Soliris and Kidney Transplant

[Deborah Deffenbaugh]

Our insurance carrier recently approved the use of Soliris in conjunction with a kidney transplant for Alyssa.  She has 2 defects in her I chromosome and a thrombomodulin alteration, any one of whcih could possibly have led to A-HUS--or possibly it took all three as well as an environmental assault such as a flu or birth control pills to push it over the edge to become active at the age of 21.  The Doctors don't really know but they do beleive that there is a 90% liklihood of recurrence of the HUS once she has a transplant, if she does not receive the Soliris.  It is our understanding that she will be the first in the world to have a transplant with the simultaneous use of this drug.  She has been on dialysis for over a year now, having first been diagnosed with A-HUS in January 1009.  We are hopeful that maybe her own kidney function might return as she has never been given Soliris before and maybe her kidneys could repair themselves if the hemolysis is completely stopped.  Unfortunately the Doctors are not hopeful that there is much chance for that but I never give up hope.   The plan is to have her go on pherisis and soliris for a week or two before the transplant. The actual transplant would take place in Iowa where they have some experience with A-HUS.   Alyssa is very fortunate to have a live donor who has volunteered and so far has been compatible.  We live in Rochester, New York so the logistics of all of this is going to be difficult but of course we will do whatever it takes.  Alyssa has been through so much and has not felt well in such a long time.  She does the peritoneal dialysis  and certainly likes that much better than the hemodialysis.  Blood pressures have been eiher way too high or more recently too low.  Since there is always the fear of infection, we are planning to move as quickly as possible now that we have the approval.  It looks like we will likely go to Iowa at the end of June or early July.   If anyone has any information or suggestions that could help us, please let us know.  I know there are several children using Soliris and I am grateful for any input. 

[Phyllis Ann Talbot]

Deborah - thank you SO much for sharing this information! Unfortunately I don't have anything to help you but we are VERY excited to watch this. Our son Hyde is 3 1/2 and has been on PD since June of 2008. He has a factor H mutation and also has a very high chance of recurrence with a kidney transplant only. Currently he's doing well - running around like a maniac, etc and what Alyssa is doing it basically our next step - thanks again for sharing your story and know that your family will be in our thoughts and prayers here in Atlanta!

[Linda Burke]

Hi Debbie,
Thanks for adding this Forum, I'm sure that many in the aHUS community will be watching for all the positive news that is sure to accompany Alyssa's transplant journey. Since a menningococal vaccine is important to have on board prior to beginning Soliris (due to the increased risk for menningitis), Alyssa's docs have probably made arrangements for giving her the vaccine (if it hasn't been given already). Apparently, 3 out of 4 strains are covered by the vaccine but the vaccine for that particularly nasty 4th kind of menningitis won't be ready yet for a couple of years. Our docs put Skyler on 250mg of Amoxycillin (one pill daily) as a proactive measure 'just in case', although I'm aware that other Soliris kids do not follow that protocol of a preventive dose of Amoxycillin
You might want to run a c5 functional assay to get Alyssa's baseline before starting the Soliris. We did Skyler's c5 functional assay at the Mayo Clinic (turnaround time is 4 days) to gather baseline data before stopping his Soliris infusions in June and hope this detailed lab test will give us further info about infusion amounts, time intervals between infusions, etc. When he first went on Soliris in April 2009, we sent Skyler's blood directly to Alexion Pharmaceutical's lab and I believe that one of the tests they ran was a c5 complement level (but not a functional assay). Check out the left side of the Home Page and under the 'Doc to Doc Directory' you'll find the lab test info under "Regarding Soliris".
Feel free to 'send a message' and ask for details or specific info, I'll be happy to respond. Alyssa and your family will be in our prayers as we send you every best wish for a speedy and successful outcome!

[Deborah Deffenbaugh]

Thanks Linda--I appreciate your comments. Alyssa is sceduled to get the menningitis vaccine but I don't really seem to be clear on whether it should be no more than 2 weeks before starting soliris or at least 2 weeks. Do you know whether the fact that Alyssa's defect is not the CFH, which is the more common defect, has any affect on the viability of the Soliris? I assume the body has the same response if HUS is active and the derivation shouldn't matter but I happened to think of the fact that most of the patients using Soliris for this disorder probably all have a different defect than Alyssa's. Hopefully that won't matter. I am also wondering the time frame for starting the Soliris since it seems to take a little time to take full effect. Do you think two weeks is reasonable? Alyssa's hemotologist is Dr. Peter Kouides and he is working in conjunction with Dr. Smith and Dr. Thomas in Iowa. I did tell Dr. Kouides about the Doc to Doc link available through this site but I don't think he has used it. Thanks in advance for any information you can give me. I was thinking maybe my doctor should be in touch directly with Alexion regarding their research on Soliris. I think he used to stay in touch with a rep as he opened a study at Rochester General when Alyssa was first admitted but by the time the study was approved, her platelet count was not low enough and her numbers did not fit the parameters of the study.

Linda Burke said:

Hi Debbie,
Thanks for adding this Forum, I'm sure that many in the aHUS community will be watching for all the positive news that is sure to accompany Alyssa's transplant journey. Since a menningococal vaccine is important to have on board prior to beginning Soliris (due to the increased risk for menningitis), Alyssa's docs have probably made arrangements for giving her the vaccine (if it hasn't been given already). Apparently, 3 out of 4 strains are covered by the vaccine but the vaccine for that particularly nasty 4th kind of menningitis won't be ready yet for a couple of years. Our docs put Skyler on 250mg of Amoxycillin (one pill daily) as a proactive measure 'just in case', although I'm aware that other Soliris kids do not follow that protocol of a preventive dose of Amoxycillin
You might want to run a c5 functional assay to get Alyssa's baseline before starting the Soliris. We did Skyler's c5 functional assay at the Mayo Clinic (turnaround time is 4 days) to gather baseline data before stopping his Soliris infusions in June and hope this detailed lab test will give us further info about infusion amounts, time intervals between infusions, etc. When he first went on Soliris in April 2009, we sent Skyler's blood directly to Alexion Pharmaceutical's lab and I believe that one of the tests they ran was a c5 complement level (but not a functional assay). Check out the left side of the Home Page and under the 'Doc to Doc Directory' you'll find the lab test info under "Regarding Soliris".
Feel free to 'send a message' and ask for details or specific info, I'll be happy to respond. Alyssa and your family will be in our prayers as we send you every best wish for a speedy and successful outcome!

[Bill Biermann]

Deborah,

like the Talbots and everyone on this site, we are so excited for Alyssa!

We look forward to hearing all about the preperation, tests ect. leading up to the actual surgery as well as how she does during and after surgery.

Please let us all know when things happen as we can muster the troops to send up prayers for you, Alyssa, and everyone who loves and supports her!

Thank you so much for the update.

[Grace]

That's wonderful news, Deborah! I hope and pray that all does well for Alyssa and she can return to her better-than-usual self ASAP!

C5 is the point of the major positive feedback loop of all complement pathways, amplifying the cascade. AHUS mutations so far negate or override the negative feedback which dampens the complement activity. As a C5 inhibitor, Soliris should in theory shut down complement before its endpoint regardless of what the inciting event is. But of course we're all waiting with bated breath to see what happens in reality.

[Heather Still]

Wonderful news for your family. Best wishes with it all and God Bless!!!

[Sylwia Antkowiak]

Hi Debbie,
My 6year old daughter has CFI and lost her kidneys though we hoped for months that they come back. She is also on pd at the moment. Our plan will be probably similiar to yours, use soliris for the kidney transplant. We are in London and our doctors still want to wait for a little bit longer for more answers from medical world .We will have to be put on the waiting list as although I am a match I have the same CFI ( though it never manifested in my case) and consequently cannot be a donor. I will be following your story closely and praying that everything goes well.

[Kerri Grey]

Hi Deborah, thats great news about your daughter, my son Ashley is using Solaris and he has no known mutation, the Solaris still wprks in the same way for all types. Ashley will one day need a transplant due to the damage that had already been done to his kidneys before he started solaris and the docs have told us that they will not do a live donor transplant with him for a couple of reasons 1. the risk that someone in the family might have the same mutation 2. the ethical issues involved in taking a kidney from a live person when the chances of recurrance are so high. Please keep us updated on how things go as my hope is that one day Ashley will have his transplant in conjunction with the use of solaris.
Cheers Kerri

[Deborah Deffenbaugh]

Hi Kerri--Thanks for the response. Our live donor is not a blood relation (niece by marriage). I am surprised your doctors think that the risk of recurrance is so high as we were told that with Soliris, there should not be any recurrance. There is a recent study in the New England Journal of Medicine with a successful A-HUS transplant with the use of Soliris. Especially since Ashley has already been on Soliris, I would think a future transplant should be less risky. Hopefully there will be more and more success stories with A-HUS and Soliris.

Kerri Grey said:

Hi Deborah, thats great news about your daughter, my son Ashley is using Solaris and he has no known mutation, the Solaris still wprks in the same way for all types. Ashley will one day need a transplant due to the damage that had already been done to his kidneys before he started solaris and the docs have told us that they will not do a live donor transplant with him for a couple of reasons 1. the risk that someone in the family might have the same mutation 2. the ethical issues involved in taking a kidney from a live person when the chances of recurrance are so high. Please keep us updated on how things go as my hope is that one day Ashley will have his transplant in conjunction with the use of solaris.
Cheers Kerri

[Cheryl Biermann]

One of the reasons for not letting live donnors from the family, especially uknown causes, is for the sake of the family member. They may have the same hidden gene and later on may be triggered with aHUS and only have one kidney to cope with the attack.

Deborah Deffenbaugh said:

Hi Kerri--Thanks for the response. Our live donor is not a blood relation (niece by marriage). I am surprised your doctors think that the risk of recurrance is so high as we were told that with Soliris, there should not be any recurrance. There is a recent study in the New England Journal of Medicine with a successful A-HUS transplant with the use of Soliris. Especially since Ashley has already been on Soliris, I would think a future transplant should be less risky. Hopefully there will be more and more success stories with A-HUS and Soliris.

Kerri Grey said:

Hi Deborah, thats great news about your daughter, my son Ashley is using Solaris and he has no known mutation, the Solaris still wprks in the same way for all types. Ashley will one day need a transplant due to the damage that had already been done to his kidneys before he started solaris and the docs have told us that they will not do a live donor transplant with him for a couple of reasons 1. the risk that someone in the family might have the same mutation 2. the ethical issues involved in taking a kidney from a live person when the chances of recurrance are so high. Please keep us updated on how things go as my hope is that one day Ashley will have his transplant in conjunction with the use of solaris.
Cheers Kerri

[Kerri Grey]

HI Deborah,
Because Solaris is not readily available here in Australia for HUS Ashley's docs can not use that at this stage as part of the consideration involved in a transplant when it comes to the risk of the disease recurring - Ashley is the first and only person in Australia using Solaris for aHUS and that is only because Alexion agreed to let him use it after everything else did not work and they are paying for Ash to use it and we dont know at this stage how long they will agree to pay for it. My hope is that one day Solaris use for aHUS patients will become readily available and that when the time comes for Ash to have a transplant we will be able to use Solaris in conjunction with transplant and then as you said there should hopefully not be such a great risk for disease recurrence but until then i dont think Ash's docs would even consider a live donor transplant.

Deborah Deffenbaugh said:

Hi Kerri--Thanks for the response. Our live donor is not a blood relation (niece by marriage). I am surprised your doctors think that the risk of recurrance is so high as we were told that with Soliris, there should not be any recurrance. There is a recent study in the New England Journal of Medicine with a successful A-HUS transplant with the use of Soliris. Especially since Ashley has already been on Soliris, I would think a future transplant should be less risky. Hopefully there will be more and more success stories with A-HUS and Soliris.

Kerri Grey said:

Hi Deborah, thats great news about your daughter, my son Ashley is using Solaris and he has no known mutation, the Solaris still wprks in the same way for all types. Ashley will one day need a transplant due to the damage that had already been done to his kidneys before he started solaris and the docs have told us that they will not do a live donor transplant with him for a couple of reasons 1. the risk that someone in the family might have the same mutation 2. the ethical issues involved in taking a kidney from a live person when the chances of recurrance are so high. Please keep us updated on how things go as my hope is that one day Ashley will have his transplant in conjunction with the use of solaris.
Cheers Kerri

[Gene Billingsley]

That's great to hear Deborah. Faith can move mountains - but they don't necessarily move all in one giant chunk. Transplant+Soliris really is an incredible step for our kids. We'll keep praying for full original kidney recovery on top of that and hopefully not need Soliris one day!

We, too, are on a kidney transplant workup path. Hopefully our case will be referred for review by the end of July.

Best of luck and all our prayers!