Kalydeco or VX-770 off lable use?

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Aboveallislove

Super Moderator
Musclemania70: I completely agree. Unfortunately our son is under 6 and I think doctors and insurance would never go for it b/c double off label. But as soon as it is approved for under 6 I am going to push. FWIW my approach is going to be that the studies show a sub-set benefits from this med. There are no known markers for who that subset is, so lets try it for 30 days to see what happens to SC and then go from there. I am still working on getting the research to back this up (Chest April 2012 published the 770 research for homo 508). I believe there are some insurance companies which have done that--approved 30 days--at least that's what I've read from other bloggers at places. Good luck! Oh, and from your other posts, I can tell you I hope our son grows up to be like you!!
 
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Aboveallislove

Super Moderator
Musclemania70: I completely agree. Unfortunately our son is under 6 and I think doctors and insurance would never go for it b/c double off label. But as soon as it is approved for under 6 I am going to push. FWIW my approach is going to be that the studies show a sub-set benefits from this med. There are no known markers for who that subset is, so lets try it for 30 days to see what happens to SC and then go from there. I am still working on getting the research to back this up (Chest April 2012 published the 770 research for homo 508). I believe there are some insurance companies which have done that--approved 30 days--at least that's what I've read from other bloggers at places. Good luck! Oh, and from your other posts, I can tell you I hope our son grows up to be like you!!
 
M

musclemania70

New member
Above all is love: That is the nicest thing a person has ever said to me. Thank you.

Do you have the link for the April chest research posting? I am searching the issue online now...
 
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musclemania70

New member
Above all is love: That is the nicest thing a person has ever said to me. Thank you.

Do you have the link for the April chest research posting? I am searching the issue online now...
 
A

Aboveallislove

Super Moderator
This is the link. http://chestjournal.chestpubs.org/
You have to pay for the article (only an abstract) and I haven't had a chance to do yet. I'll likely do in the next couple of weeks if you want to wait and I'll let you know if there is anything helpful to support the argument for coverage--sorry wish i could so sooner, but swamped now with work (from home) while doing full-time care with my dear son. Godspeed
 
A

Aboveallislove

Super Moderator
This is the link. http://chestjournal.chestpubs.org/
You have to pay for the article (only an abstract) and I haven't had a chance to do yet. I'll likely do in the next couple of weeks if you want to wait and I'll let you know if there is anything helpful to support the argument for coverage--sorry wish i could so sooner, but swamped now with work (from home) while doing full-time care with my dear son. Godspeed
 
R

Rummage

New member
There is a poster abstract that is open/free that gives the data about the few homozygous F508del whose sweat chloride dropped... Go to this website

http://onlinelibrary.wiley.com/doi/10.1002/ppul.v46.34s/issuetoc

Look for
'Poster Session Abstracts (pages 212 - 428)'
Click on
'PDF(3490K)' (Right below 'Poster Session Abstracts (pages 212 - 428)'

After it downloads to your computer, look at poster 206,
"VX-770 in Subjects with CF and Homozygous for the F508DEL-CFTR Mutation."

I apologize, I do not have the article from CHEST in front of me... somebody please correct me if I am wrong, but I do not remember the full article in CHEST talking about the few people that had a drop in sweat chloride.
 
R

Rummage

New member
There is a poster abstract that is open/free that gives the data about the few homozygous F508del whose sweat chloride dropped... Go to this website

http://onlinelibrary.wiley.com/doi/10.1002/ppul.v46.34s/issuetoc

Look for
'Poster Session Abstracts (pages 212 - 428)'
Click on
'PDF(3490K)' (Right below 'Poster Session Abstracts (pages 212 - 428)'

After it downloads to your computer, look at poster 206,
"VX-770 in Subjects with CF and Homozygous for the F508DEL-CFTR Mutation."

I apologize, I do not have the article from CHEST in front of me... somebody please correct me if I am wrong, but I do not remember the full article in CHEST talking about the few people that had a drop in sweat chloride.
 
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musclemania70

New member
ok, that helps too. However, I am looking for the phase III results of DDf508s who participated in the 770 trial. From what I understand, it was in the phase III where there was a small patient subgroup who saw an increase in fev1.
 
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musclemania70

New member
ok, that helps too. However, I am looking for the phase III results of DDf508s who participated in the 770 trial. From what I understand, it was in the phase III where there was a small patient subgroup who saw an increase in fev1.
 
S

saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> I found it! Nevermind!</end quote>

I am looking at Chest April 2012 and I can't find it. Can you post the link?
 
S

saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> I found it! Nevermind!</end quote>

I am looking at Chest April 2012 and I can't find it. Can you post the link?
 
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