5 ms·

Incredibly noble. I have a disastrous, incurable disease (PSC - primary sclerosing cholangitis) that may someday need a liver transplant (I'm not asking right n

Incredibly noble. I have a disastrous, incurable disease (PSC - primary sclerosing cholangitis) that may someday need a liver transplant (I'm not asking right now), which has lead me to thinking often about organ transplants. I'm also excited for genetic pig kidneys. https://nyulangone.org/news/studies-reveal-cell-cell-changes-caused-when-pig-hearts-kidneys-are-transplanted-humans https://nyulangone.org/news/studies-reveal-cell-cell-changes...

rented_mule 2y ago
My sister needed a liver transplant last year after many years of decreasing liver function. Given her age and other factors, they told her to expect to wait three to five years for a deceased donor, and that she likely would not live long enough for that. Of her six siblings, three of us passed the initial screenings to be donors. As we were about to start the more invasive tests, a deceased donor became available and my sister was the only match within the distance they were willing to transport the liver. Five hours later she went into surgery. She was only on the recipient list for two days. Everything went as well as could be expected and, in many ways, she's doing better than she has in decades. It's very complicated to think about the donor. All they told us was that it was an 18-year old who died in a traffic accident. We are all so grateful for the liver, and so sad for the donor's loved ones. Hopefully knowing that the donation saved another life and a part of their loved one lives on gives them some solace. I hope your case goes so well. Good luck!
- iamthemonster 2y ago
  If you don't mind me asking, what were you and your siblings told regarding the risk level for the donor? I tried googling this and I didn't find anything particularly definitive as the sample sizes were reasonably small
  - rented_mule 2y ago
    They said the primary risk was the same infection risk present in any major surgery, but they didn't put numbers on that. It's a large incision, so they said to expect significant scaring. They take half of the donor's liver. They said it will be back to ~80% of its original size in ~6 weeks and full size in about a year. They said to expect a week in the hospital, with significant pain, and a month of recovery nearby - we would have been travelling to have it done near my sister, and they wanted to be able to treat us if there were complications. The hospital covered all medical costs for donors, outside of medical insurance. It sounded like there was very little risk to liver function in the donor after recovery. They said that ongoing liver function should, in fact, be above average, as they wouldn't take a donor unless they had reason to believe it was a completely healthy liver to begin with. In our case, multiple advocates were assigned to each of the potential donors. Those advocates had no contact with my sister, and didn't even know her name or our relationship to her. It was their job to ensure minimal risk to us and to try to make it 100% okay for us to back out of donating. If we backed out or were ruled out medically, or for any other reason, their communication to my sister's team would have been the same, "they are no longer a candidate." Of course, we would be free to communicate whatever we wanted. Overall, they were very picky about selecting living donors. This was a large research hospital that is well known for liver transplants. They said they'd never had a liver donor death (as you said, not a huge sample size) and that it would be highly costly to the program, in terms or ability to attract donors, if they did have one. They said that the living donor should expect to go through a nutrition program run by their doctors to get to their ideal weight, with significant physical training involved. A therapist would also be assigned to the donor. Everything seemed geared to reducing risks to the donor.
    - iamthemonster 2y ago
      Thank you so much for your detailed reply, I really appreciate it!
slaw3 2y ago
I also have PSC. Pretty scary how little is known about how to treat it. The facebook group for it seems like a good resource. Ive been on oral vancomycin for about two years and it has normalized my enzymes, at least
- ampdepolymerase 2y ago
  You should try to convince the others on the Facebook groups to undergo full genome DNA sequencing (around $1K USD, usually not covered by insurance, I would pay for the other patients if I were you, their data is more than worth it given it’s your life on the line) and submit the data to patient networks and orphan drug groups. There are lots of bioinformatics methods now (thanks to mostly advances in ML among other things) that can derive insights into the problem, without any physical assays or laboratory tests.
  - adamredwoods 2y ago
    DNA sequencing has been done, we know the HLA[1] is a part of the disease. I don't think any more DNA will help find a cure or even a way to help. PSC is a complex "black box" disease. A person can have the DNA but not have the disease. BUT... bile and fecal samples still seem to be of high value for data collection. I've spoken with the doctors at PSC Partners to start advocating this collection, but they've noted that they don't have a way to process and store. Yet other clinicians have been doing this. I want to look into this more to help bring it all together (data is fun!), but haven't had to the time (single parent). [1] https://en.wikipedia.org/wiki/Human_leukocyte_antigen https://en.wikipedia.org/wiki/Human_leukocyte_antigen
Teever 2y ago
You may be interested in this: https://x-therma.com/news/x-therma-achieves-worlds-first-subzero-organ-transports-multiple-48-hour-transatlantic-journeys-support-first-steps-toward-tackling-organ-waitlist/ https://x-therma.com/news/x-therma-achieves-worlds-first-sub... I'm a layman so I don't know if this is genuine, but if it is it seems to be a world changing thing for people in your situation.
- adamredwoods 2y ago
  Nice! There was another technique of preserving the cadaver with oxygenation. https://www.npr.org/2024/07/08/nx-s1-5005407/transplant-surgeons-are-using-a-new-controversial-procedure-to-retrieve-organs https://www.npr.org/2024/07/08/nx-s1-5005407/transplant-surg...
CannonSlugs 2y ago
As a long time lurker here I created an account just to reply to this. I'm in the same boat! Diagnosed five years ago. It has taught me one thing about how hard finding causations is. While my liver is not fully healthy, ever since diagnosis it's been unchanged (according to scans) and my liver values have been good (for five years!). The reason? Chance, as far as I can tell. I am on the most conservative bog-standard treatment available (only UDCA), and it has been working really well. Reading online there are a lot of people doing antibiotics etc. I'm not being negative about these attempts (I'm sure it has some efficiency, studies seem promising), my point is just that if I took _anything_, I would contribute my mild progression to it and praise it like a panacea. Causation seems almost impossible to find with n=1. To me pure chance seems to dictate a lot. I am expecting a day when it all goes downhill though. With that said there does seems to be people who can go all their life without needing a transplant (the doctors seem more confident of this than the statistics, which is another strange oddity, but the statistics have always seemed overly harsh to me. I wonder if late diagnoses contribute to this).

rented_mule 2y ago

My sister needed a liver transplant last year after many years of decreasing liver function. Given her age and other factors, they told her to expect to wait three to five years for a deceased donor, and that she likely would not live long enough for that. Of her six siblings, three of us passed the initial screenings to be donors. As we were about to start the more invasive tests, a deceased donor became available and my sister was the only match within the distance they were willing to transport the liver. Five hours later she went into surgery. She was only on the recipient list for two days. Everything went as well as could be expected and, in many ways, she's doing better than she has in decades. It's very complicated to think about the donor. All they told us was that it was an 18-year old who died in a traffic accident. We are all so grateful for the liver, and so sad for the donor's loved ones. Hopefully knowing that the donation saved another life and a part of their loved one lives on gives them some solace. I hope your case goes so well. Good luck!

iamthemonster 2y ago
If you don't mind me asking, what were you and your siblings told regarding the risk level for the donor? I tried googling this and I didn't find anything particularly definitive as the sample sizes were reasonably small
- rented_mule 2y ago
  They said the primary risk was the same infection risk present in any major surgery, but they didn't put numbers on that. It's a large incision, so they said to expect significant scaring. They take half of the donor's liver. They said it will be back to ~80% of its original size in ~6 weeks and full size in about a year. They said to expect a week in the hospital, with significant pain, and a month of recovery nearby - we would have been travelling to have it done near my sister, and they wanted to be able to treat us if there were complications. The hospital covered all medical costs for donors, outside of medical insurance. It sounded like there was very little risk to liver function in the donor after recovery. They said that ongoing liver function should, in fact, be above average, as they wouldn't take a donor unless they had reason to believe it was a completely healthy liver to begin with. In our case, multiple advocates were assigned to each of the potential donors. Those advocates had no contact with my sister, and didn't even know her name or our relationship to her. It was their job to ensure minimal risk to us and to try to make it 100% okay for us to back out of donating. If we backed out or were ruled out medically, or for any other reason, their communication to my sister's team would have been the same, "they are no longer a candidate." Of course, we would be free to communicate whatever we wanted. Overall, they were very picky about selecting living donors. This was a large research hospital that is well known for liver transplants. They said they'd never had a liver donor death (as you said, not a huge sample size) and that it would be highly costly to the program, in terms or ability to attract donors, if they did have one. They said that the living donor should expect to go through a nutrition program run by their doctors to get to their ideal weight, with significant physical training involved. A therapist would also be assigned to the donor. Everything seemed geared to reducing risks to the donor.
  - iamthemonster 2y ago
    Thank you so much for your detailed reply, I really appreciate it!

slaw3 2y ago

I also have PSC. Pretty scary how little is known about how to treat it. The facebook group for it seems like a good resource. Ive been on oral vancomycin for about two years and it has normalized my enzymes, at least

ampdepolymerase 2y ago
You should try to convince the others on the Facebook groups to undergo full genome DNA sequencing (around $1K USD, usually not covered by insurance, I would pay for the other patients if I were you, their data is more than worth it given it’s your life on the line) and submit the data to patient networks and orphan drug groups. There are lots of bioinformatics methods now (thanks to mostly advances in ML among other things) that can derive insights into the problem, without any physical assays or laboratory tests.
- adamredwoods 2y ago
  DNA sequencing has been done, we know the HLA[1] is a part of the disease. I don't think any more DNA will help find a cure or even a way to help. PSC is a complex "black box" disease. A person can have the DNA but not have the disease. BUT... bile and fecal samples still seem to be of high value for data collection. I've spoken with the doctors at PSC Partners to start advocating this collection, but they've noted that they don't have a way to process and store. Yet other clinicians have been doing this. I want to look into this more to help bring it all together (data is fun!), but haven't had to the time (single parent). [1] https://en.wikipedia.org/wiki/Human_leukocyte_antigen https://en.wikipedia.org/wiki/Human_leukocyte_antigen

Teever 2y ago

You may be interested in this: https://x-therma.com/news/x-therma-achieves-worlds-first-subzero-organ-transports-multiple-48-hour-transatlantic-journeys-support-first-steps-toward-tackling-organ-waitlist/ https://x-therma.com/news/x-therma-achieves-worlds-first-sub... I'm a layman so I don't know if this is genuine, but if it is it seems to be a world changing thing for people in your situation.

adamredwoods 2y ago
Nice! There was another technique of preserving the cadaver with oxygenation. https://www.npr.org/2024/07/08/nx-s1-5005407/transplant-surgeons-are-using-a-new-controversial-procedure-to-retrieve-organs https://www.npr.org/2024/07/08/nx-s1-5005407/transplant-surg...

CannonSlugs 2y ago

As a long time lurker here I created an account just to reply to this. I'm in the same boat! Diagnosed five years ago. It has taught me one thing about how hard finding causations is. While my liver is not fully healthy, ever since diagnosis it's been unchanged (according to scans) and my liver values have been good (for five years!). The reason? Chance, as far as I can tell. I am on the most conservative bog-standard treatment available (only UDCA), and it has been working really well. Reading online there are a lot of people doing antibiotics etc. I'm not being negative about these attempts (I'm sure it has some efficiency, studies seem promising), my point is just that if I took _anything_, I would contribute my mild progression to it and praise it like a panacea. Causation seems almost impossible to find with n=1. To me pure chance seems to dictate a lot. I am expecting a day when it all goes downhill though. With that said there does seems to be people who can go all their life without needing a transplant (the doctors seem more confident of this than the statistics, which is another strange oddity, but the statistics have always seemed overly harsh to me. I wonder if late diagnoses contribute to this).