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I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory i

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished. It can't undo a life of damage to things like my pancreas, vas deferens, etc, and I still take medicine to digest my food, but overall, it's as close to "normal" as I could hope for at this point. The only real downside: weight gain. With CF the pancreas is blocked, so you lack in digestive enzymes, and it's a struggle to maintain a healthy enough weight to battle respiratory infections. After Trikafta, I gained some 30 pounds, and have a big belly on my small frame, and went up some 6 inches in the waist. To add insult to injury, it happened during the spring and summer of 2020, when buying clothes was a challenge due to the pandemic. Speaking of the pandemic, the timing of Trikafta was amazing: it kept the normally full "CF floors" of hospitals empty, opening up those beds for those with COVID and keeping CF patients less exposed. One side effect I should mention: many report extreme anxiety. However, I was starting an anxiety medication for the first time (something I should have done 20 years ago, but alas ...) and so those effects were muted or hidden to me.

az226 3y ago
What medicine do you take to digest your food? Can you tolerate onions, garlic, and wheat?
- bdcravens 3y ago
  Digestive enzymes, extracted from pigs. CF has no bearing on what I can tolerate, though the fattier the food, the more difficult it is to digest. In a situation where I had no access to my medication, I'd focus on eating simple carbs, like fruit.
DoreenMichele 3y ago
Well, my understanding is you are simply born without a vas deferens (for most men with CF). But some of the other stuff may heal at least partially.
- Scoundreller 3y ago
  ok, now you got me wondering... could a pregnant mother take this drug if they're at high risk of their fetus having CF (or confirmed via pre-testing) to prevent this? (Or possibly will continue taking it if they have CF as we can expect more CF patients to have children)
  - trollian 3y ago
    No.
  - DoreenMichele 3y ago
    It's a homozygous recessive disorder. If two carriers reproduce, there is a 25 percent chance the child has CF. If a woman with CF has children with a carrier, there is a 50 percent chance the child has CF and a 50 percent chance the child is a carrier. Ninety-five percent of men with CF lack a vas deferens, leaving them effectively sterile. The medical community is understandably reluctant to assist two people with CF in reproducing together as their child is guaranteed to have CF. If you are at risk of having a child with CF and know it, a best practice is to try to not conceive a child with CF. I doubt taking a drug during pregnancy would prevent a male child from being born without a vas deferens and see no reason to care. Just try to not have a baby with CF. It's called a Dread Disease for a reason.
  - _ihaque 3y ago
    Surprisingly, sort of. It won't alter the underlying genetic condition, but there has been at least one case report that taking these CFTR modulators during pregnancy can improve the condition of the fetus/newborn: https://www.sciencedirect.com/science/article/pii/S1569199321001004?via%3Dihub https://www.sciencedirect.com/science/article/pii/S156919932... https://med.stanford.edu/news/all-news/2023/07/cystic-fibrosis-pregnancy.html https://med.stanford.edu/news/all-news/2023/07/cystic-fibros...
    - DoreenMichele 3y ago
      Both of these babies are female. I am not at all surprised that the drug helped in the way described in the articles, but it doesn't actually indicate whether or not it could impact the vas deferens since that's a defect in the male reproductive tract. According to the CF Foundation, the cause of the lack of the vas deferens is not conclusively known. https://www.cff.org/managing-cf/fertility-men-cf https://www.cff.org/managing-cf/fertility-men-cf
trollian 3y ago
My 8yr old daughter has been on Kalydeco (one of the active compounds in Trikafta) since it was approved and it means she'll live a fairly similar life to her twin sister, health wise. We only have to do airway clearance treatment when she's sick. It's crazy for me to think what a different life shed have had even being born a decade before.
car 3y ago
Thank you for sharing this with us, I'm so glad for you! I can hardly express how amazing this development is to me! I had not heard about it before today.
matthewtse 3y ago
It's utterly amazing to hear of another group of sufferers who are the beneficiaries of modern miracle medicine. Personally, I suffered from an extreme case of eczema my whole life, which left me effectively a disabled person. Although eczema doesn't have a significant effect on life-expectancy, there wasn't all that much to live for, and I had basically given up on being able to do normal things like exercise or travel. So it was very surprising to "hear the good news" that Dupixent(https://en.wikipedia.org/wiki/Dupilumab https://en.wikipedia.org/wiki/Dupilumab) was being released in 2017 (also fast tracked as a breakthrough therapy), I got on it, and it's made me 98% a normal person. Here's to continued funding for new miracle medicines to help more groups of sufferers.
- spondylosaurus 3y ago
  Dupixent rocks! If you ever get sick of infections, it sounds like JAK inhibitors have opened the door to daily-pill eczema treatments too.
  - matthewtse 3y ago
    > If you ever get sick of infections I assume you meant "injections" :) For the value that Dupixent provides for me (effectively infinite), I'd do just about anything. So the injections don't bother me at all. Though injections do make international travel a bit more complicated. Daily-pill eczema treatments would definitely be more convenient in that respect!
    - spondylosaurus 3y ago
      Ha! Autocorrect got me, I did mean to say injections ^_^;; Although tbh I have had several nasty infections from then-untreated eczema...
    - sn9 3y ago
      I was actually on Dupixent for a while and I was one of the non-responders, so my derm put me on a JAK inhibitor daily pill. The convenience is offset by higher risk of side effects, though luckily I seem to have avoided those. The pills really seem to be the solution for me. My eczema had gotten pretty bad in recent years, and much of what you said resonates with me, though I don't think I ever got to the point of being effectively disabled. My skin is like 90% smooth now with some last stubborn patches, but I'm hoping with time they and the residual scarring will start to disappear.
jxf 3y ago
I don't have CF, but I am inspired by the pace of scientific advancement when I hear stories like this one. Thank you for sharing, and I hope every one of your years brings further boosts to your health.
dclowd9901 3y ago
My brother 12 years ago could have been you, as I read your comment. A double lung transplant and myriad complications later, and he passed on from this world. Reading your experience makes me so happy that people with CF are finally seeing a light of hope in their experiences. You do all the walks, donate the money, remind people this disease exists and it’s so destructive but it always kind of felt like science wasn’t throwing a lot of energy into CF, so I’m so glad to see this development. I’m so happy for you. I wish my brother could have had that chance, but I imagine, given his incredibly generous nature, he’d be just as happy for all the CF survivors today who now have a fighting chance.
- bdcravens 3y ago
  The CFF has actually done a good job of taking the lead. They approach it in a very startup-like manner. Trikafta is one of the results of that effort.
AnthonBerg 3y ago
I am very glad to read this. I don’t know very much about CF so I took the opportunity to read a little about it. A hunch struck me that CF seemed like a disease where N-acetylcysteine might be useful (NAC). Have had good and unexpected results with NAC in relation to Type 1 diabetes; not widely used in that context afaik but recent literature describes mechanisms and benefits. NAC is quite strange. Does a lot of things and works for a lot of stuff. Enough to seem a bit implausibly effective. It’s a mucolytic antioxidant which also replenishes certain important biological precursors and also has an effect on histamine and mast cells somehow, not just through being mucolytic (“slime-dissolving”). And more iirc. Just… does a lot. There’s literature on positive effects in congestion, anxiety, diabetes, placental health, brain damage from methamphetamine poisoning in rats (!), COVID illness and recovery, and on and on. As I said it seems kinda implausible how widely NAC seems to help. I’ve seen it have very clear and obvious benefits. T1 diabetes isn’t really something you can delude yourself about; no bargaining with it. As I read about CF I thought of NAC. I didn’t seem mention of it on the Wikipedia page for CF, but a Google Scholar search turned up some recent papers. ‘N-acetylcysteine (NAC) and Its Role in Clinical Practice Management of Cystic Fibrosis (CF): A Review’ —Guerini & co. ‘22 https://www.mdpi.com/1424-8247/15/2/217 https://www.mdpi.com/1424-8247/15/2/217 Wanted to share and ask if you had any experience with or thoughts on NAC?
- DoreenMichele 3y ago
  It's been an alternative remedy in the CF community for a while. People with CF hoard glutathione within the cell and have too little on the surface, so some people with CF use inhaled NAC to address the lack of surface glutathione in the lungs.
  - AnthonBerg 3y ago
    Thank you!, this is interesting and the insight is useful. I have to say that the distinction between alternative remedies and… idk, mainstream medicine?, this distinction feels spuriouser and spuriouser.
- jamesblonde 3y ago
  In Sweden we treat with NAC and bisolvon, amongst others, so it is not alternative. We have the highest life expectancy for CF in the world. But remember, correlation is not always causation.
jamesblonde 3y ago
I have 2 boys with CF, 12 and 14. They got ivacaftor from 4 and 5 yrs old, respectively. Pancreatic function returned for both, but has decreased over the last few years, FYI. just a data point. Don't stop the daily treatments either. It's not a full cure. The boy with a 32 mmol sweat is healthier than the boy with a 44 mmol reading.
- bdcravens 3y ago
  For sure. I do still take daily Pulmozyme, though the we have taken out the inhaled antibiotics. For reference, I'm 46.
  - jamesblonde 3y ago
    We also do inhaled natrium chloride. Congrats on taking care of yourself well enough to benefit from tricafta.

az226 3y ago

What medicine do you take to digest your food? Can you tolerate onions, garlic, and wheat?

bdcravens 3y ago
Digestive enzymes, extracted from pigs. CF has no bearing on what I can tolerate, though the fattier the food, the more difficult it is to digest. In a situation where I had no access to my medication, I'd focus on eating simple carbs, like fruit.

DoreenMichele 3y ago

Well, my understanding is you are simply born without a vas deferens (for most men with CF). But some of the other stuff may heal at least partially.

Scoundreller 3y ago
ok, now you got me wondering... could a pregnant mother take this drug if they're at high risk of their fetus having CF (or confirmed via pre-testing) to prevent this? (Or possibly will continue taking it if they have CF as we can expect more CF patients to have children)
- trollian 3y ago
  No.
- DoreenMichele 3y ago
  It's a homozygous recessive disorder. If two carriers reproduce, there is a 25 percent chance the child has CF. If a woman with CF has children with a carrier, there is a 50 percent chance the child has CF and a 50 percent chance the child is a carrier. Ninety-five percent of men with CF lack a vas deferens, leaving them effectively sterile. The medical community is understandably reluctant to assist two people with CF in reproducing together as their child is guaranteed to have CF. If you are at risk of having a child with CF and know it, a best practice is to try to not conceive a child with CF. I doubt taking a drug during pregnancy would prevent a male child from being born without a vas deferens and see no reason to care. Just try to not have a baby with CF. It's called a Dread Disease for a reason.
- _ihaque 3y ago
  Surprisingly, sort of. It won't alter the underlying genetic condition, but there has been at least one case report that taking these CFTR modulators during pregnancy can improve the condition of the fetus/newborn: https://www.sciencedirect.com/science/article/pii/S1569199321001004?via%3Dihub https://www.sciencedirect.com/science/article/pii/S156919932... https://med.stanford.edu/news/all-news/2023/07/cystic-fibrosis-pregnancy.html https://med.stanford.edu/news/all-news/2023/07/cystic-fibros...
  - DoreenMichele 3y ago
    Both of these babies are female. I am not at all surprised that the drug helped in the way described in the articles, but it doesn't actually indicate whether or not it could impact the vas deferens since that's a defect in the male reproductive tract. According to the CF Foundation, the cause of the lack of the vas deferens is not conclusively known. https://www.cff.org/managing-cf/fertility-men-cf https://www.cff.org/managing-cf/fertility-men-cf

trollian 3y ago

My 8yr old daughter has been on Kalydeco (one of the active compounds in Trikafta) since it was approved and it means she'll live a fairly similar life to her twin sister, health wise. We only have to do airway clearance treatment when she's sick. It's crazy for me to think what a different life shed have had even being born a decade before.

car 3y ago

Thank you for sharing this with us, I'm so glad for you! I can hardly express how amazing this development is to me! I had not heard about it before today.

matthewtse 3y ago

It's utterly amazing to hear of another group of sufferers who are the beneficiaries of modern miracle medicine. Personally, I suffered from an extreme case of eczema my whole life, which left me effectively a disabled person. Although eczema doesn't have a significant effect on life-expectancy, there wasn't all that much to live for, and I had basically given up on being able to do normal things like exercise or travel. So it was very surprising to "hear the good news" that Dupixent(https://en.wikipedia.org/wiki/Dupilumab https://en.wikipedia.org/wiki/Dupilumab) was being released in 2017 (also fast tracked as a breakthrough therapy), I got on it, and it's made me 98% a normal person. Here's to continued funding for new miracle medicines to help more groups of sufferers.

spondylosaurus 3y ago
Dupixent rocks! If you ever get sick of infections, it sounds like JAK inhibitors have opened the door to daily-pill eczema treatments too.
- matthewtse 3y ago
  > If you ever get sick of infections I assume you meant "injections" :) For the value that Dupixent provides for me (effectively infinite), I'd do just about anything. So the injections don't bother me at all. Though injections do make international travel a bit more complicated. Daily-pill eczema treatments would definitely be more convenient in that respect!
  - spondylosaurus 3y ago
    Ha! Autocorrect got me, I did mean to say injections ^_^;; Although tbh I have had several nasty infections from then-untreated eczema...
  - sn9 3y ago
    I was actually on Dupixent for a while and I was one of the non-responders, so my derm put me on a JAK inhibitor daily pill. The convenience is offset by higher risk of side effects, though luckily I seem to have avoided those. The pills really seem to be the solution for me. My eczema had gotten pretty bad in recent years, and much of what you said resonates with me, though I don't think I ever got to the point of being effectively disabled. My skin is like 90% smooth now with some last stubborn patches, but I'm hoping with time they and the residual scarring will start to disappear.

jxf 3y ago

I don't have CF, but I am inspired by the pace of scientific advancement when I hear stories like this one. Thank you for sharing, and I hope every one of your years brings further boosts to your health.

dclowd9901 3y ago

My brother 12 years ago could have been you, as I read your comment. A double lung transplant and myriad complications later, and he passed on from this world. Reading your experience makes me so happy that people with CF are finally seeing a light of hope in their experiences. You do all the walks, donate the money, remind people this disease exists and it’s so destructive but it always kind of felt like science wasn’t throwing a lot of energy into CF, so I’m so glad to see this development. I’m so happy for you. I wish my brother could have had that chance, but I imagine, given his incredibly generous nature, he’d be just as happy for all the CF survivors today who now have a fighting chance.

bdcravens 3y ago
The CFF has actually done a good job of taking the lead. They approach it in a very startup-like manner. Trikafta is one of the results of that effort.

AnthonBerg 3y ago

I am very glad to read this. I don’t know very much about CF so I took the opportunity to read a little about it. A hunch struck me that CF seemed like a disease where N-acetylcysteine might be useful (NAC). Have had good and unexpected results with NAC in relation to Type 1 diabetes; not widely used in that context afaik but recent literature describes mechanisms and benefits. NAC is quite strange. Does a lot of things and works for a lot of stuff. Enough to seem a bit implausibly effective. It’s a mucolytic antioxidant which also replenishes certain important biological precursors and also has an effect on histamine and mast cells somehow, not just through being mucolytic (“slime-dissolving”). And more iirc. Just… does a lot. There’s literature on positive effects in congestion, anxiety, diabetes, placental health, brain damage from methamphetamine poisoning in rats (!), COVID illness and recovery, and on and on. As I said it seems kinda implausible how widely NAC seems to help. I’ve seen it have very clear and obvious benefits. T1 diabetes isn’t really something you can delude yourself about; no bargaining with it. As I read about CF I thought of NAC. I didn’t seem mention of it on the Wikipedia page for CF, but a Google Scholar search turned up some recent papers. ‘N-acetylcysteine (NAC) and Its Role in Clinical Practice Management of Cystic Fibrosis (CF): A Review’ —Guerini & co. ‘22 https://www.mdpi.com/1424-8247/15/2/217 https://www.mdpi.com/1424-8247/15/2/217 Wanted to share and ask if you had any experience with or thoughts on NAC?

DoreenMichele 3y ago
It's been an alternative remedy in the CF community for a while. People with CF hoard glutathione within the cell and have too little on the surface, so some people with CF use inhaled NAC to address the lack of surface glutathione in the lungs.
- AnthonBerg 3y ago
  Thank you!, this is interesting and the insight is useful. I have to say that the distinction between alternative remedies and… idk, mainstream medicine?, this distinction feels spuriouser and spuriouser.
jamesblonde 3y ago
In Sweden we treat with NAC and bisolvon, amongst others, so it is not alternative. We have the highest life expectancy for CF in the world. But remember, correlation is not always causation.

jamesblonde 3y ago

I have 2 boys with CF, 12 and 14. They got ivacaftor from 4 and 5 yrs old, respectively. Pancreatic function returned for both, but has decreased over the last few years, FYI. just a data point. Don't stop the daily treatments either. It's not a full cure. The boy with a 32 mmol sweat is healthier than the boy with a 44 mmol reading.

bdcravens 3y ago
For sure. I do still take daily Pulmozyme, though the we have taken out the inhaled antibiotics. For reference, I'm 46.
- jamesblonde 3y ago
  We also do inhaled natrium chloride. Congrats on taking care of yourself well enough to benefit from tricafta.