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lcdbot27
03-08-2013, 05:46 PM
Hello,
I haven't posted on here, but I've been registered for awhile. I'm 30 with CF, my FEV1 is around 45%, and my mutations are W1282X and D579G. I hadn't done much research into Kalydeco or VX-809 because I knew both weren't really covered under my somewhat rare mutations. However, I've been at home sick for the past few days, and decided to look into it.
From what I've seen posted on here and mostly from that Vertex study Powerpoint from 2011, Kalydeco does seem to have a big effect on D579G. This slide (http://imgur.com/Brn3DYc) shows a big jump with D579G.
Is it worth the effort to get my doctor to prescribe Kalydeco off-label? What sort of information should I present to facilitate? That slide looked very encouraging, but I don't want to get my hopes up too far.

Thanks!

Aboveallislove
03-08-2013, 06:12 PM
YES. That slide should be a good start..there are some papers that discuss as wwell that dr should know. i'd call and ask dr to script or make an appointment now if they won'r over phone. many are n off labvel all ready

GenH
03-26-2013, 05:38 AM
If I were you I would try to get it, in the lab Kalydeco gets your mutation to about 90% of normal CFTR function, which is the same as being a carrier. G551D improves to 50% of normal CFTR function and yours gets to 90%, that is strong evidence. D579G is also one of the mutations potentially involved in the residual function trial: http://www.clinicaltrials.gov/ct2/show/NCT01685801?term=ivacaftor&rank=12



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lcdbot27
03-31-2013, 11:01 PM
Thanks for the replies. 90% normal function would be amazing.
Since I posted initially, I had a talk with the nurse practicioner at my clinic, I showed her the images and she said she would consult with the doctor about getting it off-label. The research coordinator and the doctor also called about the study in Denver that you listed, but the study place never called back. I'm going to contact my clinic tomorrow to follow up on the off-label route, I very much hope my insurance covers it.

ccsalema
04-05-2013, 06:53 AM
Terrible that you are having to educate your medical team, isn't it? Advocate, advocate, advocate! Well done!

lcdbot27
04-06-2013, 04:45 PM
Yeah, finding this out through my own basic research was...interesting. The main issue is that I live in a huge city and my CF center serves a lot of patients. I don't blame the CF team for having their hands full with more pressing matters and higher risk patients versus cross-referencing every genetic mutation of every patient against a Vertex slideshow...I know D579G is extremely rare in the US and that mutation probably wouldn't get noticed unless the FDA approved Kalydeco for use with it. The clinic has been a bit slow on following up on my requests for the off-label prescription, which is a little frustrating, but the head doctor has been out on vacation. Still, three weeks without a definitive "Yes, we are going to write you an off-label prescription" is getting annoying.
I also reached out on my own about the Vertex/NJH Denver study and spoke to the lady there, she said they were trying for approval to add more people to the current study. I'd prefer the off-label route, as I wouldn't have to miss work and I could be on its consistently, but if it comes down to going to Denver, I will definitely make myself available.

GenH
04-17-2013, 10:04 AM
How is it going with your doctor lcdbot27?

lcdbot27
05-01-2013, 02:05 PM
^ My doctor is trying to get me into the study at NJH, so we are on a holding pattern until we know if I got in or not.

kyeev
05-02-2013, 08:24 AM
Man, you must be going nuts, knowing this drug is available and could do wonders for you (especially having an FEV1 of 45%, your lungs are not too badly damaged).
I've often found this, you go to clinic and say "why am I not on drug X or Y".
Doc: Oh yeah, we better put you on that.
I remember going into my doctor with a sheaf of zithromax publications to get put on it back in the day.

lcdbot27
06-28-2013, 06:39 PM
Just a quick update: I got into the study. I don't think I can say much on a public forum just yet, but I'm very excited. My goal is to keep notes and I will probably start a blog with vital info once the study is complete, since it will probably be relevant to other CF patients with residual function class mutations.

carmartini
05-08-2015, 07:19 AM
Hi, can i ask you if you had tried kalydeco ?
With which results?
I m italian and my daughter has d579g