Soliris or kidney/liver transplant

[Stacey]

Please, please please help me guys. I am so confused. I have been told so many pro's and con's for the dual transplant and the same for Soliris. At the moment my son Riley's consultant is encouraging us down the route of the dual organ transplant but with so many different inputs I am starting to have doubts. Please could you inundate me on any information whether it be positive or negative on both possibilities, and how life will be after either route. Riley has been on dialysis for 12 months now and will never regain kidney function so either way the kidney transplant is vital. Any information will be helpful and fully appreciated. Thankyou

Stacey

[Phyllis Ann Talbot]

Stacey - I can only tell you our plans on for our son - Hyde will be 4 in July and has been on dialysis since April 2008 so over 2 years - we are pursuing approval for a kidney transplant along with Soliris. We have stalled making a decision because we were so very scared of the liver/kidney transplant. Our main concerns were the VERY riskines of the actual surgery as well as the fact that - we'd be transplanting a liver when 99.9% of what a liver needs to do - Hyde's does - and if at some point in the future his liver failed - there isn't a 'stall' tactic while waiting for a new liver while for a kidney - there's almost always a dialysis option to 'stall' until you can get a new kidney. That being said - we could very well have pursued the liver kidney option at some point as that was probably the best option available prior to recent developments with Soliris. So given the risks vs the benefits - we have decided to pursue Soliris with a kidney - we know it will be risky as well given that it is an experimental drug and it' been used VERY limitedly in kidney transplants so far - but several are in the works currently and we just feel that this is the best option today for Hyde. Our other hope is that going forwad - there will be contnued advancements in treatments and even a cure - so even the once ever 2, 3, or 4 week infusion might not be necessary - and compared to nightly dialysis at home these infusions seem very minor to us personally at this point from a lifestyle perspective.

Not sure anyone can tell you what's best for you and your family - I can just tell you the avenue me and my family are pursuing and the reasons behind it. Good luck and know that you are in our thoughts and prayers!

[Stacey]

Thankyou Phyllis. Has there been any kidney transplants with soliris that you know of? I know I will know what is the right path for my son in the end. Just feel as though I need to do a lot more delving first. Again thankyou for your point of view, as Hyde is in exactly the same situation as Riley x

Phyllis Ann Talbot said:

Stacey - I can only tell you our plans on for our son - Hyde will be 4 in July and has been on dialysis since April 2008 so over 2 years - we are pursuing approval for a kidney transplant along with Soliris. We have stalled making a decision because we were so very scared of the liver/kidney transplant. Our main concerns were the VERY riskines of the actual surgery as well as the fact that - we'd be transplanting a liver when 99.9% of what a liver needs to do - Hyde's does - and if at some point in the future his liver failed - there isn't a 'stall' tactic while waiting for a new liver while for a kidney - there's almost always a dialysis option to 'stall' until you can get a new kidney. That being said - we could very well have pursued the liver kidney option at some point as that was probably the best option available prior to recent developments with Soliris. So given the risks vs the benefits - we have decided to pursue Soliris with a kidney - we know it will be risky as well given that it is an experimental drug and it' been used VERY limitedly in kidney transplants so far - but several are in the works currently and we just feel that this is the best option today for Hyde. Our other hope is that going forwad - there will be contnued advancements in treatments and even a cure - so even the once ever 2, 3, or 4 week infusion might not be necessary - and compared to nightly dialysis at home these infusions seem very minor to us personally at this point from a lifestyle perspective.

Not sure anyone can tell you what's best for you and your family - I can just tell you the avenue me and my family are pursuing and the reasons behind it. Good luck and know that you are in our thoughts and prayers!

[Phyllis Ann Talbot]

Hi - as far as we know - there are only 2 published accounts so far - one was an adult in Germany who had a kidney transplant, then her AHUS relapsed and she was treated with Soliris and saved the transplant - and was still doing wll after one year. The other case was a young boy (maybe 10 or 12?) who did a kidney transplant only and then began Soliris within a few days and again - was doing well after 1 year. (This was in the last New Englan Journal of Medicine - but I think the little boy was in Germany again? definitely somewhere in Europe). There is also a young lady here in the states who is planning on doing kidney with prophylactic Soliris within the next month - so we feel sure by the time we get through all the hurdles - there will be a few more for our dr's to learn from. Good luck with whatever decision you make!

[Stacey]

The only problem is hardly anything is known about soliris over here in the UK and i have urged Rileys doctor to research it and he has but still thinks the kidney/liver transplant is the best option for Riley. I don't know what to do whether to keep pestering him or go with his advice, but like i said there is hardly any info on the drug over here. Could be years before it is even trialled.

Phyllis Ann Talbot said:

Hi - as far as we know - there are only 2 published accounts so far - one was an adult in Germany who had a kidney transplant, then her AHUS relapsed and she was treated with Soliris and saved the transplant - and was still doing wll after one year. The other case was a young boy (maybe 10 or 12?) who did a kidney transplant only and then began Soliris within a few days and again - was doing well after 1 year. (This was in the last New Englan Journal of Medicine - but I think the little boy was in Germany again? definitely somewhere in Europe). There is also a young lady here in the states who is planning on doing kidney with prophylactic Soliris within the next month - so we feel sure by the time we get through all the hurdles - there will be a few more for our dr's to learn from. Good luck with whatever decision you make!

[Bill Biermann]

We will be pursuing kidney/Soliris with Nathan, as this is what the information from all the research and evidence being accumulated is indicated is the very best option for people with aHUS. Nathan, like many, has had lots of damage over the years, so we are hoping to watch a few of the patients with less damage to their vascular systems go first, but circumstances may force us to go sooner rather than later as he also has other complications that can only be made worse by continuing on dialysis. We have full confidence in the kidney/Soliris option and would probably not be waiting if he were one of the "healthier" aHUS patients.

Stacey said:

The only problem is hardly anything is known about soliris over here in the UK and i have urged Rileys doctor to research it and he has but still thinks the kidney/liver transplant is the best option for Riley. I don't know what to do whether to keep pestering him or go with his advice, but like i said there is hardly any info on the drug over here. Could be years before it is even trialled.

Phyllis Ann Talbot said:

Hi - as far as we know - there are only 2 published accounts so far - one was an adult in Germany who had a kidney transplant, then her AHUS relapsed and she was treated with Soliris and saved the transplant - and was still doing wll after one year. The other case was a young boy (maybe 10 or 12?) who did a kidney transplant only and then began Soliris within a few days and again - was doing well after 1 year. (This was in the last New Englan Journal of Medicine - but I think the little boy was in Germany again? definitely somewhere in Europe). There is also a young lady here in the states who is planning on doing kidney with prophylactic Soliris within the next month - so we feel sure by the time we get through all the hurdles - there will be a few more for our dr's to learn from. Good luck with whatever decision you make!

[Stacey]

Does anyone have any information about when trials end? or when they start in the UK? Or even when Soliris will be approved? I know so little and so do the docs over here. I don't like to pester Riley's doctor that is why i am trying to explore more myself.

Bill Biermann said:

We will be pursuing kidney/Soliris with Nathan, as this is what the information from all the research and evidence being accumulated is indicated is the very best option for people with aHUS. Nathan, like many, has had lots of damage over the years, so we are hoping to watch a few of the patients with less damage to their vascular systems go first, but circumstances may force us to go sooner rather than later as he also has other complications that can only be made worse by continuing on dialysis. We have full confidence in the kidney/Soliris option and would probably not be waiting if he were one of the "healthier" aHUS patients.

Stacey said:

The only problem is hardly anything is known about soliris over here in the UK and i have urged Rileys doctor to research it and he has but still thinks the kidney/liver transplant is the best option for Riley. I don't know what to do whether to keep pestering him or go with his advice, but like i said there is hardly any info on the drug over here. Could be years before it is even trialled.

Phyllis Ann Talbot said:

Hi - as far as we know - there are only 2 published accounts so far - one was an adult in Germany who had a kidney transplant, then her AHUS relapsed and she was treated with Soliris and saved the transplant - and was still doing wll after one year. The other case was a young boy (maybe 10 or 12?) who did a kidney transplant only and then began Soliris within a few days and again - was doing well after 1 year. (This was in the last New Englan Journal of Medicine - but I think the little boy was in Germany again? definitely somewhere in Europe). There is also a young lady here in the states who is planning on doing kidney with prophylactic Soliris within the next month - so we feel sure by the time we get through all the hurdles - there will be a few more for our dr's to learn from. Good luck with whatever decision you make!

[Linda Burke]

Alexion Pharmaceuticals has a UK branch and the contact info is as below:

[e-mail]

14 Horizon Business Village
1Brooklands Road, Weybridge
Surrey KT13 OTJ United Kingdom

Here in the US, a nurse would take down your name, the patient's name, and a few basic details of the aHUS situation (dialysis, plasmapheresis, transplant, etc), After providing Alexion Pharmaceuticals with the patient's providers - physican, name and contact info of physican's practice, treating hospital's name- you'd be asked to have your doctor contact Alexion. By contacting Alexion, your case can be 'tracked internally' - think of it as registering your interest in learning about Soliris, so when Riley's physican makes a follow-up call they have some background details. I would imagine that the UK branch follows the identical company protocol.
Hope this helps you obtain info, I'll continue to send along any facts that might be useful to you as you ponder transplant issues. Wishing you the best, Linda

[Stacey]

Linda, thankyou for this information. I emailed alexion UK a few weeks ago and they emailed me back saying they had no information for me

Linda Burke said:

Alexion Pharmaceuticals has a UK branch and the contact info is as below:

[e-mail]

14 Horizon Business Village
1Brooklands Road, Weybridge
Surrey KT13 OTJ United Kingdom

Here in the US, a nurse would take down your name, the patient's name, and a few basic details of the aHUS situation (dialysis, plasmapheresis, transplant, etc), After providing Alexion Pharmaceuticals with the patient's providers - physican, name and contact info of physican's practice, treating hospital's name- you'd be asked to have your doctor contact Alexion. By contacting Alexion, your case can be 'tracked internally' - think of it as registering your interest in learning about Soliris, so when Riley's physican makes a follow-up call they have some background details. I would imagine that the UK branch follows the identical company protocol.
Hope this helps you obtain info, I'll continue to send along any facts that might be useful to you as you ponder transplant issues. Wishing you the best, Linda

[Linda Burke]

Which, of course, is not an acceptable answer....yet here in the United States Alexion's communications are bound by federal law, since Soliris is not yet FDA approved. I'm guessing that may be the case in England, too. Try calling again, with this framework in mind:
1) Call Alexion UK again - have the contact info for Riley's doc(s) at the ready.
2) State your interest - in your case, a renal transplant with planned Soliris therapy to prevent recurrance of aHUS
3) Ascertain if our guess is right, and that Alexion can't talk directly to parents in England, either. Ask them to contact Riley's doc(s) and provide 'internal tracking' -creating an Alexion file on Riley's case- as you intend to follow up on obtaining information about possible Soliris use for your child.
4) Alexion CAN provide info to doctors through the Chief Medical Officer, so your doctor will be able to access Soliris info and then inform you. Remember, most doctors do not yet know about Soliris as it's just being utilized for aHUS patients and most physicians never see a single case of aHUS in their entire medical career (so they wouldn't be up-to-date on the lastest breakthroughs in this field)..
Yes, I hate getting everything second-hand, too but I keep telling myself that the U.S. federal drug regulations exist to protect my family, and I do trust Skyler's docs as they work hard to keep themselves and us well informed. Besides, I wonder how much technical info I'd be able to understand even if Alexion could release all info directly to me, since the complement pathway is complex and aHUS is so highly variable in its presentations. Still, I'll be glad when Soliris is FDA approved and I can call Alexion and chat with a knowledgeable nurse representative.
I always try to remember, it's my Mom responsibility to chase down all info until you have a good handle on all options available - even if it means that you have to spend lots of time on emails, the phone, or reading research articles. If I don't spend the time and effort to inform myself, who else would do that for my child?

Stacey said:

Linda, thankyou for this information. I emailed alexion UK a few weeks ago and they emailed me back saying they had no information for me

Linda Burke said:

Alexion Pharmaceuticals has a UK branch and the contact info is as below:

[e-mail]

14 Horizon Business Village
1Brooklands Road, Weybridge
Surrey KT13 OTJ United Kingdom

Here in the US, a nurse would take down your name, the patient's name, and a few basic details of the aHUS situation (dialysis, plasmapheresis, transplant, etc), After providing Alexion Pharmaceuticals with the patient's providers - physican, name and contact info of physican's practice, treating hospital's name- you'd be asked to have your doctor contact Alexion. By contacting Alexion, your case can be 'tracked internally' - think of it as registering your interest in learning about Soliris, so when Riley's physican makes a follow-up call they have some background details. I would imagine that the UK branch follows the identical company protocol.
Hope this helps you obtain info, I'll continue to send along any facts that might be useful to you as you ponder transplant issues. Wishing you the best, Linda

[Sylwia Antkowiak]

Hi

I contacted alexion uk some time ago when I heard of soliris for the first time. I checked the address now and it looks like they moved. They are based now at the address given by Linda


Alexion Pharma UK Ltd
14 Horizon Business Village , Weybridge, Surrey, KT13 0TJ, UK
Telephone: [phone]
Fax: [phone]

When I sent them an email I received a response pretty quickly from

Dr Jon Beauchamp

Medical Affairs Manager


Mob: [phone]

Email: [e-mail]

He is the person to contact when it comes to soliris in the uk. I have also spoken to my doctor recently - Dr Rees at Great Ormond Hospital in London. The trials of soliris on pediatirc patients in the UK are supposed to start mid year . I think unless I am wrong that when they announce that the trial starts it may/ will be undertaken in different countries simultaneoulsy. ( different study centres ?) Anyway Dr Rees said that Great Ormond Hospital will be leading on these trials in the UK and I think she is the one that anyone interested in the UK trials of Soliris should talk to. Hope that helps.





Cheryl Biermann said:

Hang in there Stacey,

Linda is right-when there is no help, you create the help. 10 years ago there wasn't even a blog where parents could get a look at what is happening to their children-no researchers available to talk to you and explain the course of the disease and certainly no foundation raising money to fund research.

Every phone call with little or no results can make you feel like you've been slapped in the face. Eventually, though, someone listens with their heart and brains working in unison and says yes, maybe I have an idea that will work. Or know someone who has been down this road before & will give them your name and they call you with the steps they took and possible alternative ideas of their own-then you have a "map" & will add your own info. to it. The trick is being patient but keep working at it!

Keep coming here with what your efforts have resulted in and that way you will have a much larger group to pull ideas/solutions from. A great place to begin is to take the steps Linda outlined today. You may have to have someone in the house to help you a while-a kindly old lady or a grandparent or reliable teen, someoone who can be there to make sure the regular day-to-day gets done while you devote yourself to this important work...good luck. Please come back here also for fill-ups of encouragement when needed!

Linda Burke said:

Which, of course, is not an acceptable answer....yet here in the United States Alexion's communications are bound by federal law, since Soliris is not yet FDA approved. I'm guessing that may be the case in England, too. Try calling again, with this framework in mind:
1) Call Alexion UK again - have the contact info for Riley's doc(s) at the ready.
2) State your interest - in your case, a renal transplant with planned Soliris therapy to prevent recurrance of aHUS
3) Ascertain if our guess is right, and that Alexion can't talk directly to parents in England, either. Ask them to contact Riley's doc(s) and provide 'internal tracking' -creating an Alexion file on Riley's case- as you intend to follow up on obtaining information about possible Soliris use for your child.
4) Alexion CAN provide info to doctors through the Chief Medical Officer, so your doctor will be able to access Soliris info and then inform you. Remember, most doctors do not yet know about Soliris as it's just being utilized for aHUS patients and most physicians never see a single case of aHUS in their entire medical career (so they wouldn't be up-to-date on the lastest breakthroughs in this field)..
Yes, I hate getting everything second-hand, too but I keep telling myself that the U.S. federal drug regulations exist to protect my family, and I do trust Skyler's docs as they work hard to keep themselves and us well informed. Besides, I wonder how much technical info I'd be able to understand even if Alexion could release all info directly to me, since the complement pathway is complex and aHUS is so highly variable in its presentations. Still, I'll be glad when Soliris is FDA approved and I can call Alexion and chat with a knowledgeable nurse representative.
I always try to remember, it's my Mom responsibility to chase down all info until you have a good handle on all options available - even if it means that you have to spend lots of time on emails, the phone, or reading research articles. If I don't spend the time and effort to inform myself, who else would do that for my child?

Stacey said:

Linda, thankyou for this information. I emailed alexion UK a few weeks ago and they emailed me back saying they had no information for me

Linda Burke said:

Alexion Pharmaceuticals has a UK branch and the contact info is as below:

[e-mail]

14 Horizon Business Village
1Brooklands Road, Weybridge
Surrey KT13 OTJ United Kingdom

Here in the US, a nurse would take down your name, the patient's name, and a few basic details of the aHUS situation (dialysis, plasmapheresis, transplant, etc), After providing Alexion Pharmaceuticals with the patient's providers - physican, name and contact info of physican's practice, treating hospital's name- you'd be asked to have your doctor contact Alexion. By contacting Alexion, your case can be 'tracked internally' - think of it as registering your interest in learning about Soliris, so when Riley's physican makes a follow-up call they have some background details. I would imagine that the UK branch follows the identical company protocol.
Hope this helps you obtain info, I'll continue to send along any facts that might be useful to you as you ponder transplant issues. Wishing you the best, Linda

[Linda Burke]

The International Conference for HUS, MPGN, and PNH met from June 13th to June 15th, 2010 in Innsbruck Austria and featured presentations from many top aHUS researchers from around the globe. Alexion Pharmaceuticals has issued a news release outlining conference presentations, which included 6 cases of aHUS renal transplants that were supported by Soliris therapy. If you are pondering a the possibility of a renal transplant supported by Soliris to prevent a recurrance of aHUS, these case studies would provide vital information to consider. http://www.alxn.com/News/article.aspx?relid=479502