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Thread: taking Ivacaftor?

  1. #1
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    taking Ivacaftor?

    I just saw my healthcare team and was told that I qualify for an experimental treatment protocol, apparently Ivacaftor now covers 23 mutations and I have one of those mutations. I am still waiting to see if I get accepted into the study, but I was wondering if anyone is/has taken Ivacaftor and what you think of the medication. I have already had the discussion with my doctor but I really just want to hear from someone who has taken the medication

  2. #2
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    I've been taking it since 2014 - I'm sure there are a number of other people on here too. I heard they expanded the label for Kalydeco recently so I assume you have one of those mutations? I have a very rare residual function mutation and am unfortunately still off label (my mutation is too rare to appear in the CFTR2 database and hasn't been studied!). I've had a great experience on it - no side effects, my PFTs increased, sweat chloride decreased, gained weight, etc. It hasn't been a dramatic change but has definitely stabilized things. My maximum FEV1 used to top out around 60% and it's increased to 65% - and previously, when I had exacerbations my FEV1 used to drop into the 40s and 30s. Now, even when I'm feeling quite sick, I seem to always stay above 50% and recover quickly to baseline once I get on the right antibiotics. I always had very mild GI issues but they're now practically nonexistent (I require almost no enzymes). Hope this is helpful!

  3. #3
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    My advice is to try getting Kalydeco if you possibly can!

    I’ve been very fortunate in that my CF center prescribed Kalydeco for me off-label over three years ago. It’s been a real life changer. After 60 years, my cough and thick mucus production disappeared! It took less than a day to start feel its positive effects, and there haven’t any negative reactions that I’m aware of.

    As I’ve said before, it’s a shame that all CF centers were not as proactive as mine, and resisted to even try getting Kalydeco off-label for all their patients with Residual Function mutations. I don’t know if they were afraid to put in the extra effort that might have been required, or were just not familiar with the positive data that existed. My center was eventually successful in getting Kalydeco off label for six of the eight patients for whom they tried.

    My D1152H mutation was among those just approved by the FDA. While I never had any real problems getting it off-label, there were two instances when renewals were initially denied. Both times my CF center promptly submitted appeals, and the denials were reversed within two days.

    Again, I say go for it. Kalydeco doesn’t seem to have the adverse side effects reported for Orkambi.

    Good luck!
    75 Y/O with CF (D1152H and G542X) and Broncheiectesis.
    FEV1 Low 40s%.
    On Kalydeco since March 2014 and doing great.

  4. #4
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    If I read the paperwork I had to sign correctly this is supposed to be a trial, from what I recall they (the company) are trying to get the FDA to approve its use

  5. #5
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    I see - check out this link from Vertex (the manufacturer) that lists the 23 additional mutations that it was recently approved for: https://www.kalydeco.com/additional-mutations/

    I'd suggest discussing further with your doctor if your mutation is already approved!

  6. #6
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    Hi everyone. My daughter has D110H and will soon be starting kalydeco. She is 4 and has never been on antibiotics or been hospitalized. She only has cultured staph. Her doctor said now is the time to start this medicine. How will I know it's working?? (She isn't very symptomatic)

  7. #7
    My gene was on the recently approved list for Kalydeco! I started a week ago. It's working so well! Within a day, I had significant reduction in sputum, and much thinner. I can't wait for a PFT. I feel like it will be greatly improved. Good Luck!

  8. #8
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    we discussed it at length before I was ever given any paperwork to sign, I am just waiting to see if I get approved, my lung function isn't getting any better and only really gets worse when I'm sick, but I have been stuck in the 30's for the last 9 years, I am hoping that I get approved, anything to help my lung function

  9. #9
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    Jeannie85: Talk with her doctors, but I'd suggest doing a sweat test before and after starting it. It's a quantitative measure of improvement that might be useful if you have any issue with insurance companies in the future, and is generally good to know! That's great she is so healthy. Ideally, Kalydeco will keep things that way and prevent any damage to her lungs in the future. I have read the blog of a woman who also had a child start on Kalydeco at age 4 (http://luckycfmom.blogspot.com/) - as I recall, he was also not very symptomatic but they noticed a big improvement in his sinuses. I personally had an improvement in my GI issues (which were pretty minor to start with). Good luck - very exciting!

  10. #10
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    Jeannie85: definitely do sweat tests before and after starting ivacaftor (4-8 weeks after). You should also do blood work to get baseline values for liver function and pancreatic function (amylase, lipase). My daughter started at 7 in a similar situation - very healthy so no dramatic difference in her day to day life. But her sweat test came down to a NORMAL value (I still can't say that without yelling NORMAL), and her pancreatic values which had been a little elevated came back to normal as well. The liver function tests are to make sure that ivacaftor doesn't fuss her liver.

    Having my daughter's sweat test go from clearly diagnostic for CF down to normal lifted an enormous weight off me that I'd been carrying for seven years.

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