I am wondering if any of you out there taking Kalydeco that do not have G551D had before and after sweat chloride checked. And if so would you mind sharing your experience with me? Abby will be getting her post Kalydeco sweat test done the latter part of July and was just kind of wondering what you all have experienced. Thanks so much!!!
Off Label Kalydeco Question
- Thread starter JENNYC
- Start date
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marcijo
Guest
Sure-I am off label-mine was 81 before I started Kalydeco, and 36 after 4 weeks on Kalydeco! I am DF508 and R117H.
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marcijo
Guest
ccsalema-you are correct-they are starting testing on R117H any day now. My clinic said my sweat chloride level drop was more than their other patients who are G551D!
Yes-I venture to say that it does work on R117H as well as G551D - I virtually have no cough anymore-and don't cough up any gunk! After a month my FEV1 only went up 4% (from 83% to 87%).....so I am hoping for a bigger jump when I go back in about 3 weeks!
I can go into a quiet room and not worry about freaking other people out with my cough...it is amazing.
Yes-I venture to say that it does work on R117H as well as G551D - I virtually have no cough anymore-and don't cough up any gunk! After a month my FEV1 only went up 4% (from 83% to 87%).....so I am hoping for a bigger jump when I go back in about 3 weeks!
I can go into a quiet room and not worry about freaking other people out with my cough...it is amazing.
Go Kalydeco!!! Marci, that is amazing about your sweat chlorides!!! I do hope you see better PFT's!! I was pleasantly surprised last night....I got home from work and had Abby do a few PFT's for me and this is what she did.........1.52.......1.51........1.49!!!!! SO EXCITED!!!!
kittenface
Member
Marcijo - You have the same gene combination that I do. I was diagnosed at 37 and I'm now 47.
Have had lung infections, sinus infections etc and haven't had to take meds for my digestion at all. I'm now starting to have issues with my digestion and this last Xmas I had the worst lung infection ever. Of course the CF clinic was closed so I had to go over the holidays hardly being able to breath. How are you doing with your CF symptoms? My lung function is now normal - 3 years ago went down very slightly - just dropping 7%.
What does the medication do for you? what are the side effects? I live in Canada and have a great drug plan through my company. Thank you
Have had lung infections, sinus infections etc and haven't had to take meds for my digestion at all. I'm now starting to have issues with my digestion and this last Xmas I had the worst lung infection ever. Of course the CF clinic was closed so I had to go over the holidays hardly being able to breath. How are you doing with your CF symptoms? My lung function is now normal - 3 years ago went down very slightly - just dropping 7%.
What does the medication do for you? what are the side effects? I live in Canada and have a great drug plan through my company. Thank you
CyrilCrodius
New member
kittenface, the only way you could have access to it in Canada is through the Special Access Programme. Unfortunately, their decision making process is based on evidence rather than simply on safety.... so your doctor would have to provide evidence that it would work on you for you to have access to it. Yes... it's very dumb because such evidence doesn't exist.
Worth a try though. If you successfully gain access to it, do post an update, there are many in Canada who would like to have it.
Worth a try though. If you successfully gain access to it, do post an update, there are many in Canada who would like to have it.
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marcijo
Guest
JennyC-yes-it is pretty awesome. I have a clinic appt on Aug 9th-anxious to see if my PFT's have improved more!!
Kittenface-I posted in your DF508/R117H post as well! I don't take any enzymes for digestion either - so far I am pancreatic sufficient. My CF symptoms are pretty much non existent now that I am taking Kalydeco-it is amazing. I hardly cough at all anymore! I was on oral meds probably every 6-8 weeks or so for lung infections before I started Kalydeco-sometimes more in the winter. I used to cough up mucus everyday-now I don't at all.
My lung function (FEV1) is at 87% (was at 83% before I started Kalydeco). I am hoping to see that improve at my next clinic appt in a couple of weeks. What number is your FEV1?
I have had no side effects while using Kalydeco - it is truly a miracle drug. I am hoping you are able to have access to it-because it makes such a huge difference.
Kittenface-I posted in your DF508/R117H post as well! I don't take any enzymes for digestion either - so far I am pancreatic sufficient. My CF symptoms are pretty much non existent now that I am taking Kalydeco-it is amazing. I hardly cough at all anymore! I was on oral meds probably every 6-8 weeks or so for lung infections before I started Kalydeco-sometimes more in the winter. I used to cough up mucus everyday-now I don't at all.
My lung function (FEV1) is at 87% (was at 83% before I started Kalydeco). I am hoping to see that improve at my next clinic appt in a couple of weeks. What number is your FEV1?
I have had no side effects while using Kalydeco - it is truly a miracle drug. I am hoping you are able to have access to it-because it makes such a huge difference.
baseballfrank
New member
MarciJo,
This is the best post I have ever seen. I can't beleive what I just read! You have no CF symptoms? That is amazing!!! You don't have to take enzymes any more? You have no mucous? I can't tell you how excited I am. I am trying to get in on the Phase 3 Kalyedeco/VX 809 trials.
How long have you been on Kalydeco? Please keep sharing this fantastic news.
This is the best post I have ever seen. I can't beleive what I just read! You have no CF symptoms? That is amazing!!! You don't have to take enzymes any more? You have no mucous? I can't tell you how excited I am. I am trying to get in on the Phase 3 Kalyedeco/VX 809 trials.
How long have you been on Kalydeco? Please keep sharing this fantastic news.
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marcijo
Guest
Not really baseballfrank - it's amazing! The main things are that I don't cough much at all, and I don't cough up gunk anymore-I used to cough up gunk from my lungs daily!
I have been on Kalydeco since the end of March/early April (can't remember the exact date) <img src="i/expressions/face-icon-small-smile.gif" border="0">
I have been on Kalydeco since the end of March/early April (can't remember the exact date) <img src="i/expressions/face-icon-small-smile.gif" border="0">
Hi I just joined this because I saw marcjo had a the same issue as me. I have been on Kalydeco for 8 months and the company I work for changed insurance companies (from Oxford to Cigna) and Cigna will not approve the Kalydeco for me because I don't have G551D. My doctor and I put an appeal in which was then denied and then the final stage is the peer to peer review which took place last night between my doc and Cigna's Medical Director. I saw this post about the peer to peer review getting approved so I thought I would. However, I was denied eventho my doctor said the medical director was knowledgeable about CF and was going to approve it. I am very very upset (pretty much flipping out) and I will not stop until this is approved. Has this happened to anyone else and what else can I do to get this approved? Does anyone know what was the procedure after the Medical Director approved it, is their someone higher than that? I am not going to stop calling Cigna until I get an answer on this.
Anyway a little history on me, I was diagnosed with CF at 16, I am now 26, my mutations are R347P and L1065P (both very rare). Does anyone else have these mutations?? R347P is class 4 gating mutation and I googled L1065P and only found one case of it in Southern Italy and the patient had mild CF. I am mild CF and pancreatic sufficient. My pfts range from 75% - 81% for the past 3 years. I run 4-5 times a week and work a full time job, I try not to let CF control my life.
After being on Kalydeco I have improved my lung function slightly, I wake up in the morning with no gross thick dark mucus to cough up, and my sweat chloride went from 95.9 to 85 (still not normal, but this was after only 2 months on it, I haven't redone it). But while being on Kalydeco I am able to live a better qualtiy of life and do my vest less and I am currently on no other meds. I definitely see an improvement and have the documents to prove it to the insurance company. Please any advice/feedback would be greatly appreciated!
Thanks!
Anyway a little history on me, I was diagnosed with CF at 16, I am now 26, my mutations are R347P and L1065P (both very rare). Does anyone else have these mutations?? R347P is class 4 gating mutation and I googled L1065P and only found one case of it in Southern Italy and the patient had mild CF. I am mild CF and pancreatic sufficient. My pfts range from 75% - 81% for the past 3 years. I run 4-5 times a week and work a full time job, I try not to let CF control my life.
After being on Kalydeco I have improved my lung function slightly, I wake up in the morning with no gross thick dark mucus to cough up, and my sweat chloride went from 95.9 to 85 (still not normal, but this was after only 2 months on it, I haven't redone it). But while being on Kalydeco I am able to live a better qualtiy of life and do my vest less and I am currently on no other meds. I definitely see an improvement and have the documents to prove it to the insurance company. Please any advice/feedback would be greatly appreciated!
Thanks!
Aboveallislove
Super Moderator
Natalie B,
There's a Phase II study that you should qualify for (under third criteria of diagnosis after 12 and having a gating mutation) if the appeal doesnt work and once you finish it is "open label" extension so you could keep taking it. So sorry you are facing this.
hugs.
There's a Phase II study that you should qualify for (under third criteria of diagnosis after 12 and having a gating mutation) if the appeal doesnt work and once you finish it is "open label" extension so you could keep taking it. So sorry you are facing this.
hugs.
Natalie I am so glad you posted your experience! It hasn't crossed my mind yet about my husbands work changing insurance companies!! I even stopped midway through typing this and immediately typed Michael's boss an email!! I believe they usually change insurance in October!! I am so very thankful you posted this!! I would assume that the cost of the insurance is going to go up thanks to us! I'll be saying some extra prayers tonight!!!
musclemania70
New member
Kalydeco is not effective on people with DDF508.
It's not effective on most DDF508. I went to a CF education day last weekend and asked the Vertex Rep point blank, and she said it did have an effect on some, just not as good as the G551D, and they have no explanation as to why. For instance these things that we are seeing with Abby, is it the DF508 or her stop mutation 2585delT that is getting some protein up to the cell surface? I don't know, all I know is her quality of life has improved. And I am one thankful momma. 
Well God was quick to answer that prayer!! Michael boss just called back and told me that they reevaluate insurance once every 5 years and it was this year and they did that on Oct 1st and decided to stay with the insurance that we have for the next 5 years!!! Thank you God!! And Natalie
Hi NatalieB,
I'm so sorry to hear about your denial. My 14 yo daughter with CF is on Kalydeco off label as well. She is F508del and 621 +1G>T (splicing mutation). She was on Kalydeco for one month in April before prior authorization was required through BCBS. Her FEV1 went up 11% in one month (from 46% to 57%). She was denied access for 3 months, even after a 4 page formal appeal from our doc. In only 2 months off of Kalydeco, her FEV1 fell to 37%. Finally, one day in July when she had a coughing spell that lasted about 10 minutes straight, I was distrought and I decided to call the pharmacy myself (BCBS uses Medco/Accredo) and I asked to speak to someone in the appeals dept. I got a pharmacist who patiently listened to me and I literally broke down telling him our story. At that time I not only had the positive results of Kalydeco, but the negative ones from being off of it as well. He asked that I send the data directly to him and label it as "additional information to first appeal." That was on a Friday and by the following Wednesday, we had approval! However, the approval runs out this month and we are submitting new data again this week and praying that the approval goes through once again. Since July 20th, my daughter's FEV1 has improved 24%! Last week she was 61% FEV1 on her PFT. She had lost 3 pounds in May and June and has not only gained that weight back, but 10 more to go with it for a total of 13 pounds. She has also grown 1.5 inches in height in 10 weeks and ran a 5K on August 29th. The difference in this child is amazing. I will not go down without a fight if she is denied again! So, all of this to say, don't give up. Call and ask for someone in the appeals department. Send them your data and I hope you don't have to wait until you decline, like we did, before someone sees the light. However, if you continue to be denied, I would STRONGLY recommend getting PFT's monthly and getting your sweat chloride retested since you saw a drop. It is really all about the data in the end to those who are just looking at cost and numbers. I wish you all the best and hope you get good news very soon!
I'm so sorry to hear about your denial. My 14 yo daughter with CF is on Kalydeco off label as well. She is F508del and 621 +1G>T (splicing mutation). She was on Kalydeco for one month in April before prior authorization was required through BCBS. Her FEV1 went up 11% in one month (from 46% to 57%). She was denied access for 3 months, even after a 4 page formal appeal from our doc. In only 2 months off of Kalydeco, her FEV1 fell to 37%. Finally, one day in July when she had a coughing spell that lasted about 10 minutes straight, I was distrought and I decided to call the pharmacy myself (BCBS uses Medco/Accredo) and I asked to speak to someone in the appeals dept. I got a pharmacist who patiently listened to me and I literally broke down telling him our story. At that time I not only had the positive results of Kalydeco, but the negative ones from being off of it as well. He asked that I send the data directly to him and label it as "additional information to first appeal." That was on a Friday and by the following Wednesday, we had approval! However, the approval runs out this month and we are submitting new data again this week and praying that the approval goes through once again. Since July 20th, my daughter's FEV1 has improved 24%! Last week she was 61% FEV1 on her PFT. She had lost 3 pounds in May and June and has not only gained that weight back, but 10 more to go with it for a total of 13 pounds. She has also grown 1.5 inches in height in 10 weeks and ran a 5K on August 29th. The difference in this child is amazing. I will not go down without a fight if she is denied again! So, all of this to say, don't give up. Call and ask for someone in the appeals department. Send them your data and I hope you don't have to wait until you decline, like we did, before someone sees the light. However, if you continue to be denied, I would STRONGLY recommend getting PFT's monthly and getting your sweat chloride retested since you saw a drop. It is really all about the data in the end to those who are just looking at cost and numbers. I wish you all the best and hope you get good news very soon!