Dual Transplant - Kidney/Liver

[KaTrina Slaughter]

Learned that our transplant team is leaning towards doing a double transplant. I know I had read some things that many people were not certain of this transplant and where sure that the risk out weighed the benefits. Was just wondering if anyone knows of any successful double transplants or if there is any more literature that would support this??

[Stacey]

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[KaTrina Slaughter]

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[Linda Burke]

At the Oct. 2010 aHUS conference at the University of Iowa, information was presented on a variety of topics, including renal transplants. Although individual patients and their families must weigh their situations carefully with input from both their regular physicians and their transplant teams, currently doctors seem to be leaning toward the "Kidney Transplant with Soliris" as the most viable option rather than a "Kidney/Liver Dual Organ Transplant".
Two strikes are against the Soliris option - one is that many physicians still have not heard of Soliris, which is only FDA approved to stop hemolysis in adults with PNH, so they cannot recommend an option they have not heard of before (Alexion cannot contact doctors, advertise, etc until Soliris obtains FDA approval for aHUS patients.) The second strike is that while aHUS cases are beginning to start the renal transplant option with Soliris, the transplant eval and wait is time-consuming, so no aHUS patients (of which we are aware) have completed a renal transplant with planned Soliris support. ( Apparently, there has been evidence of renal tranplantation that involved Soliris as a successful 'rescue' when problems surfaced post-transplant.)
Two strikes exist against the dual kidney/liver transplant as well- one is that this operation is extremely complex even for physicans/surgeons who have already successfully completed kidney/liver transplants for other aHUS patients. The second strike is that you cannot survive the failure of the liver transplant - the patient always dies in less than 48 hours.
In all, it seems as if the choice for many boils down to this- the Soliris option is less risky and the patient will still live even if the kidney transplant fails (a 2nd kidney transplant would still be a possible future option). The dual kidney/liver transplant used to be the only available option until a few months again because it takes care of the problem of aHUS recurring, yet not all aHUS patients survive. The Soliris option is currently the choice of two adult and several pediatric aHUS patients - though they are all at different stages on their transplant journey. I'm aware of only two aHUS patients/families (KaTrina and Stacy) currently looking at the kidney/liver transplant. Since many of you have sent me your transplant plans by "Send a Message" rather than our public comment walls or Forums, I'll respect your privacy and defer to you as to whether you'd like to announce your transplant news and views to the aHUS online community.

[Bill Biermann]

As we all know, having kidneys in place and receiving plasma infusions or plasmapheresis leaves our children/selves in danger of relapse at any time.

The treatment must be given weekly, for many of the patients, to keep the disease innactive. However, the disease only remains innactive if the patient does not get sick, or have a line infection. Every relapse may become life-threatening and may lead to permanent damage.

The advent of children being treated with Soliris, in all ranges of renal function/failure; has led to the elimination of relapses, so far as the evidence shows thus far, once the damage that is already there completes its cycle. Therefore, to say that kidney transplant with Soliris is the same as receiving FFP or plasmapheresis is not an accurate comparison at all. In addition, there is no evidence supporting the statement that the disease will eventually come back just as it does with FFP or plasmapheresis. As a matter of fact, all the evidence indicates exactly the opposite is true.

The evidence may be clearly tracked by a complete review of the Soliris Forum, where parents of children in all stages of renal function have kindly shared their results and a few are even increasing the times between treatments.

This accurate record keeping in such a public forum is an unpresidented event. As far as I know, it is the only disease where parents/patients have ever been able to see the results of a new medication/treatment while it is still being reviewed by the FDA so easily.

We are indebted to the generous families who have so unselfishly shared their medical records that others may benefit from their experiences! Thank you to all of the families on this site as well for adding your expertise for the record.



Linda Burke said:

At the Oct. 2010 aHUS conference at the University of Iowa, information was presented on a variety of topics, including renal transplants. Although individual patients and their families must weigh their situations carefully with input from both their regular physicians and their transplant teams, currently doctors seem to be leaning toward the "Kidney Transplant with Soliris" as the most viable option rather than a "Kidney/Liver Dual Organ Transplant".
Two strikes are against the Soliris option - one is that many physicians still have not heard of Soliris, which is only FDA approved to stop hemolysis in adults with PNH, so they cannot recommend an option they have not heard of before (Alexion cannot contact doctors, advertise, etc until Soliris obtains FDA approval for aHUS patients.) The second strike is that while aHUS cases are beginning to start the renal transplant option with Soliris, the transplant eval and wait is time-consuming, so no aHUS patients (of which we are aware) have completed a renal transplant with planned Soliris support. ( Apparently, there has been evidence of renal tranplantation that involved Soliris as a successful 'rescue' when problems surfaced post-transplant.)
Two strikes exist against the dual kidney/liver transplant as well- one is that this operation is extremely complex even for physicans/surgeons who have already successfully completed kidney/liver transplants for other aHUS patients. The second strike is that you cannot survive the failure of the liver transplant - the patient always dies in less than 48 hours.
In all, it seems as if the choice for many boils down to this- the Soliris option is less risky and the patient will still live even if the kidney transplant fails (a 2nd kidney transplant would still be a possible future option). The dual kidney/liver transplant used to be the only available option until a few months again because it takes care of the problem of aHUS recurring, yet not all aHUS patients survive. The Soliris option is currently the choice of two adult and several pediatric aHUS patients - though they are all at different stages on their transplant journey. I'm aware of only two aHUS patients/families (KaTrina and Stacy) currently looking at the kidney/liver transplant. Since many of you have sent me your transplant plans by "Send a Message" rather than our public comment walls or Forums, I'll respect your privacy and defer to you as to whether you'd like to announce your transplant news and views to the aHUS online community.

[Stacey]

We are heading to the national liver centre in the UK on the 20th june for a full week for Riley to have transplant assessments. There I am going to ask for more information as even though we are going it is not set in stione that this is the way. I was leaning more towards soliris, but half of me now thinks the liver/kidney is the best option. The doctors are so reassuring and very confident that he is well enough and will do fine. I want Riley to have the best quality of life available and this seems the only option for this. So many people have tried to put me off saying that the risks outweigh the benefits, but in my eyes the doctors know what they are doing and if it was that much of a risk they would have us holding out for soliris instead of encouraging the transplant. I am torn myself but my son is my world and I can't imagine taking him to hospital every week to have this drug administered for the rest of his life. He thrives and he deserves so much better. I will discuss it further when we go for the week, but I think that this is the case at the moment.

KaTrina Slaughter said:

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[Jodi Kayler]

Hi Stacey, I haven't followed your story very close but I just noticed your post and had a couple questions/comments for you.
I wondering why you think he would have to go to the hospital every week for Soliris? Soliris is only once per month and takes 35 min. From what we've experienced over the past 14 months, the quality of life on Soliris is far less complicated than life after a transplant. Coen's life is 100% normal besides a quick infusion. A transplant recipient is on daily lifelong anti-rejection meds which have an entirely new set of complications and risks. Life after a transplant is difficult. It still requires at least monthly labs draws (equal to the trips to the hospital for Soliris) and transplants can sometimes last only last a few years. A recipient is always at risk for graph vs. host disease or other rejection problems. Most kidney transplants only last about 10-15 years. Liver transplants are even more risky than kidney transplants.

I'm only bringing this up so that you have all the information to make your decision. I just don't want you to think that a successful double transplant is a cure and a start of a perfectly normal life. If you can wait until the clinical trials are done on Soliris, you may have more data to base your decision on. As of now, doctors are only hearing about Soliris word of mouth. The manufacturers are not allowed to market the benefits until FDA approval. Because of this, there are many of the very best doctors who still not learned the details about it.

You have big decisions. I don't mean to add to the pressure of making the decision that is best for your family. Best of luck in your quest to find answers.

Stacey said:

We are heading to the national liver centre in the UK on the 20th june for a full week for Riley to have transplant assessments. There I am going to ask for more information as even though we are going it is not set in stione that this is the way. I was leaning more towards soliris, but half of me now thinks the liver/kidney is the best option. The doctors are so reassuring and very confident that he is well enough and will do fine. I want Riley to have the best quality of life available and this seems the only option for this. So many people have tried to put me off saying that the risks outweigh the benefits, but in my eyes the doctors know what they are doing and if it was that much of a risk they would have us holding out for soliris instead of encouraging the transplant. I am torn myself but my son is my world and I can't imagine taking him to hospital every week to have this drug administered for the rest of his life. He thrives and he deserves so much better. I will discuss it further when we go for the week, but I think that this is the case at the moment.

KaTrina Slaughter said:

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[Stacey]

Hi Jodi,
I fully appreciate your information. I live in the UK and my sons consultant hadn't even heard of Soliris. He has looked into a little I think and he is still encouraging us to go for the kidney/liver transplant. I am so confused. I am trying to listen to what the doctors are sayiong here but then listening to others about soliris. Then there are the success stories of both options. My main worry with Soliris is what if it just stops working and all this happens over again. With the liver transplant it will be gone. Please do advise me more anybody because I am ver confused and scared about making the right decision. I have people in one ear saying this is the best route for all these reasons, and then people in the other ear for the other. I just want the best for Riley. To be honest I don't even know where I am anymore or what is going on. It is scary to have such a weight on my back hoping that I do make the best decision for him.

Jodi Kayler said:

Hi Stacey, I haven't followed your story very close but I just noticed your post and had a couple questions/comments for you.
I wondering why you think he would have to go to the hospital every week for Soliris? Soliris is only once per month and takes 35 min. From what we've experienced over the past 14 months, the quality of life on Soliris is far less complicated than life after a transplant. Coen's life is 100% normal besides a quick infusion. A transplant recipient is on daily lifelong anti-rejection meds which have an entirely new set of complications and risks. Life after a transplant is difficult. It still requires at least monthly labs draws (equal to the trips to the hospital for Soliris) and transplants can sometimes last only last a few years. A recipient is always at risk for graph vs. host disease or other rejection problems. Most kidney transplants only last about 10-15 years. Liver transplants are even more risky than kidney transplants.

I'm only bringing this up so that you have all the information to make your decision. I just don't want you to think that a successful double transplant is a cure and a start of a perfectly normal life. If you can wait until the clinical trials are done on Soliris, you may have more data to base your decision on. As of now, doctors are only hearing about Soliris word of mouth. The manufacturers are not allowed to market the benefits until FDA approval. Because of this, there are many of the very best doctors who still not learned the details about it.

You have big decisions. I don't mean to add to the pressure of making the decision that is best for your family. Best of luck in your quest to find answers.

Stacey said:

We are heading to the national liver centre in the UK on the 20th june for a full week for Riley to have transplant assessments. There I am going to ask for more information as even though we are going it is not set in stione that this is the way. I was leaning more towards soliris, but half of me now thinks the liver/kidney is the best option. The doctors are so reassuring and very confident that he is well enough and will do fine. I want Riley to have the best quality of life available and this seems the only option for this. So many people have tried to put me off saying that the risks outweigh the benefits, but in my eyes the doctors know what they are doing and if it was that much of a risk they would have us holding out for soliris instead of encouraging the transplant. I am torn myself but my son is my world and I can't imagine taking him to hospital every week to have this drug administered for the rest of his life. He thrives and he deserves so much better. I will discuss it further when we go for the week, but I think that this is the case at the moment.

KaTrina Slaughter said:

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[KaTrina Slaughter]

Jodi,
Did you have a kidney transplant or are you just doing Soliris?? I am with Stacey, on the fence as to what is the right decision or the best decision. So much information to weigh.

Stacey said:

Hi Jodi,
I fully appreciate your information. I live in the UK and my sons consultant hadn't even heard of Soliris. He has looked into a little I think and he is still encouraging us to go for the kidney/liver transplant. I am so confused. I am trying to listen to what the doctors are sayiong here but then listening to others about soliris. Then there are the success stories of both options. My main worry with Soliris is what if it just stops working and all this happens over again. With the liver transplant it will be gone. Please do advise me more anybody because I am ver confused and scared about making the right decision. I have people in one ear saying this is the best route for all these reasons, and then people in the other ear for the other. I just want the best for Riley. To be honest I don't even know where I am anymore or what is going on. It is scary to have such a weight on my back hoping that I do make the best decision for him.

Jodi Kayler said:

Hi Stacey, I haven't followed your story very close but I just noticed your post and had a couple questions/comments for you.
I wondering why you think he would have to go to the hospital every week for Soliris? Soliris is only once per month and takes 35 min. From what we've experienced over the past 14 months, the quality of life on Soliris is far less complicated than life after a transplant. Coen's life is 100% normal besides a quick infusion. A transplant recipient is on daily lifelong anti-rejection meds which have an entirely new set of complications and risks. Life after a transplant is difficult. It still requires at least monthly labs draws (equal to the trips to the hospital for Soliris) and transplants can sometimes last only last a few years. A recipient is always at risk for graph vs. host disease or other rejection problems. Most kidney transplants only last about 10-15 years. Liver transplants are even more risky than kidney transplants.

I'm only bringing this up so that you have all the information to make your decision. I just don't want you to think that a successful double transplant is a cure and a start of a perfectly normal life. If you can wait until the clinical trials are done on Soliris, you may have more data to base your decision on. As of now, doctors are only hearing about Soliris word of mouth. The manufacturers are not allowed to market the benefits until FDA approval. Because of this, there are many of the very best doctors who still not learned the details about it.

You have big decisions. I don't mean to add to the pressure of making the decision that is best for your family. Best of luck in your quest to find answers.

Stacey said:

We are heading to the national liver centre in the UK on the 20th june for a full week for Riley to have transplant assessments. There I am going to ask for more information as even though we are going it is not set in stione that this is the way. I was leaning more towards soliris, but half of me now thinks the liver/kidney is the best option. The doctors are so reassuring and very confident that he is well enough and will do fine. I want Riley to have the best quality of life available and this seems the only option for this. So many people have tried to put me off saying that the risks outweigh the benefits, but in my eyes the doctors know what they are doing and if it was that much of a risk they would have us holding out for soliris instead of encouraging the transplant. I am torn myself but my son is my world and I can't imagine taking him to hospital every week to have this drug administered for the rest of his life. He thrives and he deserves so much better. I will discuss it further when we go for the week, but I think that this is the case at the moment.

KaTrina Slaughter said:

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[Jodi Kayler]

I just want to say my heartaches thinking about how stressful both your (Stacey & KaTrina) upcoming decisions must be. I’ll give you info from our experiences but please don’t take it as criticism of whatever you decide to do.

My son is 7 years old and was diagnosed at 11 mos. For 6 years, his relapses were constant and violent and has been on dialysis on-and-off again through out his life. Once we started Soliris (4th in the world that we know of), his life has totally changed for the better. The disease is completely non-existent now. Coen’s kidneys do function on their own and he has not had a transplant but there has been significant damage. He will need a transplant down the road and we plan on doing it in conjunction with the Soliris.

The way I see it, AHUS is different than a disease like cancer. aHUS is basically just a dysfunctional immune system—not really a disease. Soliris has successfully 100% blocked the C5 complement of the immune system in every patient it has been used on (both PNH and HUS). There is no way for the HUS activity to resume as long as the C5 is blocked. There are PNH patients that have been on the drug for over 8 years and the drug is still as effective as the day it started—and there are no side effects.

The benefit of Soliris is two-fold: it blocks a portion of his immune system that is confused—but it will also work as an anti-rejection drug for the kidney transplant. Even though we are using Soliris to stop HUS, the added bonus is it will help keep the immune system from fighting the new transplanted organ. As you know, aHUS almost always comes back after a transplant because a foreign object was introduced into the body causing the “confused immune system” to freak out again. Soliris won’t allow the immune system to activate. That’s why we are so confident that it will not only help for a successful transplant but prevent any chance of reoccurrence of the HUS activity.

The way I see it, the nice thing about Soliris, is if it doesn’t work, then you can just stop it. The downside to a liver transplant there is no “Undo”.

On another note, I used to beat myself up that I couldn’t give Coen “a normal life” but then it was pointed out to me that he is happy and optimistic and my guilt was my problem—not his. These kids are strong, compassionate, and optimistic about their future. I think as parents all we can do is make the safest decisions we can and let science catch up. Good luck.


KaTrina Slaughter said:

Jodi,
Did you have a kidney transplant or are you just doing Soliris?? I am with Stacey, on the fence as to what is the right decision or the best decision. So much information to weigh.

Stacey said:

Hi Jodi,
I fully appreciate your information. I live in the UK and my sons consultant hadn't even heard of Soliris. He has looked into a little I think and he is still encouraging us to go for the kidney/liver transplant. I am so confused. I am trying to listen to what the doctors are sayiong here but then listening to others about soliris. Then there are the success stories of both options. My main worry with Soliris is what if it just stops working and all this happens over again. With the liver transplant it will be gone. Please do advise me more anybody because I am ver confused and scared about making the right decision. I have people in one ear saying this is the best route for all these reasons, and then people in the other ear for the other. I just want the best for Riley. To be honest I don't even know where I am anymore or what is going on. It is scary to have such a weight on my back hoping that I do make the best decision for him.

Jodi Kayler said:

Hi Stacey, I haven't followed your story very close but I just noticed your post and had a couple questions/comments for you.
I wondering why you think he would have to go to the hospital every week for Soliris? Soliris is only once per month and takes 35 min. From what we've experienced over the past 14 months, the quality of life on Soliris is far less complicated than life after a transplant. Coen's life is 100% normal besides a quick infusion. A transplant recipient is on daily lifelong anti-rejection meds which have an entirely new set of complications and risks. Life after a transplant is difficult. It still requires at least monthly labs draws (equal to the trips to the hospital for Soliris) and transplants can sometimes last only last a few years. A recipient is always at risk for graph vs. host disease or other rejection problems. Most kidney transplants only last about 10-15 years. Liver transplants are even more risky than kidney transplants.

I'm only bringing this up so that you have all the information to make your decision. I just don't want you to think that a successful double transplant is a cure and a start of a perfectly normal life. If you can wait until the clinical trials are done on Soliris, you may have more data to base your decision on. As of now, doctors are only hearing about Soliris word of mouth. The manufacturers are not allowed to market the benefits until FDA approval. Because of this, there are many of the very best doctors who still not learned the details about it.

You have big decisions. I don't mean to add to the pressure of making the decision that is best for your family. Best of luck in your quest to find answers.

Stacey said:

We are heading to the national liver centre in the UK on the 20th june for a full week for Riley to have transplant assessments. There I am going to ask for more information as even though we are going it is not set in stione that this is the way. I was leaning more towards soliris, but half of me now thinks the liver/kidney is the best option. The doctors are so reassuring and very confident that he is well enough and will do fine. I want Riley to have the best quality of life available and this seems the only option for this. So many people have tried to put me off saying that the risks outweigh the benefits, but in my eyes the doctors know what they are doing and if it was that much of a risk they would have us holding out for soliris instead of encouraging the transplant. I am torn myself but my son is my world and I can't imagine taking him to hospital every week to have this drug administered for the rest of his life. He thrives and he deserves so much better. I will discuss it further when we go for the week, but I think that this is the case at the moment.

KaTrina Slaughter said:

Stacey,
My doctor says the same. We have the exact same symptoms, aHUS with Factor H. Our doctor says that even with Solaris, it is the same as doing Fresh Frozen Plasma weekly treatments like we are doing now. She feels that just a kidney transplant is dangerous and only a matter of time before we have another relapse of HUS. Syd has already had one relapse and it caused major damage.
Our doctor feels that for quality of life the kidney/liver is the best option. With all that said and having talked to Jamie, I am still torn. Fortunately for me, I get to live in my dream world a little while longer as we have yet to hear back from the doctor who said she would take our case and perform the Kidney/Liver transplant. It is a very difficult decision to make. One that I think about all the time when I look at my precious angel and wonder what is the best choise. Just last week she got pertinitis/ear infection and the beginnings of pneumonia. Every time she gets sick, I lie awake wondering if this is it. When those feelings hit, then I want the kidney transplant right away, just so we can have as much free time away from dialysis as possible. But then I wonder, if I would always worry if the aHUS was rearing its ugly head. Not sure what is the right answer, or if one is better than the other. Mostly just think, there has to be a better answer, one that will allow us to look at the night sky, watch fireworks and not rush home to get on the machine, or pay the price of not being able to go anywhere the next day as we finish up our treatment. :) Best wishes. Please stay in touch and let me know how it goes or what option you decide to take. Sorry to dump on you, today is just one of those days with the enormity of it hit. :)

Stacey said:

Katrina,
At the moment i am waiting for Riley's consultant to get back to me with information. He has been researching Soliris. I still think he will advise the kidney/liver transplant for a few reasons. Riley has been so well on PD for a year and obviously for the best quality of life in the future. The doctors in care of Riley are all very confident about this step so now i am just awaiting more info. How are you doing? x

KaTrina Slaughter said:

Stacey. Believe me I understand. We can't find a transplant team willing to take the risk. Where are you at?
At the same time, I am not sure I want an answer either. Would be nice to be off of dialysis and have a "night" life and be able to make it to 8:00 am soccer games. But the risk is so scary. Please keep me posted.

Stacey said:

Reading these messages really worries me. My sons doctors have advised us the best way forward for him is to have the combined kidney/liver transplant. He is ahus with the factor H mutation. We are supposed to be going for transplant assessment soon and i am now becoming really anxious and having second thoughts as to what to do for the best for my son. It would be nice to hear more of your story Jaime Sheeder.

[Joy Lewis O'Brien]

Wow, Jody, what an eloquent response. KaTrina and Stacey, I feel so much for you that you're facing having to make this decision when all you want is a healthy child in the end and you don't know which route is the safest one to take. I wanted to point out that in the UK health system, as I understand, there is often a lag in following new treatments until they've had time to get established and be proven, particularly with pharmaceutical treatments (versus surgical treatments, etc.) Since Soliris hasn't gone through all the approval steps yet, physicians are understandably cautious and want to advise what they KNOW, what they've seen consistently in the research literature over the years. And since aHUS doesn't get a lot of press, they probably have seen the same limited information that we've seen published.

It's probably also important to consider the existing damage to your child's kidneys. If there is minimal damage, then Soliris is probably an excellent way to manage the aHUS with good kidney function. If there is significant diminishment of kidney function, then a physician would probably be more inclined to consider the riskier dual transplantation, since Soliris will only prevent further damage without improving kidney function. Another option to consider is doing just the kidney transplant to replace a poorly functioning kidney, with prophylactic Soliris to prevent relapse (see the article below).

I would emphasize that the transplantation success rate seems to be highly influenced by the type of mutation causing aHUS. There is an article on the research forum with a chart on pp. 1683-1684. Please read that!

The U.S. government has a statistical database that tracks survival rates of recipients of donor organs based on the organ and the age of the recipient. There aren't enough dual liver/kidney transplants for them to track, but for liver alone, the 5 and 10 year survival rates for children aged 6-11...... This is not taking into consideration the kidney transplant as well, OR the aHUS. That's what's these articles mean when they talk about the significant risk of the surgery itself. When the transplant or plasmapheresis for life were the only two options, the transplant surgery looked pretty good by comparison since it was a one time fix (although not a quick fix), and because plasmapheresis offers so many opportunities for future infection as well as the life altering nature of it. Now that there's Soliris available, Soliris is generally considered to be the more conservative approach—as long as the patient's kidneys are still functional.

I can only offer you a few links to some medical journal articles with information about transplants for aHUS, information about the efficacy of Soliris, and the use of Soliris after a kidney transplant (not after a dual transplant).

--Article by Dr. Saland, the transplant physician at Mt. Sinai about the risks and about a successful transplant candidate: http://cjasn.asnjournals.org/cgi/content/full/4/1/201

--Article by Dr. Remuzzi about the failure of a dual transplant in a CFH mutation

--New England Journal of Medicine Article about the successful initial trials of Soliris

--New England Journal of Medicine Article about the use of Soliris after just a kidney transplant

--Before eculizumab hit the scene, a consensus protocol was developed around who was a candidate for transplant and who wasn't. Here's that publication from 2007, which would be important to consider, but does not include Soliris as a treatment option.

--Lastly, an article on the American Journal of Kidney Diseases that says, "Long-term prophylactic plasma exchange and combined liver-kidney transplant have prevented graft loss caused by recurrence. However, the mortality associated with liver transplant is not negligible, and prophylactic plasma exchange requires permanent vascular access and regular hospitalization and exposes the patient to potential allergic reactions to plasma. Eculizumab is a high-affinity humanized monoclonal antibody that binds to C5 and thus prevents generation of C5a and the membrane attack complex. "

[Stacey]

Your information is also very useful. Riley lost his kidney function in June a year ago and has been on dialysis every night since. His kidney function will never return hence he would need a kidney transplant either route. There is no mention of Soliris here in the UK and I have not found any information on any trials. It could be months even years before it becomes available. I have added a new forum Soliris or kidney/liver transplant and asked for peoples stories with the two and any information that any1 has to help me. I am really so confused. Thankyou for all your info x

Joy Lewis said:

Wow, Jody, what an eloquent response. KaTrina and Stacey, I feel so much for you that you're facing having to make this decision when all you want is a healthy child in the end and you don't know which route is the safest one to take. I wanted to point out that in the UK health system, as I understand, there is often a lag in following new treatments until they've had time to get established and be proven, particularly with pharmaceutical treatments (versus surgical treatments, etc.) Since Soliris hasn't gone through all the approval steps yet, physicians are understandably cautious and want to advise what they KNOW, what they've seen consistently in the research literature over the years. And since aHUS doesn't get a lot of press, they probably have seen the same limited information that we've seen published.

It's probably also important to consider the existing damage to your child's kidneys. If there is minimal damage, then Soliris is probably an excellent way to manage the aHUS with good kidney function. If there is significant diminishment of kidney function, then a physician would probably be more inclined to consider the riskier dual transplantation, since Soliris will only prevent further damage without improving kidney function. Another option to consider is doing just the kidney transplant to replace a poorly functioning kidney, with prophylactic Soliris to prevent relapse (see the article below).

I would emphasize that the transplantation success rate seems to be highly influenced by the type of mutation causing aHUS. There is an article on the research forum with a chart on pp. 1683-1684. Please read that!

The U.S. government has a statistical database that tracks survival rates of recipients of donor organs based on the organ and the age of the recipient. There aren't enough dual liver/kidney transplants for them to track, but for liver alone, the 5 and 10 year survival rates for children aged 6-11...... This is not taking into consideration the kidney transplant as well, OR the aHUS. That's what's these articles mean when they talk about the significant risk of the surgery itself. When the transplant or plasmapheresis for life were the only two options, the transplant surgery looked pretty good by comparison since it was a one time fix (although not a quick fix), and because plasmapheresis offers so many opportunities for future infection as well as the life altering nature of it. Now that there's Soliris available, Soliris is generally considered to be the more conservative approach—as long as the patient's kidneys are still functional.

I can only offer you a few links to some medical journal articles with information about transplants for aHUS, information about the efficacy of Soliris, and the use of Soliris after a kidney transplant (not after a dual transplant).

--Article by Dr. Saland, the transplant physician at Mt. Sinai about the risks and about a successful transplant candidate: http://cjasn.asnjournals.org/cgi/content/full/4/1/201

--Article by Dr. Remuzzi about the failure of a dual transplant in a CFH mutation

--New England Journal of Medicine Article about the successful initial trials of Soliris

--New England Journal of Medicine Article about the use of Soliris after just a kidney transplant

--Before eculizumab hit the scene, a consensus protocol was developed around who was a candidate for transplant and who wasn't. Here's that publication from 2007, which would be important to consider, but does not include Soliris as a treatment option.

--Lastly, an article on the American Journal of Kidney Diseases that says, "Long-term prophylactic plasma exchange and combined liver-kidney transplant have prevented graft loss caused by recurrence. However, the mortality associated with liver transplant is not negligible, and prophylactic plasma exchange requires permanent vascular access and regular hospitalization and exposes the patient to potential allergic reactions to plasma. Eculizumab is a high-affinity humanized monoclonal antibody that binds to C5 and thus prevents generation of C5a and the membrane attack complex. "