The Foundation for Children with Atypical HUS

Currently an aHUS patient using or considering treatment with Soliris? Please add your voice to this Forum!
Many members of this website have children or adult family members whose aHUS is being treated with IV doses of Soliris, a complement inhibitor that is FDA approved for paroxysmal nocturnal hemoglobinuria (PNH) and for atypical hemolytic uremic syndrome (aHUS). Created by Alexion Pharmaceuticals, Soliris (eculizumab) is an antibody against terminal complement protein C5 that inhibits complement activation. Data currently available suggests that Soliris can create a blockade of complement c5 to maintain patient kidney function with reduced platelet consumption and hemolysis. Our interest in the aHUS community began with reports that Soliris apparently halted hemolysis in adults with PNH, preventing the kidney damage that is also a devastating hallmark of aHUS activity.  Clinical trials for Soliris use in aHUS patients resulted in 2011 FDA approval for Soliris use in aHUS patients (http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm272990...).

 

Soliris is a recombinant humanized monoclonnal antibody, derived from human IgG2 and IgG4 sequences. Composed of two 448 amino acid (protein) heavy chains and two 214 amino acid light chains, Soliris is supplied in 30 mL single-use vials containing 300mg of eculizumab and administered via IV infusion.  Further technical details are available on this website in the "How Soliris Works" Forum with information courtesy of www.rxlist.com/soliris-drug.htm .

 

The company website at www.alxn.com has a tab dedicated to Soliris and PNH patients, where participants in clinical trials noted that Soliris was generally well tolerated with some patients reporting adverse side effects such as headache, runny nose (nasopharyngitis), back pain, nausea and tiredness (fatigue).   It is very important to note that patients using Soliris are at increased risk of meningococcal infections, and so need to receive a meningococcal vaccine at least 2 weeks prior to beginning treatment with Soliris.

 

Information is available at 1-888-SOLIRIS, Alexion's OneSource connection staffed with knowledgable RNs who will assist aHUS patients and their families with various facets involved with Soliris therapy. Alexion's corporate website at www.soliris.net provides information about Soliris as it pertains to PNH, a rare disorder with some similar hemolyzing characteristics.  With the advent of FDA approval for Soliris use in aHUS patients on Sept. 23, 2011, it's anticipated that Alexion will be able to expand patient and family contact with a wide variety of aHUS patient supports and a specialized website devoted to aHUS concerns, issues, and innovative solutions.

 

Alexion Global Headquarters (Tel: 203-272-ALXN) is in Conneticut, but the 'Contact Us' area of their website lists contact info for France, Belgium, Germany, Italy, Spain, England (UK), Tokyo (Japan), and Australia.  Parents/aHUS patients who call (203) 272-ALXN first will be asked to give their name, followed by a request for the physcian's contact information.  Consider asking your physician to utilize the "Doc to Doc Registry" on our Home Page if you are interested in Soliris for an aHUS patient.  Physicians with inquiries regarding Soliris may contact the Medical Information Department of Alexion Pharmaceuticals, Inc at 1.888.SOLIRIS (765-4747)

 

Physicians formulating letters seeking insurance coverage of Soliris therapy to maintain aHUS patient's renal grafts can request the Foundation's helpful template.  Please email linda@atypicalhus.org with your office/hospital contact info.
 
Parents- For information regarding Soliris therapy, please direct questions to Alexion Pharmaceuticals at 1-888-SOLIRIS .
Video:  Presenting at Dr. Tim Goodship's aHUS Family Conference held in the UK in June 2011, Neil Sheerin presents information focusing on the the mechanics of the disease process and treatment of rare disease caused by excessive and uncontrolled complement activation. The role of complement, a description of eculizumab (Soliris) as an anti-C5 monoclonal antibody, clinical trial info, and actual patients using this therapy are the included in this presentation. http://ahus.org.uk/videos/Neil/
 
Neil Sheerin is the Professor of Nephrology at Newcastle University and a Consultant Nephrologist at the Freeman Hospital, Newcastle upon Tyne. His laboratory research has focused on understanding how abnormal activation of the immune system leads to kidney disease, in particular his the role of the complement system. http://ahus.org.uk/speakers.html#neil-sheerin
 
NOTE regarding potential for aHUS damage in organs other than the kidneys
The topic of aHUS complications has surfaced in multiple commentary, referring to potential effects of aHUS damage that may impact other vital organs such as the heart, bowel, or brain.  Multi-organ involvement in aHUS patients has been referenced by multiple researchers, and patients/families/medical personnel may wish to begin their exploration of this topic at  www.ahussource.com , a website provided by Alexion Pharmaceuticals (makers of Soliris, or eculizumab).  Commentary at  http://atypicalhus.ning.com/profiles/blogs/ahus-complications-poten...

 

(Note:  Unfortunately, I'm sorry to report that our Ning Network platform has no mechanism to allow the most current posts to appear first in a Forum.  Please start on the last page for most recent updates and comments.)

Tags: Alexion, Soliris, eculizumab

Views: 4341

Reply to This

Replies to This Discussion

Good news, Sveta - I know you're been anxious for Anna to start Soliris.  Please keep us posted on her progress and labs so that our other aHUS families can better consider their options.

Svetlana Finley said:

HI everyone, Anna started her Soliris treatments last week. Even her bloof work was good we decited to start Soliris based on her kidney biopsy results, constantly having blood and proteine in her urine and back pains. This week it was finally she didnt had any blood or proteine that was great news for us. My doctor is planning to do Soliris for 6-12 month ten we will decide if we want to stop.

Anna just finished her 4th treatment. 

before first treatment her labs:

PLT 245

Hemoglobin 11.8

Hematocrit 35

2nd

PLT 267

Hemaglobin 12.7

Hematocrit 37.4

3rd she had very small cold

PLT 260

Hemoglobin 12.4

Hematocrit 36.7

4th

PLT 323

Hemoglobin 13.1

Hematocrit 38.6

She is been doing very well. Before her treatments she had at least 3 headaches a week now she had only once in this 4 weeks.

Only little problem we got in to when she had cold and she got treatment on same day she got worse by that evening running fevers they had to give her strong shot antibiotics to stop her what we believe was cold virus. Next week we will be getting bigger dose and then go by weekly. Anyone had any problems with colds? 

At 4:59pm on October 13, 2012, Barbara Farchersaid…   

am hoping someone can help shed some ligt on progress??  My son was pretty darn sick two weeks ago and has been told he had aHUS.  No genetics yet but all the signs are there.   He presented on a friday for the first time, really really sick platelets went down to 14,000.  hemoglobin down to 6, creatinin 12 BUN 85, LDH over 2000 and very jaundice oh and BP 200/100    by the way my son is 21 years old 6' and 200 lbs and currently starring as Gaston in the national tour of Beauty and the Beast so i can promise you this kid was in GREAT shape. Doesn't drink doesnt' do steroids, works out, runs, eats good...pretty much all around good health.  Which we hope is what helped save him.  Anyway...thank God for great diagnosis at the ER they immediately suspected TTP which we have a cousin who died of TTP so the genetics may be there.  They got plasmapharesis going in less than 24 hours, dialysis shortly thereafter and began Soliris in 72 hours.   They pharessed him 5 times before Solaris infusion and not since.  He did get 4 units of blood over the first week and not since.   His platelets hit 95,000 the first week.  He got his next Solaris infusion and platelets went to 88,000 and now today 4 days after his 2nd Solaris platelets are at 72,000   Can anyone else relate to this kind of cycle?  The Hemo dr came out and said "not everyone responds to Soliris"  and he told us my son may still die.   I am hoping anyone out there can help shed light on this roller coaster we are all on??      His BP is being nicely controlled with oral meds, his hemogloben is around 8.3 no transfusions in 10 days, he doesn't vomit, holds down food , LDH is at 1150, Creatinin between 8 and 9 depending upon if he gets dialysis.  The alexion people are not throwing in the towel they think he is healing...but can anyone who has been through this tell me your story and hopefully give me a happy ending?

So sorry to hear about your son.  There are many similarities between my story and his. When I started getting Solaris, my platelets started rising within days. Once they got to a normal range they did drop once, but nothing out of normal range. My prayers and thoughts are with your son, you, and your family.  When I read your story to my mom, we both couldn't believe how familiar it sounded.  I am hoping that it will just take more time for his platelets to respond and rise because they were so low.  The lowest mine were was 65,000.  Sounds like your medical team is very on top of things. I am guessing that they ruled out the TTP?  

  


Barbara Farcher said:

At 4:59pm on October 13, 2012, Barbara Farchersaid…   

am hoping someone can help shed some ligt on progress??  My son was pretty darn sick two weeks ago and has been told he had aHUS.  No genetics yet but all the signs are there.   He presented on a friday for the first time, really really sick platelets went down to 14,000.  hemoglobin down to 6, creatinin 12 BUN 85, LDH over 2000 and very jaundice oh and BP 200/100    by the way my son is 21 years old 6' and 200 lbs and currently starring as Gaston in the national tour of Beauty and the Beast so i can promise you this kid was in GREAT shape. Doesn't drink doesnt' do steroids, works out, runs, eats good...pretty much all around good health.  Which we hope is what helped save him.  Anyway...thank God for great diagnosis at the ER they immediately suspected TTP which we have a cousin who died of TTP so the genetics may be there.  They got plasmapharesis going in less than 24 hours, dialysis shortly thereafter and began Soliris in 72 hours.   They pharessed him 5 times before Solaris infusion and not since.  He did get 4 units of blood over the first week and not since.   His platelets hit 95,000 the first week.  He got his next Solaris infusion and platelets went to 88,000 and now today 4 days after his 2nd Solaris platelets are at 72,000   Can anyone else relate to this kind of cycle?  The Hemo dr came out and said "not everyone responds to Soliris"  and he told us my son may still die.   I am hoping anyone out there can help shed light on this roller coaster we are all on??      His BP is being nicely controlled with oral meds, his hemogloben is around 8.3 no transfusions in 10 days, he doesn't vomit, holds down food , LDH is at 1150, Creatinin between 8 and 9 depending upon if he gets dialysis.  The alexion people are not throwing in the towel they think he is healing...but can anyone who has been through this tell me your story and hopefully give me a happy ending?

Yes totally ruled out TTP, Thank goodness the doctors here in Houston (we are from NY but he was here for the show) are VERY familiar with TTP, we are going to send out for genetic testing hopefully to the U. of Iowa labs.  But so many things are making it very clear it's aHUS.  Such an awful disease when it presents big like this?!  We are now looking back at some early warning signs but nothing that would send you to a Dr nevermind a hospital and with his touring schedule being exhausted made perfect sence!  As I said we are staying positive and the Soliris people feel he has a longer journey back from such a low platelet count.  We like the way they think and we will continue the infusions for as long as possible.

Thank you so much for responding...and my heart goes out to you and your mom!  I pray for your continued healing.

Barb
 
Jennifer Christopher said:

So sorry to hear about your son.  There are many similarities between my story and his. When I started getting Solaris, my platelets started rising within days. Once they got to a normal range they did drop once, but nothing out of normal range. My prayers and thoughts are with your son, you, and your family.  When I read your story to my mom, we both couldn't believe how familiar it sounded.  I am hoping that it will just take more time for his platelets to respond and rise because they were so low.  The lowest mine were was 65,000.  Sounds like your medical team is very on top of things. I am guessing that they ruled out the TTP?  

  


Barbara Farcher said:

At 4:59pm on October 13, 2012, Barbara Farchersaid…   

am hoping someone can help shed some ligt on progress??  My son was pretty darn sick two weeks ago and has been told he had aHUS.  No genetics yet but all the signs are there.   He presented on a friday for the first time, really really sick platelets went down to 14,000.  hemoglobin down to 6, creatinin 12 BUN 85, LDH over 2000 and very jaundice oh and BP 200/100    by the way my son is 21 years old 6' and 200 lbs and currently starring as Gaston in the national tour of Beauty and the Beast so i can promise you this kid was in GREAT shape. Doesn't drink doesnt' do steroids, works out, runs, eats good...pretty much all around good health.  Which we hope is what helped save him.  Anyway...thank God for great diagnosis at the ER they immediately suspected TTP which we have a cousin who died of TTP so the genetics may be there.  They got plasmapharesis going in less than 24 hours, dialysis shortly thereafter and began Soliris in 72 hours.   They pharessed him 5 times before Solaris infusion and not since.  He did get 4 units of blood over the first week and not since.   His platelets hit 95,000 the first week.  He got his next Solaris infusion and platelets went to 88,000 and now today 4 days after his 2nd Solaris platelets are at 72,000   Can anyone else relate to this kind of cycle?  The Hemo dr came out and said "not everyone responds to Soliris"  and he told us my son may still die.   I am hoping anyone out there can help shed light on this roller coaster we are all on??      His BP is being nicely controlled with oral meds, his hemogloben is around 8.3 no transfusions in 10 days, he doesn't vomit, holds down food , LDH is at 1150, Creatinin between 8 and 9 depending upon if he gets dialysis.  The alexion people are not throwing in the towel they think he is healing...but can anyone who has been through this tell me your story and hopefully give me a happy ending?

That must have been scary.  I have never heard of anyone not responding to Soliris. We have had someone come off because they had severs allergic reaction, but have not heard any more on that.  The wobbling numbers may indicate more frequent infusions, but hopefully, your docs are in direct contact with Alexion, makers of the drug.   We had to increase our son's dose following a post transplant growth spurt.  Besides tranfusions and plasma pheresis, there are some patients who respond to steroids check out that option with the doctors.  Keep the news coming, you'll also have a lot of people praying for him.

Is Alexion telling you not everyone responds to Soliris?  That's where I would look for the answer to that question.  They have all the studies.  The wobbling numbers are common and comming back from a devastatingly severe flare up is hard.  There are other ways to treat this, sometimes they use steroids to control activity which works for some people to a certain extent.  

There is a theory that may explain some of this.  We know that certain people seem to flare up withaHUS as adults and that is the first time.  Sometimes, however, they may have been having low level activity too minute to measure, it could have been going on in any of the major organs.  Then something happens, a trigger,(which isn't important, really except that it causes aHUS to ramp up the person, now an adult is very sick.  The science we know is low levels of activity in multiple organs, but no diagnosis or classic signs of aHUS.    

            

Hi Barbara,
So sorry to hear about your son!!! I'm so glad there has been an improvement and that Soliris was available for your son to start straight away. I am from Australia and my son Ashley has been on Solariis for a little over 3 and a half years. When he first began Soliris he didn't respond that quickly either, he continued to have plasma exchange 3 times a week followed by a Soliris infusion for about 3 months (in that 3 months slowly drew it out to be forthrightly). So I understand your concern, we thought we were going to that 1 person Soliris was not going to work for, however Ashley is doing great now!!! Ashley still has low hb levels and his LDH levels have NEVER been in the normal limits so Ashley's doc says he will never stop treatment but even if there is a little bit of background disease activity it is at this stage not enough to do any damage!!! I hope the platelets start improving for you so that you can breath a little easier!!!
Cheers Kerri

Cheryl Biermann said:

Is Alexion telling you not everyone responds to Soliris?  That's where I would look for the answer to that question.  They have all the studies.  The wobbling numbers are common and comming back from a devastatingly severe flare up is hard.  There are other ways to treat this, sometimes they use steroids to control activity which works for some people to a certain extent.  

There is a theory that may explain some of this.  We know that certain people seem to flare up withaHUS as adults and that is the first time.  Sometimes, however, they may have been having low level activity too minute to measure, it could have been going on in any of the major organs.  Then something happens, a trigger,(which isn't important, really except that it causes aHUS to ramp up the person, now an adult is very sick.  The science we know is low levels of activity in multiple organs, but no diagnosis or classic signs of aHUS.    

            

My daughter Isla was diagnosed with ahus at the beginning of October.  She is now 8 months old.  She presented with acute renal failure, severe swelling, high blood pressure, and hemolysis.  We started Soliris on the third day of hospitilization.  She took three treatments weekly and we are now on a three week schedule.  By the third treatment, and five transfusions, her hemoglobin and platelets seemed to stabilize and her kidney function returned normal.  I attribute this to a combination of good doctors, prayer, and soliris(not in any specific order).  I do have some questions concerning the soliris treatment.  We are on our second three week treatment.  Her hemoglobin has been declining slowly.  It is now at 10.2 vs 11.9 when we left the hospital.  Her platelets have also been steadily declining but went up a little on last weeks blood test.  Are most of you all on a three week treatment or are the treatments two weeks apart.  I am sure everyone is different but I wondered if there is better stability with the two week treatments or if these fluctuations in blood chemistry are expected no matter frequency of treatments.  My other question is on compromised immunity.  I know soliris is an immunosuppresant.  My question is how careful should we be.  Right now we have been extra careful.  We have skipped out on taking her to Thanksgiving functions but would really like to take her to our family Christmas.  My family has several children who go to school and we are debating on it because of the possible influx of germs.  We also avoid public places such as everywhere except the hospital in fear of contracting something that could trigger a relapse.  I know most of you have dealt with this disease for much longer than we have and thought you all might have some advice on the social interaction part.  Any information is much appreciated.  Also i would just like to say that this site is great.  With this disease being so rare it is extremely helpful to have a site that we can all share information with others battling this disease.

My daughter is 14yr and she gets Soliris every other week, that is what on Soliris protocol, idk if anyone have longer space in between at least i didn't hear, but i thin everyone is on the same. You can contact Alexion and ask them any question about Soliris treatment, they are awesome there. Do you have case nurse set up with them? if not you should do that. 

 I think u can take Isla to family Christmas party, just let everyone know that she can't be around sick people, if there is anyone with cold they should stay home :) I think until she gets better stable numbers you should stay away from big public crowds and unknown people. 

Thank you so much for your information.  We will definitely contact Alexion and set up a case nurse.  Most kids I have read about on here seem to be on the two week treatment.  I thought it might be for her age and size, but I have read some others that have shorter and longer treatment intervals.  Thank you again for your information.


 
Svetlana Finley said:

My daughter is 14yr and she gets Soliris every other week, that is what on Soliris protocol, idk if anyone have longer space in between at least i didn't hear, but i thin everyone is on the same. You can contact Alexion and ask them any question about Soliris treatment, they are awesome there. Do you have case nurse set up with them? if not you should do that. 

 I think u can take Isla to family Christmas party, just let everyone know that she can't be around sick people, if there is anyone with cold they should stay home :) I think until she gets better stable numbers you should stay away from big public crowds and unknown people. 

Until my daughter was stable the doctor watched her numbers to see when to give next Soliris treatment. Twice they gave it to her early. Now we are on a schedule of every other Thursday and she is doing well with it. My daughter who is 11 was diagnosed this September. Out of hospital ar end of Sept. we kept her home for about a month until numbers were in normal range then she was allowed to return to classes and dance. I think you could take your daughter to Christmas so long as nobody is sick. Honestly my daughter has had worst cold and germ exposure from her siblings, we are a family of seven. With diligent hand washing, keeping sick person away from her and lots of prayer she hasn't gotten any of their colds.

Reply to Discussion

RSS

IN REGARD TO MEMBERSHIP REQUESTS

To ensure proper processing of your membership, please make sure to set your email filters to accept emails, from Linda@atypicalhus.org.

The Foundation for Children for Children with Atypical HUS is an all-volunteer organization. Please allow at least 72 hours for an email response confirming your membership request.  If you do not receive an email, please check your spam folder or email directly to Linda@atypicalhus.org

Membership is open to patients, family, friends, caregivers research and medical personnel.

WELCOME - JOIN US!

The Foundation for Children with Atypical HUS encourages patients and investigators to share information and explore options/resources as we work together to gain insight into this rare complement disorder. By increasing contact opportunities with researchers and medical personnel interested in helping the aHUS community, our stories foster a better understanding of atypical hemolytic uremic syndrome.

Sharing information, inspiration and support for one another, we seek to gather together people and knowledge as we strive to improve the lives of patients and families dealing with a diagnosis of aHUS.


NEW DIAGNOSIS OF aHUS?
Be proactive! Get the medical basics of aHUS, what lab values to monitor, and areas of concern...check out the "aHUS Bootcamp" and "About aHUS" tabs at the top of this page!
If your doctor has never treated a case of aHUS, please print out our 'Doc to Doc Registry' and ask him/her to contact a physician versed in the complexities of aHUS and new options for 2011 genetic testing and treatment.

************************

NOTE: Our 'Send a Message" function on each Member's page allows for private discussion of personal content. As with any social network, be cautious about giving personal contact information (home email info, phone number) until you have an established relationship with another person, organization, or associated website.

 

Did you know...

CFH (Serum Complement Factor H) is a regulatory protein. The secreted protein product of CFH consists of 20 repetitive units named "short consensus repeats" or SCRs (each approximately 60 amino acids). In patients with aHUS the last 5 "pearls" in the twenty pearl strand protein, SCR16 - SCR20, should bind to protect cells but do not- they are defective in one or more of the last 5 SCR locations. If they cannot bind or stick to the kidney to protect that tissue, the platelets clump into clots that affect the glomeruli of the kidney -potentially causing acute renal failure.
  
• • • • • • • • • • • •
  
It is estimated that there are about 300 cases of aHUS in the U.S., and it is most common with young children. The condition is life threatening and either can be chronic or can recur at intervals.
  
more factoids...

Help us fight the battle

Your tax-deductible purchase of one or more bracelets will directly fund research to help that aHUS families live with every day. Each bracelet has an appraised value of $825, but your purchase price is only $295.

  

  
Or you can donate a specific dollar amount-every dollar will help our research efforts of the Foundation for Children with atypical HUS at www.atypicalhus.org and www.atypicalHUS.net


Donations may be made via credit card or Paypal. If you prefer, checks made payable to The Foundation for Children with Atypical HUS may be mailed to:

Foundation for Children with Atypical HUS
C/O Deborah Lewis
PO Box 333
Barnhart, MO 63012


For pearl bracelet orders, please allow extra time for processing checks. Questions about aHUS donations? Please email Linda@atypicalhus.org


Badge

Loading…

© 2014   Created by ALPHA MARKETING.   Powered by

Badges  |  Report an Issue  |  Terms of Service