Best option for a transplant?

[Kamal D Shah]

Hi all,

 

Here's a brief summary of my medical condition:

- Diagnosed with aHUS in July 1997 following vaccination

- Immediately put on hemodialysis

- Transplant in November 1998 - mother donated

- Recurrent HUS after 11 days

- Back on dialysis

- On PD for 6 years

- Currently on daily nocturnal home hemodialysis

- Hepatitis C Positive

- CFH/CFHR1 hybrid gene detected

I would like to go for a transplant. I had a few questions:

1. What is the best option for a transplant - wait for eculizumab availability? Or take a shot with aggressive Plasmapheresis?
2. Is a live related donor transplant ruled out in aHUS?
3. Is prophylactic plasmapheresis or prophylactic eculizumab generally preferred for a transplant?
4. If live related transplant is contraindicated, how can prophylactic therapy be administered if cadaveric transplant is being performed?
5. Does this mean only live unrelated is an option for aHUS transplants?

Thanks a ton!
Kamal

[Kamal D Shah]

Thanks Jessica! Did you take any treatment for the Hep C?

[Jessica Olivia Frysz]

Kamal: I am also Hep C positive, but I have the antibodies present in my blood, so my liver is fine. I would say in my opinion to go for the Eculizumab as well as a live donor. As I am told, a live donor is much more beneficial when using the Eculizumab. Plasmapheresis requires a dialysis access, so you would need to make sure you have an open access to use for that and it can be demanding on the body.  I am told that with my aHUS condition, that unrelated live donor is better, because in my particular situation, my parents as well as myself all have the factor H deficiency, so by donating an organ, that could make them sick as well as making me sick in return.

[Kamal D Shah]

Thanks so much all of you for your responses! I can't emphasize enough how much your responses have helped me put things in perspective.

[Dana M Simone]

Hi Kamal,

 My son, Jack, received a kidney transplant with Soliris (Eculizumab) just 3 1/2 weeks ago. He had a deceased donor and hours prior to surgery underwent plasma pherisis and then received his first infusion of Soliris. He had experienced prophalactic FFP early in his disease, just to put it into remission, and then not again until the transplant. Jack turns 18 tomorrow; he presented with aHUS at 13 and was on dialysis the entire time. We opted not to have genetic testing done, so we don't know where this is coming from. We have two older sons who are healthy. Although our first instinct was to have them tested, we were counseled not to for two reasons: (1) if they have a gene mutation, there is nothing they can do about it, and (2) potential jeopardy to future health insurance. Needless to say, this made living, related donation out of the question. Now, although we don't know Jack's donor, we were told that he/she was a perfect match. We will always be grateful for the generosity of the donor and his/her family, and Jack intends to write a letter to the family. As a side note, I coordinate the blood drives at the company I work for, and we had a drive last week. It was wonderful to be able to share with everyone the story of Jack's transplant and acknowledge that because of donors, he was able to receive the blood he required. Everybody loves a personal story - we had a record turnout and Mass General Hospital (MGH), where Jack had his transplant, collected much needed pints!

[Cheryl Biermann]

Hi Kamal,

 

It appears that FDA approval for Eculizumab is anticipated for this fall.  That said, anticipated is not fact.  Also, Nathan had five years of prophalactic FFP, he was in the hospital constantly.  Dialysis was a God send for him.  He has had far fewer life and death situations and has had a pretty typical American childhood except for missing school three days a week.  I'm not saying dialysis in PD or Hemo form in any way is normal, I'm just letting you know what our life was like using FFP to control his disease.  It maybe more effective for you since you didn't get it until you were older.

 

We are going with Eculizumab, we have compassionate use here in the states so whether or not is is approved we will be able to use it.  Nathan's donor is me, his mother.  So live donation is preferrable to cadaver per the statistics here in the states, there isn't a huge difference, but I would say a significant difference.  Living related donation may be a problem for you depending on whether your willing and able family carry the gene or not.  My husband and Nathan's siblings are excluded because they have a MCP mutation, which puts Nathan with at least two mutations.  The MCP my husband has isn't inidcated in the literature as causing the disease, but as a precaution they are excluded. The other option would be non-related living donation of a kidney.

 

I certainly hear the frustration in your voice and I definitely know the panic you must be feeling with the co-morbidity setting in, but the thought of relying on FFP scares the heck out of me, it was the best they could do for him at the time, but it just doesn't do a good job of controlling the disease.  I know you have more obstacles to overcome being in India, but watching what is happening with the current people we are watching here, who are members of this site makes me confident that waiting until all the I's are dotted and Ts are crossed to get Eculizumab, despite my impatience is definitely worth it for us.  Good luck and please keep us updated. 

[Phyllis Ann Talbot]

Hi Kamal - OK - here's just my understanding - there may be other answers but for what it's worth.  First - did they try plasma pheresis in your earlier t'plant that failed?  assuming no since it was a while back and not sure if they understood all the ramifications back then. 

1. I'd say first choice is t'plant with Eculizimab but not sure if you want to wait.  latest news I've seen is that Alexion is hoping/ expecting the FDA here in the states to make a decision late in 2011 - so hopefully if it's approved that might make it more readily accessible in India? But I'd also not bank on end of this year - seems to sometimes take a bit longer than expected (as does everything in the medical world!).

2. We personally didn't do a live related donor but I know that at least one other family is pursuing this.  We did a live non-related donor for Hyde.

3.  We did prophylactic eculizimab only - but it seems like several of the other t'plants here in the US have done prophylactic plasmapheresis followed by eculizimab - think you'd probably do both since you have hemo lines available - Hyde had been on PD so he didn't have a line that could be used for pheresis and since he's small - we didn't want to use a vein for a short term treatment and our dr's didn't feel it was necessary with the Eculizimab and his specific mutation.

 

4.  I think if you can find a live donor it's great - for reasons other than AHUS (ie longer life of the t'plant, etc) - but our dr's didn't seem to concerned if we had had to do cadaveric (and I know at least one of the t'plants with Eculizimab here in the US was done with a cadaveric donor).  It's nice if a living donor is an option because you can time the Eculizimab in advance but if we had not had that option - the plan was to do the first dose of Eculizimab while prepping for the surgery with cadaveric and then following the 'normal' dosing after.

 

5. As I said - we did a non-related living donor but I know at least one other family that is doing a related donor that didn't have the same mutation as the child.

 

Hope this helps a little - and again - just our experience and what our dr's had told us - there may be other opinions ......good luck!

Phyllis