The Atypical HUS Foundation

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 .
Outside of the USA and Interested in Soliris?  Alexion Pharmaceuticals has a corporate site which lists contact points in 50 nations.  Click HERE for contacts in various nations.
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.)

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Everyone is different.  She is a baby, but our docs watched the labs, talked to Iowa and Alexion on a regular basis and we are now every two weeks.  Initially, I've noticed the numbers are less stable for active aHUS paients when they first received the Soliris.  This is to be expected as the damage has already occurred even though the disease process is on hold.  They did think our son might need to go more often at first because of the wobbling labs, but suddenly he grew and they just increased his dose which worked like a charm.  I expect they are keeping a very close eye on her weight.  Knock on wood, Nathan hasn't had a single cold despite going to school and having a kindergarten sibling, and you know what germ actories they are!  Menengitis is the one thing to fear, and with close monitoring, we can feel relatively relaxed.  Newborns are all more sensitive to germs, but if you are a momy tiger and can limit wishes of oeople to crowd her, I think it would be okay to have Christmas with yiur family, I wouldn't go to stores or anything like that, though.


Dustin G. Wiggins said:

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.

It's a tough balancing act between safeguarding your child against germ 'hotspots' and creating a nuturing, 'norma' childhood experience for your daughter and family - Isla Grace's medical term is best positioned to give advice there.  Personally, our family opted to reduce viral/bacterial load by eliminating close contacts with crowds in enclosed spots during cold season and using lots of anti-bacterial hand and surface wipes.  We chose to live life as fully as we could, using sound judgment to avoid 'hotspots'.

Consider calling 1-888-SOLIRIS for informed discussion about Soliris intervals, immunune issue concerns, and the matters noted in your post.



Dustin G. Wiggins said:

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 son Matt, was diagnosed on Sept 28,2012  he received his first dose of Soliris on Oct 2, it took more than 5 doses of the drug before he made any real significant improvemet, but with his 6th and all subsequent doeses the recovery has been expendential!   He came off dialysis two weeks after being discharged from the hospital, and is back performing on stage.

   I will gladly share more of his labs to show anyone who wants to know how REALLY sick he was, and how close we were to loosing him.   His doctors wanted to take him "off drug" because they didn't think he was responding but because I fought and wouldnt allow them to, he is still with us and off chasing his dreams again.  Keep the faith people, Soliris works!

  It can't repair the damage that occurs BEFORE it's infused, it can stop further assault so that your body can mend itself, so it is CRITICAL, to get on drug ASAP and don't go off drug!

   I just returned from speaking at the Alexion National Sales meeting where I learned even more about my sons case and more about the drug.  Lets not loose one more person/child, because of the delay in getting on drug or because they discontinue drug (because of it's expense) and they want miraculous responses after one or two doses.   My son is proof it can take more!

  God Bless anyone dealing with this disease!

Barb

I am currently getting Soliris every other week and my numbers have been doing good. The only issue I have is that the nurses have a really hard time getting the IV started in my veins. The veins either blow or they move away from the IV needle. Is there anyone that does not have a port have any tips that help you before getting the IV?

 

I have tried drinking water the day before and the day of. They wrap my arms in warm blankets. They suggested that I eat salty foods the day before (but we all know I can't do that b/c of the kidneys). So I'm stuck as to what else to do. Just today they had to stick me 3 times before they finally got one started. It was in my hand, which I hate! :-( Any suggestions are welcome! Thank you!

   When my son began taking his Soliris, he too had trouble with the veins, his nurses used pediatric needles and that seemed to help.  It did get better as he healed and got healthier.  Now they are able to use regular IV needles without any trouble at all.   Actually he only had trouble for about the first 3 months of taking Soliris.

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WELCOME - JOIN US!

The Atypical HUS Foundation 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.

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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 2 cases of aHUS in the U.S. per 1,000,000 of population, and about 60% of aHUS patients are diagnosed as children. The condition is potentially life threatening, and either can be chronic or can recur at intervals.
  
more factoids...

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