off. Obviously it was so much adhered that it was not possible to remove the rest of the tube (~15 cm). Have you heard about such a case before? Which complications are involved? In retrospect, I ask myself if the port could have stayed unrinsed in the body? This would have saved me at least the infection risk of the operation. Best regards, M. N.
16.06.2015
Sweat test
Dear expert team, Our 4-year-old daughter has recurring obstructive bronchitis since three years. We were in the children’s hospital twice for having a sweat test done. The values were on the borderline (41 and 51 Na). The test that was done in the outpatient department was negative (16 Chloride). Can Cystic Fibrosis be excluded for sure? How can such differences of sweat test results be explained? Elastase detection was negative while she had never any problems with bowel movement! Many thanks for giving your opinion!
16.06.2015
Hygiene among siblings
Dear CF team, I am a female CF patient myself and have an older brother, who also suffers from CF. After having talked to my physician I pay attention to not using the same bathroom nor to be near the food of the other one if we meet at family meetings. That means, that we both do not prepare any food. This has great conflincts as a consequence, as my brother thinks I am exaggerating. However, my physician recommended it to me. Do we really exaggerate or do we follow the right hygienic measures?
16.06.2015
Disinfection of poolwater
Hello dear expert team, First of all many thanks that you are there with your advice. Now to my question. We will install in the summer a pool with a diameter of 5 m. Last year, the water had been disinfected with chlorine, that worked more or less well. Now we have heared about disinfection with active oxygen and about disinfection with salt. Hereby, the salt is put into the poolwater and is changed via a pump and a chemical reaction (I hope that I understood everything right); the salt content would be with this method at 0.4%. Could you give a recommendation, which procedure is the right one? Many thanks in advance.
16.06.2015
CF-diabetes
Does a very strong parncreatic insufficient CF patent will inevitably develop sometime a diabetes? Thus, is there a connection between a strong exocrine impairment and a strong endocrine impairment? If yes, at what timepoint should one investigate a very strong pancreatic insufficient child concerning a diabetes? Would a strong consumption of sugar support the occurrence of a diabetes? Many thanks for your help.
16.06.2015
Bronchiectases
Hello, Is it possible that existing bronchiectases heal up? Our child has bronchiectasis and after having moved to another place we have a new CF outpatient clinic. They say that regular antibiotics administration is outdated? Best regards
12.06.2015
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