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View Full Version : Off Label Kalydeco-My follow up Appt.



marcijo
04-27-2012, 01:19 AM
I had my 4 week follow up appt for Kalydeco. I am DF508 and R117H and am on it off-label. I asked my doctor to prescribe it, and insurance covered it.
I went in today to have repeat PFTs and a repeat sweat test done. My FEV1 was at 83% a month ago, today it was at 87%. I was hoping it would be higher-but the fact that it increased 4% I am happy with!
My last sweat test (before I started Kalydeco) was 81. Anything over 60 is considered CF, 40-60 is borderline, and under 40 is normal.
My sweat test today was 36. Which means my body is working normally! So Kalydeco does work on R117H! I am THRILLED!

marcijo
04-27-2012, 01:19 AM
I had my 4 week follow up appt for Kalydeco. I am DF508 and R117H and am on it off-label. I asked my doctor to prescribe it, and insurance covered it.
I went in today to have repeat PFTs and a repeat sweat test done. My FEV1 was at 83% a month ago, today it was at 87%. I was hoping it would be higher-but the fact that it increased 4% I am happy with!
My last sweat test (before I started Kalydeco) was 81. Anything over 60 is considered CF, 40-60 is borderline, and under 40 is normal.
My sweat test today was 36. Which means my body is working normally! So Kalydeco does work on R117H! I am THRILLED!

GenH
04-27-2012, 02:34 AM
Thats excellent Marci! 36 is a great number!

GenH
04-27-2012, 02:34 AM
Thats excellent Marci! 36 is a great number!

Incomudrox
04-27-2012, 08:33 AM
This is fantastic news keep at it! Are you exercising at all?

Incomudrox
04-27-2012, 08:33 AM
This is fantastic news keep at it! Are you exercising at all?

saveferris2009
04-27-2012, 09:28 AM
Congrats! That is so exciting!

saveferris2009
04-27-2012, 09:28 AM
Congrats! That is so exciting!

cindylou
04-27-2012, 10:33 AM
Marci, I am SO EXCITED!!! This is the best news I've heard all day, woohoo!

cindylou
04-27-2012, 10:33 AM
Marci, I am SO EXCITED!!! This is the best news I've heard all day, woohoo!

marcijo
04-27-2012, 02:00 PM
Incomudrox-I am an occasional exerciser...this past week I went on a walk/run for a total of 1.75 miles....only 9 minutes of it was running though.

I definitely want to get back into running though-I think it definitely helps my lungs!

marcijo
04-27-2012, 02:00 PM
Incomudrox-I am an occasional exerciser...this past week I went on a walk/run for a total of 1.75 miles....only 9 minutes of it was running though.

I definitely want to get back into running though-I think it definitely helps my lungs!

bigstar
04-27-2012, 03:40 PM
Marci Im so happy for you! I hope cases like yous open up the way for everyone! Congrats!

bigstar
04-27-2012, 03:40 PM
Marci Im so happy for you! I hope cases like yous open up the way for everyone! Congrats!

JENNYC
04-27-2012, 04:01 PM
Wow!! So happy for you!!! Thank you so much for sharing your experience with us!!! That is super fantastic!!

JENNYC
04-27-2012, 04:01 PM
Wow!! So happy for you!!! Thank you so much for sharing your experience with us!!! That is super fantastic!!

cfgf28
04-27-2012, 05:40 PM
Wow fantastic results! Bf goes to the dr on maonday, def think its worth inquiring about the drug!!

cfgf28
04-27-2012, 05:40 PM
Wow fantastic results! Bf goes to the dr on maonday, def think its worth inquiring about the drug!!

jmiller
04-27-2012, 06:19 PM
How incredible! Words cannot describe all that this must mean for you and Sam and Ella!

jmiller
04-27-2012, 06:19 PM
How incredible! Words cannot describe all that this must mean for you and Sam and Ella!

Gorf
04-27-2012, 06:33 PM
Congrats

Gorf
04-27-2012, 06:33 PM
Congrats

Gorf
04-27-2012, 06:36 PM
Just curious, may be to soon, I have heard of others getting more junk up like the lungs clearing out what it coudln't before, have you? How is your excersise tolerance level compared to before, are you able to notice a difference?

Gorf
04-27-2012, 06:36 PM
Just curious, may be to soon, I have heard of others getting more junk up like the lungs clearing out what it coudln't before, have you? How is your excersise tolerance level compared to before, are you able to notice a difference?

jessykt
04-27-2012, 11:41 PM
I want to know what Gorf asked....my husband said he's noticed a higher tolerance in me to walking more, but I didn't bring a lot of junk up AT ALL and wonder why as I'm hearing most people are bringing stuff up.

jessykt
04-27-2012, 11:41 PM
I want to know what Gorf asked....my husband said he's noticed a higher tolerance in me to walking more, but I didn't bring a lot of junk up AT ALL and wonder why as I'm hearing most people are bringing stuff up.

Dayna1
04-28-2012, 05:32 AM
Im soooo very happy with your test results!!! I have a 17 year old with CF and has only been hospitalized twice for IV Antibiotic Therapy, but Im glad to hear that there is hope for the future. Best of luck to you and please keep posting your results.

P.S. Where can I find out more information on Sweat Test results, and will my Dr. give my daughter tests? Thank you.

Dayna1
04-28-2012, 05:32 AM
Im soooo very happy with your test results!!! I have a 17 year old with CF and has only been hospitalized twice for IV Antibiotic Therapy, but Im glad to hear that there is hope for the future. Best of luck to you and please keep posting your results.

P.S. Where can I find out more information on Sweat Test results, and will my Dr. give my daughter tests? Thank you.

marcijo
05-01-2012, 01:32 AM
Thanks everyone!

I brought up gunk the first week I was on it.....but I was also finishing up antibiotics so I don't know if the gunk I coughed up was due to the antibiotics or the Kalydeco. I didn't really have the ton of gunk that some others talk about coming up....but maybe its because my lung function was in the 80's (FEV1). I have no idea. I rarely cough up anything now....I don't think I could give a sputum sample-I just don't think there is anything in my lungs.

As far as my exercise tolerance-I think it is much better-because I don't have to cough anymore when I am exercising! My lung function was 83% when I started Kalydeco, and went up to 87% after 3+ weeks on the meds. I am hoping to see it go up even more, but we'll see.

I also think I had a cold and didn't have to go on antibiotics! My kids had a sore throat/bad cough....I came down with a sore throat and just felt crummy-a bit of coughing as well. It is on its way out (I hope!) and I didn't have to go on antibioitics-which has NEVER happened for me.

I will keep you guys posted on any updates! I don't go back to my CF clinic until mid July.....

marcijo
05-01-2012, 01:32 AM
Thanks everyone!

I brought up gunk the first week I was on it.....but I was also finishing up antibiotics so I don't know if the gunk I coughed up was due to the antibiotics or the Kalydeco. I didn't really have the ton of gunk that some others talk about coming up....but maybe its because my lung function was in the 80's (FEV1). I have no idea. I rarely cough up anything now....I don't think I could give a sputum sample-I just don't think there is anything in my lungs.

As far as my exercise tolerance-I think it is much better-because I don't have to cough anymore when I am exercising! My lung function was 83% when I started Kalydeco, and went up to 87% after 3+ weeks on the meds. I am hoping to see it go up even more, but we'll see.

I also think I had a cold and didn't have to go on antibiotics! My kids had a sore throat/bad cough....I came down with a sore throat and just felt crummy-a bit of coughing as well. It is on its way out (I hope!) and I didn't have to go on antibioitics-which has NEVER happened for me.

I will keep you guys posted on any updates! I don't go back to my CF clinic until mid July.....

JoyousMom
05-01-2012, 08:31 PM
Marci,
I am new to the forum, and very intrigued by your experience with Kalydeco. My daughter, 16, Joy, just saw her CF Team last week and we talked with her Dr. about Kalydeco. Joy has Delta F508. Her doctor said that the medicine would NOT be available until approved by the FDA/or more research conducted etc., and we had about two years of waiting ahead of us.
How did you manage to get this pill?!!!! Is there any hope for the rest of us to get it sooner?

JoyousMom
05-01-2012, 08:31 PM
Marci,
I am new to the forum, and very intrigued by your experience with Kalydeco. My daughter, 16, Joy, just saw her CF Team last week and we talked with her Dr. about Kalydeco. Joy has Delta F508. Her doctor said that the medicine would NOT be available until approved by the FDA/or more research conducted etc., and we had about two years of waiting ahead of us.
How did you manage to get this pill?!!!! Is there any hope for the rest of us to get it sooner?

marcijo
05-02-2012, 12:51 AM
JoyousMom - My mutations are DF508 and R117H. In-vitro studies showed hope that Kalydeco would work with the R117H. I asked my doctor if he would write a prescription for me-he said he would. Insurance covered it - and here I am! I am taking it off-label.

Is your daughter DDF508 (are both her mutations DF508)?

marcijo
05-02-2012, 12:51 AM
JoyousMom - My mutations are DF508 and R117H. In-vitro studies showed hope that Kalydeco would work with the R117H. I asked my doctor if he would write a prescription for me-he said he would. Insurance covered it - and here I am! I am taking it off-label.

Is your daughter DDF508 (are both her mutations DF508)?

saveferris2009
05-02-2012, 11:18 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>JoyousMom</b></i> Marci, I am new to the forum, and very intrigued by your experience with Kalydeco. My daughter, 16, Joy, just saw her CF Team last week and we talked with her Dr. about Kalydeco. Joy has Delta F508. Her doctor said that the medicine would NOT be available until approved by the FDA/or more research conducted etc., and we had about two years of waiting ahead of us. How did you manage to get this pill?!!!! Is there any hope for the rest of us to get it sooner?</end quote>

I have DF508 and I have been on Kalydeco for nearly 2 months.

I've seen positive results as well <img src="i/expressions/face-icon-small-smile.gif" border="0">

saveferris2009
05-02-2012, 11:18 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>JoyousMom</b></i> Marci, I am new to the forum, and very intrigued by your experience with Kalydeco. My daughter, 16, Joy, just saw her CF Team last week and we talked with her Dr. about Kalydeco. Joy has Delta F508. Her doctor said that the medicine would NOT be available until approved by the FDA/or more research conducted etc., and we had about two years of waiting ahead of us. How did you manage to get this pill?!!!! Is there any hope for the rest of us to get it sooner?</end quote>

I have DF508 and I have been on Kalydeco for nearly 2 months.

I've seen positive results as well <img src="i/expressions/face-icon-small-smile.gif" border="0">

jdmd
05-02-2012, 07:28 PM
Holy Cow! That is amazing...the sweat chloride value is so exciting! So happy for you, and in advance for those of us waiting for the magic combination cocktail:-)

jdmd
05-02-2012, 07:28 PM
Holy Cow! That is amazing...the sweat chloride value is so exciting! So happy for you, and in advance for those of us waiting for the magic combination cocktail:-)

JoyousMom
05-02-2012, 09:25 PM
Yes, I believe she is double DF508.

JoyousMom
05-02-2012, 09:25 PM
Yes, I believe she is double DF508.

JoyousMom
05-02-2012, 09:29 PM
Ok, is this drug available to those who just happen to have doctors who are willing and insurance that is willing? Is anyone with DF508 taking this? I've been told by our CF doctor that it is NOT available for my daughter right now. Two years of waiting. But like most of you I would move heaven and earth (or at least take out a big loan!) to find a drug that would not only extend life but take away her symptoms.....
I live in Texas, near Austin---any suggestions? Also, she is on Medicaid/Medically Dependent Children's Program.

JoyousMom
05-02-2012, 09:29 PM
Ok, is this drug available to those who just happen to have doctors who are willing and insurance that is willing? Is anyone with DF508 taking this? I've been told by our CF doctor that it is NOT available for my daughter right now. Two years of waiting. But like most of you I would move heaven and earth (or at least take out a big loan!) to find a drug that would not only extend life but take away her symptoms.....
I live in Texas, near Austin---any suggestions? Also, she is on Medicaid/Medically Dependent Children's Program.

saveferris2009
05-02-2012, 10:33 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>JoyousMom</b></i> Ok, is this drug available to those who just happen to have doctors who are willing and insurance that is willing? Is anyone with DF508 taking this? </end quote>

I just told you three posts ago that I have DF508 and I'm on it.

saveferris2009
05-02-2012, 10:33 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>JoyousMom</b></i> Ok, is this drug available to those who just happen to have doctors who are willing and insurance that is willing? Is anyone with DF508 taking this? </end quote>

I just told you three posts ago that I have DF508 and I'm on it.

marcijo
05-02-2012, 10:33 PM
As far as I know-you need a doctor willing to write you a prescription and an insurance company that will pay for it. I started by calling my insurance company...they said it would be covered. Then I asked my doctor. Good luck!

marcijo
05-02-2012, 10:33 PM
As far as I know-you need a doctor willing to write you a prescription and an insurance company that will pay for it. I started by calling my insurance company...they said it would be covered. Then I asked my doctor. Good luck!

JoyousMom
05-03-2012, 02:03 PM
Is there anyone taking Kalydeco who has Df508 double gene?
If so, please tell me how you obtained it.
Thanks you!

JoyousMom
05-03-2012, 02:03 PM
Is there anyone taking Kalydeco who has Df508 double gene?
If so, please tell me how you obtained it.
Thanks you!