She started taking Kalydeco on thursday. And so far we haven't noticed anything other than she is running 100 fever, which could be normal for her since she was starting to run a slight fever before we started. She has no cough, no sinus drainage, no nothing. I keep asking her if anything feels different and she says no but she is 7 and the child NEVER complains so I don't know. I can tell you that I have a Piko Meter and her PFT's were the highest they have ever been on the 15th (1.41) and when she started running a slight fever the highest she blew was a 1.35 the whole last week until Saturday. On Saturday she blew a 1.44!!! So I have no clue whether it is working or not. Good thoughts!! <img src="i/expressions/face-icon-small-smile.gif" border="0">
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Abby Update
- Thread starter JENNYC
- Start date
E
echolaura
Guest
Hi Jenny, My 18 year old never gave me feedback either, all these years, diagnosed at five, (asked DAILY how she feels! haha). so it came as no surprise to me when she (a g551d) started kaly and pretty much feels no different. She just 'stopped coughing' about the first week, but otherwise nothing physically has changed. Her PFTs have bumped up slightly. (fev1 1.9 in the fall is now 2.36) but she hasn't had the shock value recovery we anticipated. It's okay tho, I'll take recovery in any form! Good luck ABBY!!!!
CyrilCrodius
New member
Even at 23, I'm very very bad at noticing whether meds make a difference or not for me, unless it's very very obvious (like taking antibiotics) or I pay very close attention BEFORE so I can compare after.
Some people just aren't very self-aware.
Maybe you should make her run? (Once she got over whatever she got that is making her sick) and ask her if there is a difference in how much she coughs, is tired and out of breath she is at the end?
Some people just aren't very self-aware.
Maybe you should make her run? (Once she got over whatever she got that is making her sick) and ask her if there is a difference in how much she coughs, is tired and out of breath she is at the end?
Well Abby is on day 13 of Kalydeco. I am still not sure if it is working or not but these are some of the things that I have noticed. Her PFT's are up again!! She blew a 1.45, which is the highest she has ever done....that being said the next day she may only blow a 1.33...so not really sure if she is just not putting her all into it every time or what, but I'll take the 1.45 any day!! We live in Texas so we have had the normal crazy Texas weather of temperature and humidity changes, and she has also been swimming in the pool quite a bit also and I am pleased to report NO Coughs, NO sniffles, no anything!!! She is still running 100.0 from time to time. But normally after all of the weather changes and swimming she would have a cough or a sniffle or something!! So I am very anxious for the sweat test which will be the end of July sometime. I hope it shows improvement because she can't tell me if she feels different and her dr will not continue to give us a refill if her sweat tests aren't perfect. But I have not seen the sweat chloride numbers on the heterozygous 809 study to see how they did. The Vertex June 28th press release told that Homozygotes had a significant decrease in sweat chloride of 6.4 mmol/L.... I am not really good when it comes to scientific stuff but I try really hard to keep up. Does anyone have any clue what kind of drops that means? 20 points? 30 points? Back to normal sweat chloride?
Aboveallislove
Super Moderator
My understanding is that the 6.4 is points. So really not much at all. BUT I think you should push your doctor on it then and explain that there is an improvement and if not great, but her lung function is improving, her weight, and other symptoms, that that shows it is working. Afterall, they are moving the combo to Phase 3 even though SC only reduced about 10 points total (including part 2), and that the FEV improvement is more important. So if she has that improvement, that should justify continuing.
That's kind of what I thought, but was second guessing myself because they used the word "significant decrease" and 6 points is not significant to me either. Thanks for giving me your input on the subject. At least I can use this with her dr, I think I will have a fight on my hands if she doesn't drop by 20 points or more, but I will fight if I need to. <img src="i/expressions/face-icon-small-smile.gif" border="0">
Aboveallislove
Super Moderator
Jenny,
The total drop was actually about 10 points for the entire time period. When they say "significant" they mean statistically significant, meaning that the drop is most likely caused by the medicine as oppose to chance. The second drop wasn't statistically significant, meaning the addition 3+ drop could have been chance--or the meds. But I think the key is that even with a "small" drop, the FEV improvement was also very statistically significant. And since you can do FEV with Abby, if she continues to blow much higher--6 - 10%, I'd point out that is what those in study did and that was considered statistically significant. And also an increase in that amount justifies Pulmyzme, hyper-sal (for older patients), Cayston, etc. Good luck!
The total drop was actually about 10 points for the entire time period. When they say "significant" they mean statistically significant, meaning that the drop is most likely caused by the medicine as oppose to chance. The second drop wasn't statistically significant, meaning the addition 3+ drop could have been chance--or the meds. But I think the key is that even with a "small" drop, the FEV improvement was also very statistically significant. And since you can do FEV with Abby, if she continues to blow much higher--6 - 10%, I'd point out that is what those in study did and that was considered statistically significant. And also an increase in that amount justifies Pulmyzme, hyper-sal (for older patients), Cayston, etc. Good luck!
Ok so yesterday she blew a 1.46!! And more good news is she is due for refill on the 15th so I called insurance to see if I needed to get a prior authorization and they said no ma'am she is good for refills until June 2014!!!! I am one excited momma right now!!!! Watch out CF cause we are gonna kick your hiney!!!
Aboveallislove
Super Moderator
I LOVE that! Now make sure you order before your visit!! Have you tried to do the aquatic wrinkling test??? (I think many on Kalydeco indicated their hands stopped wrinkling so quickly. Average for CF is 3 minutes, carriers 7, and "normal" 10 (I THINK). Our son's wrinkle almost instanteously. Just something else that would be cool to see!!! SOOOO happy for you!! And also hope that your case will get other CFers with unknown mutations able to try too!!
No I haven't thought to try that, but I will be trying it when I get home!! Course I have no idea what she did before. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I would love for Abby's story to help others!! <img src="i/expressions/face-icon-small-smile.gif" border="0">
JennyC, that is so encouraging to hear about your daughter. What was her health status when she was put on it? I am wondering if Kal is only precribed when a patient is doing poorly or are doctor's giving it to everyone that has that mutation. Also, do you still have to do her normal treatments? Thanks so much!
CyrilCrodius
New member
Wait, they will give you refills for a year, no question asked about her second mutation? There are a few peeps who might be interested in what company you are with...
Aboveallislove
Super Moderator
Some thoughts on the "unknown" mutation and why kalydeco would have a good chance of working: I'd think that the more "severe" mutations would be more likely to be "known" ones because they'd be diagnosed sooner/in more people. Soooo, that would make me think that if there is still an unknown mutation, that mutation is much more likely one that has residual CFTR function, i.e., there IS protein on the cell surface, which would be why Kalydeco would be likely to work for those with unknown mutations. Thank God you pushed your doctor on this. I can understand why a doctor won't prescribe for a ddf508 (what our son has) and other mutations that are known not to have either protein or protein on the cell surface, but if it is unknown or one known to have protein on the cell surface, it should be tried--even if only for a month! What's the date for the SC??
cabadie1 Abby was and is pretty healthy, her dr only put her on it because he knew that I would not rest until he let her try it. And yes she is still doing all of her treatments as this is just a trial and we are not certain that it will or is working. I am trying to be skeptical but it's so hard!!! She is really doing good!!
CyrilCrodius actually 2 years!! We are so excited about that!! We have Humana, but from what I understand from others is it all depends on which Humana plan you have. My husband is a sheriff's deputy and has insurance through the county who has lots of employees so I'm sure that helps too.
Aboveallislove Abby's SC was over 100 on both arms and it was done on June 28, 2012. She was tested for the 50 most common mutations when she was 2. From what I understand they should have started testing for her second mutation. They said it could take up to 60 days once started. So frustrating since they received her blood on May 9th!! But nothing I can do about that. I promise the second I know I will share with everyone!! I will also let you know about her next sweat test coming up on July 26th!! We are so excited and hopeful!! I hope more are able to get on it and try it!! The only problem that we encountered was getting the dr to prescribe it, insurance didn't do anything but ask for Prior Authorization the one time and instantly approved it for 2 years!!! Her dr really was against letting her try it. He will be very critical with her results as well. But I am ok with that because he "did" give her the prescription and if he disapproves in the future and I feel like it was the meds that helped her. I will go else where since I now know that my insurance is good with it. <img src="i/expressions/face-icon-small-smile.gif" border="0">
CyrilCrodius actually 2 years!! We are so excited about that!! We have Humana, but from what I understand from others is it all depends on which Humana plan you have. My husband is a sheriff's deputy and has insurance through the county who has lots of employees so I'm sure that helps too.
Aboveallislove Abby's SC was over 100 on both arms and it was done on June 28, 2012. She was tested for the 50 most common mutations when she was 2. From what I understand they should have started testing for her second mutation. They said it could take up to 60 days once started. So frustrating since they received her blood on May 9th!! But nothing I can do about that. I promise the second I know I will share with everyone!! I will also let you know about her next sweat test coming up on July 26th!! We are so excited and hopeful!! I hope more are able to get on it and try it!! The only problem that we encountered was getting the dr to prescribe it, insurance didn't do anything but ask for Prior Authorization the one time and instantly approved it for 2 years!!! Her dr really was against letting her try it. He will be very critical with her results as well. But I am ok with that because he "did" give her the prescription and if he disapproves in the future and I feel like it was the meds that helped her. I will go else where since I now know that my insurance is good with it. <img src="i/expressions/face-icon-small-smile.gif" border="0">
Aboveallislove
Super Moderator
Thanks for all the info. I had forgotten they were testing for the other mutations....so she well could have one believed to benefit from Kalydeco and you were able to jump start the process--GREAT JOB MOM!! I would bet it is one of those too given her response and also my thoughts re the less common ones more likely have better function b/c not diagnosed as early. Give her an extra kiss and maybe have her send some extra prayers up for the double deltas waiting soooooo anxiously. Hugs!
Aboveallislove
Super Moderator
Thanks for all the info. I had forgotten they were testing for the other mutations....so she well could have one believed to benefit from Kalydeco and you were able to jump start the process--GREAT JOB MOM!! I would bet it is one of those too given her response and also my thoughts re the less common ones more likely have better function b/c not diagnosed as early. Give her an extra kiss and maybe have her send some extra prayers up for the double deltas waiting soooooo anxiously. Hugs!
OK... so you all are going to get tired of my updates but I am just so excited!! When I got home yesterday afternoon I had her do some PFTs for me and this is what she did: 1.52.......1.51........1.49 up from a starting PFT of 1.41!!!!!! She blew a 1.52 before but I didn't believe it because she could not reproduce it! 3 times in a row!!! Thank you Jesus and all of the wonderful people who made Kalydeco!! I could kiss you all!!! And I am praying that everyone else gets their miracles too. Thanks you all for the encouraging words!! And for all of you out there with the multiple mutations....PUSH...push your doctors, push your insurance, you never know until you try and I am so glad that we did!!