Orkambi works even if FEV doesn't improve

[Aboveallislove]

I know I'm tracking carefully what people are saying about Orkambi and specifically the FEV improvement. The Vertex conference noted one of the presentations at the NACFC which showed that there was no correlation between the FEV improvement at a specific point and reduced excerbations. In other words, even if FEV isn't improving, Orkambi is still reducing excerbations.
Here's the poster presentation: http://www.vrtx.com/assets/pdfs/con...EV1_PulmonaryEx_LUM_IVA_NACFC15_Poster241.pdf

Something to keep in mind if you aren't seeing an FEV improvement and think it's a waste of time, money, etc.

 

[Ank]

Ya i agree ..orkambi is just for correcting disorder..lung can not improve much as they are already damaged.

 

[stephen]

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My feeling has been that too much emphasis is being placed on FEV-1 improvement. I’ve written to both Vertex and the FDA concerning this.

Even without any improvement in FEV-1, I would consider Kalydeco a real life changer. The small improvement in FEV-1 it has provided is almost insignificant in comparison to the other benefits I’ve experienced.

I do understand that measurable parameters are very important in evaluating the results of a drug study. FEV-1 is something easy to quantify. However, for many of us with lung damage, significant FEV-1 improvement may not be possible without actually repairing this damage. Kalydeco and Orkambi were not designed to do this.

Unfortunately it appears that these drugs don’t really address digestive issues either.

 

[Aboveallislove]

Re the digestive issue: I think some have had some positive improvement and also the weight gain would indicate it must be doing something GI wise. One thing I'm definitely going to do is a photo journey with DS's hands because it is so severe and I think it will be so amazing if I can capture something like the aquagenetic wrinkling improvement. It might not be with Orcambi, but at some point we'll be there. And that is so wonderful what you shared and shared with FDA/Vertex.

My feeling has been that too much emphasis is being placed on FEV-1 improvement. I’ve written to both Vertex and the FDA concerning this.

Even without any improvement in FEV-1, I would consider Kalydeco a real life changer. The small improvement in FEV-1 it has provided is almost insignificant in comparison to the other benefits I’ve experienced.

I do understand that measurable parameters are very important in evaluating the results of a drug study. FEV-1 is something easy to quantify. However, for many of us with lung damage, significant FEV-1 improvement may not be possible without actually repairing this damage. Kalydeco and Orkambi were not designed to do this.

Unfortunately it appears that these drugs don’t really address digestive issues either.

 

[stephen]

As for weight gain with Kalydeco and Orkambi:

I attribute my unwanted weight gain to the fact that I'm no longer constantly fighting chronic lung infections. I had been told that fighting infections, especially those in the lungs, burns quite a few calories.

 

[aslong]

Hello Above all- I was in the shower the other day and noticed that the "aquagenetic" wrinkling was worse! I am in my 4th week and my hands wrinkled up so fast (faster than usual). I have been so dehydrated lately too... I cannot get enough water and gatorade. Hopefully it all gets better soon!

 

[Gammaw]

As long - how interesting! What other changes have you noticed with Orkambi?

 

[Aboveallislove]

Amy: How absolutely bizzare. Thanks for sharing. I'm so baffled by that! Augh. But yes, I'd love to hear any other changes you noticed too.

 

[rubyroselee]

I have noticed a significant improvement in my sinuses since starting Orkambi 2 months ago. I no longer have the continuous thick drainage or sinus headaches and can breathe better through my nose. I have noticed no GI improvements. I have not had an exacerbation since starting, where I had two in the 2 months prior to starting the drug. Mucus is easier to expel as well. So for me, that is enough improvement to call it a success and completely worth it. I have my first PFTs in 2 weeks since starting Orkambi, and even if they haven't improved, I still want to stick with the drug.

 

[Aboveallislove]

rubyroselee: Thanks for sharing. The sinus improvement intrigues me because I know a mom whose dd uses Kalydeco off label with a class 1 + df508 and she has had VAST improvement in sinus, even though it isn't suppose to work. Makes me think the cftr disfunction for sinus is either improved easier or has a different mechanism of operation.

 

[Ratatosk]

DS (age 12) has gained a few pounds since he started the end of July and has grown several inches. Checked shortly after he started regarding "aquagenetic" wrinkling and didn't notice an improvement at the time. Will have to check again now that he's been on it for awhile. He wasn't complaining this summer after spending a significant amount of time in the lake and usually I notice the wrinkling hours after he gets out of the water. His sinuses seem better -- no mouth breathing, his nose will run from time to time -- something that never happens. Breathes quieter at night.

 

[Gammaw]

That is very interesting ruby. As a mom of a severe polyp sufferer, I have developed a pet theory that the sinuses are the main source of exacerbations for at least my little guy. Not a scientific trial, just Mom.

 

[saintoffeon]

Re the digestive issue: I think some have had some positive improvement and also the weight gain would indicate it must be doing something GI wise.

At the risk of sounding gross, my GI flipped a switch almost from the moment I started taking it.

I eat very healthy, and my "formation" was relatively inconsistent. Mostly softer than not, but occasionally agonizingly non-productive (impaction) and then super-super soft. BUT, the thing that binds it all together? Horribly greasy, at least 80% of the time.

After Orkambi, I am the most regular person I know, there's not a hint of a drop of "grease," pleasant color, better smell, well-formed, etc.

What's weird is, I always had problems with digestive enzymes. They never felt like they did anything different to me, they never produced anything different, I never gained weight, sometimes lost it, and had uncomfortable side effects. If it weren't for my genotyping it'd make me think I might not have CF because some CFers say if they didn't take enzymes, they'd die, instead of just poorly absorb like I do.

I wonder if I have a flukey mostly-good enzymatic situation, that still had normal-CF characteristics as far as the stool was concerned, but allowed me to somehow be brought "over the line" into normal stool.