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. 

[Deborah Deffenbaugh]

Hi Christie....We were very fortunate to have our insurance company approve Soliris and we believe it was due in large part to the efforts of Alyssa's hematologist who wrote an impassioned letter about Alyssa to the carrier and requested a meeting with the head doctor of the carrier.  Her hematologist also has an excellent reputation and a good relationship with the carrier's doctors.   They approved it very quickly.  The approval has to be reviewed every six months but our doctor is optimistic that it will continue to be approved if he can demonstrate it is continuing to work.  She receives 900 mg every two weeks now.  She was receiving 1200 but had some discomfort in terms of facial  flushing and muscle pain (nothing unbearable) so the dose was reduced and she is doing better as far as side effects go.  It will be a trial and error type approach as no one knows the proper dose since this is a new treatment for a-HUS.  We feel especially fortunate that the company (BC/BS Excellus) approved the drug for Alyssa as it is so expensive (your figures are right!) and Alyssa has a close friend, Jill (also on this site) who cannot get approval after several tries.  We are still hopeful something will come through for her ...this is her year for a successful transplant.  Maybe the more patients who have success with a transplant on Soliris will also help convince the carriers to approve as it will be less  experimental.  We wish you all the best in your efforts--if there is anything we can get for you that might help--just let us know.
Ethan Ardoin said:

Hello Deborah,

Can you tell me the steps you took that convinced your insurance carrier to approve the use of Soliris in conjunction with a kidney transplant? Our sons doctor is very interested in moving in this direction. He has indicated that without insurance the cost of the medicine would be extreme ($30,000/month)???  

thanks,

Christie