Trikafta Q&A, Experiences, etc.

[Hail2Pitt]

Hi all,

I know that a lot of folks are currently taking Trikafta, or at least are taking one of the other modulators. For me, I haven’t been able to take a modulator before, and I have questions about what to expect. If folks could chime in and tell a little about their experiences, I’d greatly appreciate it!

Here are some of the questions that I have:

My doctor talked to me a bit about some side effects that patients have had when taking Trikafta. The two that stand out as reasons you may have to stop taking the drug are liver issues and severe, itchy rash that you can’t get rid of. The possibility of having a side effect and needing to stop the drug is admittedly tempering my excitement slightly, so I wanted to see what experiences folks have had. For Trikafta, what kinds of side effects, if any, have you had to deal with?

I’ve heard patients talk about a mucus “purge” that happens after you start the drug. It sounds like for a lot of folks, they clear a crazy amount of stuff for a number of days after starting, and then things calm down. What was this like for folks? How long did it last?

Once you’re on Trikafta, what does it mean for your airway clearance? I do a minimum of 2 sessions every day (sometimes 3) plus exercise, and if I miss even one treatment, I can really tell. Do you still need to do airway clearance like you used to? Or, are you able to do shorter and/or fewer sessions a day?

What have people’s experiences been with the infections in their lungs? I have an especially pesky and resistant case of M. Abscessus, and I’m wondering what might happen when I’m on Trikafta. While I’m trying to not enter this with any expectations, my doctors seem optimistic that at the very least, the bacterial burden will decrease. Have people seen this with their infections? Did you seem them become less of a factor, or even go away?

What other experiences can you share?

Thanks, everyone!

Jim

 

[kenna2]

I have taken Orkambi and was switched over to Symdeko once it became available. I was switched over because of the better results that Symdeko had, but did fine on Orkambi. I went through two purge periods starting both drugs, however the first one was the worst because it was the first time I had something new and working in my system. That purge period lasted about 3 months for me on Orkambi and about a month on Symdeko; but it's different for everyone. You'll be amazed at what you can get out! You're lungs will be sore from coughing up all the plugs but you'll feel so much better once it's out.

When it comes to the liver stuff on these drugs there has been some new development on figuring out how to take it. I was someone who's liver functions got better on these meds, then got worse. However, over the past couple of months doctors and researchers have found out that our livers are holding onto too much of the drug Ivacaftor. Which is one of the drugs in all three (Kalydeco, Symdeko, Trikafta) modulators. In Symdeko and Trikafta, there is a nightime dose which is essentially just a booster of the Ivacaftor. They found that if they have us stop taking the nightime dose, our liver functions have improved and we have remained stable with just taking the morning dose. This is probably what will continue happening for us with Trikafta. So good news there!

When it comes to medications and airway clearance, nothing has changed for me. Some people have been able to reduce what they take, but many are on their same routine. When it comes to airway clearance in general, it never hurts to do it as many times as you can, but ultimately that will be up to you. When it comes to infections, my lungs have been stable with few infections. I still culture the same bugs and resistance hasn't really changed. I feel so much better though and my lungs are so much clearer. I still am able to get up some crazy huge plugs a couple of times a week which is great! I feel my life and health has gained some stability and things are starting to become more predictable, lung health wise. I still have random stuff that happens but doesn't affect my lung function at all.

 

[Hail2Pitt]

Thanks so much for your comments, kenna2!

I imagine that my purge will be pretty wild. Every once in a while, I'll have a day where I suddenly have a ton of junk to cough out, and it lasts all day. I don't really know why - sometimes it's after a hard workout, other times it just happens. The stuff that comes out looks crazy, I cough it out all day, and then the next day it's done. I assume there's a lot of that stuff just stuck in there, and every once in a while some of it breaks loose. I can't wait to get it all out!

And what you are saying about the liver stuff makes me feel better. Overall, my liver numbers are good, but they went up the last two times I was on long-term antibiotics. Once it got really bad, and we had to stop treatment altogether. While that doesn't necessarily mean I'd have trouble on Trikafta, it concerns me. It's great to hear there's at least one option!

 

[kosdancer]

I was in the phase 2 trial for Trikafta and have taken both Orkambi and Symdeko. I had such a different experience starting Trikafta than the other two that I'll just focus on Trikafta.
I absolutely had a mucus purge. It started about four hours after the first dose, and lasted for the whole first day - then it stopped. Over the next few weeks my PFTs inched up and my coughing went to pretty much nothing. I was a person who did not have an amazing PFT response - and I think it's important to know that that might happen. BUT, I still saw the highest PFTs I had seen in years so I'm not complaining one bit.
As far as bacterial infection, that's something I'm quite curious about myself. For the phase 2 trial I only got a month of drug but I sure felt like I didn't need inhaled antibiotics during that time. I think I'm about to start treatment for MAC and I'm really hoping that Trikafta will help with that - at the time of the trial I just had my Pseudomonas. Based on the data from Kalydeco, nobody should expect to get rid of their bacteria, but I think we can expect to have a lower bacterial load. And we definitely can expect fewer exacerbations.
For airway clearance, I think everybody will be different. I do know that some people have backed off on their airway clearance. I do not plan to, but I only do twice a day normally anyway. I'm hoping that I'll be able to go from continuously cycling Cayston and colistin to doing one month on one, a month off everything, a month on the other, etc. I also hope that I could sometimes skip Pulmozyme, but I'm not willing to bet on anything else at this point.

 

[Hail2Pitt]

Thanks for the info! It's really helpful to hear your experience.

I've been dealing with M. Abscessus for about 13 years, and have never been able to get rid of it. And I've been smear positive for several years now, despite treatments. I just finished one "failed" round of treatment, but before we try another one, my doctors want to see what happens once I'm on Trikafta. I'm hoping the Abscessus will become less of a factor just by me being on the drug. And, who knows, maybe my next round of treatment, whenever that is, will be more effective because of the Trikafta.

 

[Hail2Pitt]

Hi kenna2 - I wanted to ask you some follow-up questions about your experience on Orkambi and Symdeko.

I got my Trikafta, and started taking it a week ago. It's hard for me to know if it's doing much yet, since I came down with a respiratory infection right before I started taking it. My wife got the infection before I did, and it's taken her nearly two weeks to get back to normal, so it's a rough one and i know I still have a ways to go.

What's striking and frankly kind of concerning is the amount of junk I'm clearing from my lungs (and blowing out my nose) each day. My doctors have me on Cipro and Tobi, but given the amount of crap coming out of me, we're thinking about IVs. But, I'm wondering if this isn't only because of the infection, and is at least partly due to the Trikafta. When you went through your purges after starting modulators, what was it like? What I'm experiencing is that about an hour after I wake up, things seem to loosen up and I start clearing stuff. And then it goes on literally all day. This has been happening all week. I've been home from work, and it's hard to imagine going back with all the coughing I'm doing.

Thoughts?

 

[kenna2]

So the amount you are getting up is probably from both the infection and from the Trikafta. I've been seeing lately that people who are taking it are bring up a ridiculous amount of old junk. Someone had to ask their doctor about the color of the stuff they were bring up because they've never seen that color before..lol. Each time I changed meds, I got more and more stuff out and I think that is because of how much more effective the med is. I brought things up that were black, bloody plugs, hunter green to light green, grey, and different shades of yellow and white. The whole mucus rainbow. I coughed for days/ weeks upon end getting all stuff out and I was in a healthy spot. With the jump your making from Orkambi to Trikafta, it doesn't surprise me that you're going to have way more mucus clearance. It's also not uncommon for us to be put on IV meds when we change meds because of everything we're coughing up and the toll it takes on us. Also remember, with all that movement, it's clearing out and opening up new areas of infection that haven't been hit before. So it's not unheard of. I personally would push for IV meds, because it might take a bit of the edge off and help with the transition a bit too. Also make sure you hydrate like crazy too...that will help as well. I hope you feel better soon!

 

[Hail2Pitt]

Thanks for your reply! It's helpful to hear what you and others experienced with the junk you were bringing up. This is actually my first modulator, so I didn't entirely know what to expect.

Though, for you (and others), how long did it take for you to feel better? I've read where some people say they felt better quickly, i.e., , within a few days, they had more energy, better lung function, etc. For me, granted I'm still dealing with an infection, but I feel worse right now. I don't know how my lung function is, but I definitely am more tired than I usually am. I also don't see my purge ending any time soon, as my nose remains a faucet and I still have a productive cough all day. I was kind of expecting that I'd start the drug and feel great within days.

 

[kenna2]

For me it took me a couple of days for me to start feeling the changes, but it took me a couple months to look back and realize "wow I feel better". I noticed my body wanted to cough, but I didn't actually need to cough and that was weird. I think I went though a period we're I just coughed for no reason, because I was so used to coughing all the time. Seeing how much I brought up and how old some of those pieces were and the size was shocking too. I still wonder where those huge pieces of plugs came from. I really was focused on that for all those month so it could have been sooner. My first admission where I was sick with something else that would normally bring down my lung function and it didn't was amazing. My lung function was actually stable the entire time and I couldn't believe it. That's when it really hit me and I knew it was actually working. The purge period for me slowed down slowly but it does end I promise. I'm excited for you and hopeful that you get some great relief soon!

 

[Jeana Lynn]

kenna2 with all the different junk you were coughing up, what was your starting fev1? In a way, I am a little terrified of all the coughing from the "purge" because with my coughing fits I get SOB and panic, even with oxygen.

 

[kenna2]

Before starting Orkambi I was at 46% and before Symdeko I was at 58%. I am now at 65% and still waiting for insurance to approval. I completely understand being terrified, but in my experience there were breaks of hours or days before another huge purge hit. But once you see what comes up, I think that excitement takes over and the hope that more and more comes out takes over. After a while too, the purge period becomes easy and plugs come up so much faster with less work. It's worth it, trust me.

 

[ChaosEst1979]

Thank you all for your questions and responses. I took the first two modulators briefly and had horrific side effects to both causing me to stop them within the first two weeks. I have been very afraid to try Trikafta honestly. However, hearing your stories here has made me really want to give it a valiant effort even if it does make me initially violently ill for a few weeks. I have two young daughters and I have enlisted help this time around so that if I must go into the hospital for awhile to get through the initial side effects, I can give this medicine a better and longer try. I am seriously afraid because it contains the same two medicines that made me so sick the first two times but I also know how good it has been for others and how good it can be for me in the long run. I currently baseline my PFT’s at anywhere from 75-90% so I am very blessed and healthy with this disease already, but I do obviously want to live longer and live my best life with CF. Prayers and well wishes to all of you!

 

[hammerpocket]

I thought I would add some of my experiences and address your questions about reducing therapies. I've been on Trikafta for about 18 months. (There were actually two candidate combinations in the study. I was on the one that was dropped until I switched a couple of months ago, but I haven't noticed any difference.) What has been described as the "purge" wasn't as dramatic for me as some people are describing, but I did bring stuff up. I also quit having hemoptysis, which had been a chronic issue for years (usually small amounts, but daily). When I get sick now, like a common cold, I can usually recover without ending up needing antibiotics. Also, I've noticed that my mouth is not as dry as it used to be and I have an easier time eating stuff like bread that would often ball up instead of breaking down in my mouth.

As of now, I still take all the meds and therapies I did before; however, missing a vest/aerosol therapy isn't as noticeable. If I'm out late at night, I don't worry about getting in the vast before bed. I've even gone on vacations of a few days without my vest (but still with nebulizers) and it's been fine. I can't take colistin or TOBI anymore, so my months off from Cayston used to be hard, but now I don't feel much different on those off months. I had to slightly reduce my enzyme dose (from 6 capsules with meals to 5) because of changes from Trikafta.

Btw, when I start coughing from a sickness now, I realize how little I normally cough now, and it's already kind of unbelievable to think of how bad things used to be.

 

[kenna2]

Chaos...I'm so glad to hear that you're pfts are amazing and that you're willing to give Trikafta a shot. Can I ask what symptoms you were experiencing with the other two modulators? Just curious because, I may have some insight to help calm your nerves.

 

[pink]

Trikafta

So I don't usually write in these forums but I wanted to share my experience with Trikafta. First I want to start with my experience with Orkambi. I took about 2.5 doses of this and was sick like the flu for 7 days. I was miserable, fever, cough in bed. I didn't continue the orkambi. I was really hesitant with Symdeko. But I had no side effects at all. It even took me quite awhile before I felt that it was even doing anything. But it was and I did see improvement. I started Trikafta and the first day I coughed a lot of stuff up for me but it was really easy coughing. Next morning I had a low grade fever. The coughing was much less but much more intense, I felt like i needed to cough stuff out and I couldn't like it was really deep. The fever got worse and the next day it got up to 101. This was Sunday. My son had had pneumonia recently and my daughter had a bad cough, I started worrying I had caught something from them. I called the pulmonary fellow on call and they told me to go to ER. ER was worried I did have pneumonia - which I didn't. So I was admitted (first time in 25 years - I'm 44). They put me on IVs, still was running fever, but the cough had pretty much stopped. After a few days they did a PFT. It was down 8 %. I was not surprised as my lungs felt very tight. I convinced my doctors it was the medicine not an exacerbation. They put me on predisone thinking maybe I was having asthma type attack from the med, which helped. I was sent home after almost a week in hospital and was feeling good. (I got lots of help coughing stuff out and rest!) The next day, 9 days on Trikafta, I broke out in the itchy rash. At this point I was told to stop the med, which I did and switched back to Symdeko. But at this point I felt great and was definitely feeling positive effects of the meds. My home fev1 monitor showed improvement and I just had so much energy. I am off the med now for a week, but I plan on trying it again. My rash is gone but still kinda itchy. I really think this med is going to have really positive effects for people. I know if you've had bad side effects it can be scary but I would really give it a try! (Also my PFt's are in the exact same range as you Chaos) Hoping my body will tolerate it the second time around. (Also I've had horrible sinuses all my life and those first few days on Trikafta I was miserable with sinus pain, but things seemed to clear out and I think my sinuses are pretty darn clear right now!)

 

[ChaosEst1979]

Kenna2.... I had pretty much everything you can think of... body aches, fever, severe diarrhea, severe vomiting, terrible itching all over, a horrible cough that just didnÂ’t quit and made me vomit more, extreme fatigue where I couldnÂ’t get out of bed (which always pairs great with diarrhea and vomiting), lots of trembling/shaking. I am sure there was more but I canÂ’t recall anything else right now. I felt miserable in every way.

 

[damndisease]

I’ve been on Trikafta for a week exactly. Orkambi and Symdeko did nothing for me (2 copies f508del ). I was at my wit’s end with the return of the phlegm daily and the Trikafta has been like an “off” switch. I had a 5 hour purge that was so damn emotional. Phlegm is almost non existent since then. Barely cough and always able to take deep breaths. I was losing the CF battle at a rapidly accelerating rate and was starting to think about the inevitability of transplant sooner than I’d hoped. I haven’t had a PFT since starting and I’m not expecting much since I can feel my lung damage. That’s okay, it’s just a number. Every body is so unique you are better off trying not to anticipate anything “normal”. It’s hard to do, but you’ll drive yourself crazy. I went through a range of emotions for the month after approval and before I got it and I couldn’t focus on life. No big whoop, but it was a waste of time in so many ways. On the other hand, enjoy the anticipation !!! Good luck!

 

[Mekikya]

My son just started taking Trikafta this week, we are on day 3. So far haven't seen much of a change in his cough or purging yet but we are lucky in that he's always been pretty healthy, 80% or higher on his PFTs. I'm most interested to see how this will effect his growth and digestion and blood sugar, because he's started to have raised blood sugar levels (due to puberty) and he's always been around the 25th percentile on the growth chart. I am hoping that we can at least decrease the length of treatments. Right now he does a lot compared to what I see most people do on here, thats kind of by my choice not by his doctor's. Hour and a half of vest twice a day, with 30 minutes of each medicine nebbed, alb, saline, pulmo with it. I am thinking if he shoots up alot in his PFTs next time at clinic, maybe I will let off him a bit. Maybe only an hour of vest each day. (Yes, I am that mom, but my hope was that if I could just keep him as healthy as possible, save damage to his lungs, then maybe something like Trikafta would come along since he's so young, 13)

 

[chempharm23]

My experience with Trikafta has been awesome. Before I started Trikafta two weeks ago, my pfts were in the low 20s and I was on O2 5 liters, 24/7. Even dressing in the morning, eating, going to the bathroom, etc. was extremely hard and tiring. I probably didn’t have much time left.

Enter Trikafta. About four hours after my first dosage, the purge began. For two days I was coughing up thick mucus every ten seconds. It actually was very tiring but it was worth it. On the third day, suddenly I realized I didn’t need oxygen anymore just sitting around. By the fifth day, I didn’t need it for normal activities. It is literally a miracle drug for me. I still use O2 for exercise and sleep, but that’s it! My life has been given back to me, thanks to the scientists and everyone else who made this miracle possible. I barely cough at all and I’m never short of breath anymore. The only side affects that have affected me is itching on my arms and a few other places. I already have high liver enzymes so I’m hoping it won’t raise them any higher. Fingers crossed! I pray daily that the ten percent of CFers who can’t benefit from Trikafta will eventually be helped by

 

[Maria55]

Hi. I was the same as you. Spitting up a lot the first day, than it stopped. But I can breath better and less winded.