Soliris

[Linda Burke]

Currently an aHUS patient using or considering treatment with Soliris? Please add your voice to this Forum!
Several members of this website have children whose aHUS is being treated with IV doses of Soliris, a complement inhibitor that is FDA approved for paroxysmal nocturnal hemoglobinuria (PNH). 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 is that Soliris apparently halts 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 have resulted in 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 [e-mail] 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.)

[Sarah Brewer]

[Sarah Brewer]

[Cheryl Biermann]

We've seen it!  People working with us sent the link last week, and it is wonderful, I reposted to fb and emailed everyone I know who would actually watch it.  It is great to actually hear these stories of success before the child has long-lasting damage, so it is a recognition to our family and friends who have stood by us and raised money and awareness for atypicalHUS for the last ten years...Yes, ten years!  This March will be our tenth anniversary of fund raising!  What a year it has been.  Thank you for sharing the good news, it's like icing on the cake!

[Svetlana Finley]

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.

[Linda Burke]

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.

[Svetlana Finley]

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?