Orkambi Updates

[Ratatosk]

Just figured it might be nice to hear updates on those who are on Orkambi.

DS age 13, started on Orkambi July 31, 2015. He's grown taller, gained weight. Culture results this June again showed normal flora and epithelial cells. Since he was 2-3 months old he always cultured something -- enterbactor choclae (sp), pseudomonas a and florens, H. Flu and in more recent years strains of steno maltophilia pretty much resistant to everything.

I wish I'd kept track of how much he weighed at his appointment in June. His CF doctor was amazed that he'd grown 3-4 inches and gained 8 pounds in about 5 months. This summer he's grown even more and catching up with me --- He was about 5' 2" and he has less than an inch before he reaches my height of 5' 6". He now weighs 123 pounds, he weighed 112 at the June appointment as well.

 

[kenna2]

I've been on Orkambi for 4 months now and am doing well with it. I've seen an improvement in my x-rays, pfts, sugars, weight, and other areas as well. I'm also trying to get used to sweating like a normal person. I feel like a newborn baby experiencing things for the first time. It only took 31 years...lol. I am however starting to cough up a little bit of blood every couple of days in the evening. Not enough to call it in though. I'm guessing this is a side effect of the med. Has anyone else experienced this? Did it subside on it's own?

 

[cmorgan]

I've been on it since March. I do recall some light streaking for the first few months. However, for me that was welcome replacement from the blood baths that would occasionally occur before it. If its just streaking and less than a teaspoon of blood, you should be fine. Monitor it for frequency and changes in volume/color (Bright red & Frothy = fresh bleed, etc.) and make an issue if it does change/advance. I used have cup size bleeds before it, so I'm still onboard.

 

[Ratatosk]

I licked my child's forehead the other night. From the time he was a baby, I'd kiss his forehead and my lips would almost burn from the salt. He's not salty at all. I asked my husband if he thought that was odd. His response was "yes, it's weird that you licked your child". I meant weird he's not so salty!

I have noticed looser stools again and I've increased enyzmes, but he's been trying new foods, eating much much more, so I'm almost thinking it's probably time to move up in dosage. He's been on creon 12000 forever. Will check with his doctor in a month or so.

 

[EMILIAsParents]

My Daughter (11) started taking Orkambi about a week ago. As of now, we haven't seen any benefits or side effects.
I wanted to ask, In your experience, how long did it take to start noticing changes and what did you noticed first...second...?
Are there any recommendations on what to do to make it more effective (other than eating fat with it) , any tips?
Thanks!

 

[Ratatosk]

When he first started taking it he had cold like symptoms -- sniffles, throat clearing/cough and he had very very loose stools. I recall thinking since most of his symptoms are sinus and digestive that the so called purge people talk about was mucus from his digestive tract because his stools weren't formed at all for a week or so, wasn't typical greasy cf poop either.

Benefits were gradual, almost non-discernible until we noticed while sleeping he was sleeping so quietly. It was sort of a lightbulb moment "Hey, do you hear that.... it's so quiet". Soft breaths -- no snoring or mouth breathing. When he got a cold or came in from the cold his nose would run. His nose never ever runs. When he was a baby we had to use the booger sucker and when he was 4 we started doing sinus rinses to get that extra thick mucus out.

As I mentioned before his culture reports have been clear -- we'll find out if that's still the case in a couple months and he has grown significantly. Always hovered at the 50th percentile for height and weight with a LOT of work pushing calories. Now he's closer to 75th in height and weight. Has a good appetite.

 

[jricci]

Vertex Presents Long-Term Data Demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) Show the Potential to Modify the Progression of CF

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=995880

 

[mom2two]

Boy, I'd sure like to see a study of people who had to stop Orkambi due to severe side-effects and how it negatively effected THEIR outcomes.
I never recovered from taking that drug. My Orkambi journey will end with a transplant, if I am lucky.

 

[desnot2]

I have the double Δ508 mutation. Furthermore, I'm in my 30s, and have lung function studies with a FEV1 above 90%.

However, my lung functions were on a consistent downward trend over the past few years(more so than in the past). Last summer(2015), I started Orkambi, and did not experience any of the side effects mentioned in this forum. A week after beginning treatment, I noticed my exacerbation's had reduced to almost nothing, where before I had a consistent cough every few minutes all day long.

I felt almost like a normal person would without the condition. However, in late December, it was found out that my liver enzymes started to skyrocket. AST, and ALT levels were at critical, and my head started to feel consistently very uncomfortable. The feeling could most closely be associated with light headedness.

I was taken off Orkambi until my liver enzymes were reduced to normal levels again. My CF center attempted reduced levels of Orkambi to see if I could tolerate it. Ultimately it still caused my liver enzymes to skyrocket, and had to be taken off indefinitely.

Liver enzyme levels were definitely mentioned as one of the side effects of the drug, I wonder how many people are affected by this particular side effect.

 

[kenna2]

Hey everyone. I had a quick question regarding this topic. I have been on Orkambi for about 10 months now and my experiences have been much different when it comes to PFTs. I'm being hospitalized about every 3-4 weeks because I keep getting "sick", but my lung functions are stable and improving each time I'm hospitalized or after every clinic appointment. My PFTs are the highest they've been now since 2012 (63%)! I am bringing up multiple plugs a day and while I'm happy I'm improving each time and seeing the increase; I'm tired of the constant hospitalizations. My doctor has said she's never seen anything like this. Is anyone else experiencing this?