no changes on Kalydeco

[echolaura]

My daughter has been taking Kaly for close to a year, and she is a G551d and a possibly nonsense gene. She hasn't had any obvious changes at all. Not sweat, PFT's, or symptoms. Anybody else out there?

 

[Aboveallislove]

i know some folks' changes aren't as dramactic but most have some improvement. i don't know if the studies showed as subgroup who didn't have any benefit...you might ask your child's doctor or even look in packaging info to see percentage who don't have improvement. also you might consider whether it could be better optimized re dosing and fat and also if any interactions. This blog has some great info:
http://magicbluepill.wordpress.com/info-for-people-on-kalydeco/

 

[JENNYC]

So you can't tell a difference in cough or sniffles? May I ask how old she is? I'm so sorry.

 

[keefer11]

I'm also curious what her PFT's were before she began Kalydeco?

 

[GenH]

I'm also curious what her PFT's were before she began Kalydeco?

And if her pfts did not vary much before she started? The reason I am wondering what her pfts were like pre Kalydeco is because many people have reached their 6 month to 2 year high. For those that have been at the same point for a while they generally have not improved as much, but are more stable.

Has she been more stable whilst on Kalydeco and needed less antibiotics?

Digestion, absorption and drug metabolism could all affect the amount of Kalydeco- we have limited control over most of these factors but is she having it with a reasonable amount of fat, approx every 12 hours?

 

[echolaura]

No changes in cough at all. Her PFTs declined rapidly in the two years prior to initiating Kalydeco, and haven't bumped back up much. There are other factors I should have included in my first note...On her own she decided to stop Cayston, and that could be playing a role in this. She is a young adult now and I'm not actually "invited" to her CF appointments anymore, which actually coincided with starting Kaly. There are probably alot of things I need to look into, such as her sputum cultures. She doesn't take it twice a day, she takes it once a day. And the only place I have heard about taking it with fatty foods is here on this forum, not thru my daughter. checking "magicbluepill" now, thank you for sending that.

 

[Aboveallislove]

oh, mom. This must be so hard on you wanting to ring her neck for throwing away what could really get her in great shape, but knowing she is an adult and you have no control. Have you read Lisa Greene's book on Parenting Children with Special Needs? If not, I'd get it today before you talk with DD and then formulate some ideas of how you can approach to have a discussion. "I was wondering if I might share with you some of the things I read about others with Kalydeco." "What do you think about X." etc.

 

[GenH]

No changes in cough at all. Her PFTs declined rapidly in the two years prior to initiating Kalydeco, and haven't bumped back up much. There are other factors I should have included in my first note...On her own she decided to stop Cayston, and that could be playing a role in this. She is a young adult now and I'm not actually "invited" to her CF appointments anymore, which actually coincided with starting Kaly. There are probably alot of things I need to look into, such as her sputum cultures. She doesn't take it twice a day, she takes it once a day. And the only place I have heard about taking it with fatty foods is here on this forum, not thru my daughter. checking "magicbluepill" now, thank you for sending that.

The transition period is quite difficult, I dropped about 10% when I transferred and luckily started taking better care of myself and my pfts stabilised. I can imagine the transition would be hard to manage if you also start Kalydeco at the same time. If she is interested, perhaps it would help her to read about other people on Kalydeco so she can see what they have done to improve and what treatments they need now? I write the magicbluepill blog & I have tried to add as much info as possible about Kalydeco. I also have many links to other Kalydeco blogs on the right side of my blog. If she is interested there are also facebook groups such as Kalydeco Miracles which she could join so she can talk to other people on Kalydeco: www.facebook.com/groups/359954544023650/

Is she on another medication which means she has to have it once a day? The normal dose is twice a day, with 10-20g of fat each time.

 

[nmatthews]

I have been on Kaly for a year and the change has been pretty amazing. Your daughter should be taking her pill every 12 hours with a fatty meal. This helps absorption. If she is not taking it as perscribed then that is most likely the reason for no changes. My lung functioning had declined steadily for several years prior to beginning the Kaly, and was at about 50% with me being in the hospital every 3 months. I would have to do 3 full (pulmo and hypertonic) treatments a day and rotated Tobi and Collistin months also. Upon starting the Kaly within a few days I was able to breath much better and could reduce my treatments to 2x a day and felt great between them. This pill is NOT a cure, and our lungs will still have the bacteria and staph that we've always grown, so it is still necessary to do treatments and use antibiotics as well, hopefully hospitalizations will lessen and the progression of the disease will slow. I am currently at a baseline PFT of 68 and am hospitalized only every 8 months or so. I feel better between treatments and do only 2 a day, but again use collistin and tobi inhaled to keep the staph and bacteria at bay. Your daughter is welcome to contact me if she'd like to chat! Best wishes.