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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Skyler was diagnosed with factor H related aHUS activity on March 26, 2009 and promptly began 11 days of plasmapheresis. We were already well versed about aHUS and had knowledeable medical staff ready - our wonderful son Hunter died in May 2008 during an attempted kidney/liver transplant.
Skyler received his first of 4 weekly doses of 300mg of Soliris on April 7th. On May 5, 2009 Skyler received his first 600mg "megadose" of Soliris with treatment of this IV infusion to continue once every two weeks. Tomorrow is his 4th 600mg infusion of Soliris, with no labs or other medical interventions in between. Skyler's only medication now is one 250mg Amoxycillin dose daily, a prophylactic dose just as a preventive measure. Hooray, Skyler is an active and clever boy whose labs have remained at or near normal for a well over a month now!
Hi there - we are also watching all developments with Soliris. Currently we are on PD and doing well. We don't qualify for the first round of studies on Soliris both b/c of age and also the fact that we are on dialysis. So Hyde would need to do Soliris in conjunction with a kidney transplant.

We most likely won't be able to wait long enough to be in an actual trial - and have discussed requesting a compassionate waiver with our dr's - but while he's stable - we are going to wait to see how much other information comes out before pursuing other options.
Coen has been receiving Soliris since April 22. For all those who love labs, I’ll try to give a rundown of lab values since the start of Soliris. I may have dates off by a day or two…let me know if you have questions
At the start of Soliris, Coen was hospitalized and getting daily pheresis, IVIG twice a week, hemo dialysis and getting RBC transfusions as needed. Once Soliris is started pheresis and dialysis were stopped completely. Soliris protocol is identical to Skyler's.
• April 22: First dose of Soliris: Platelets 220. Very high BP. Creatinine 3.0.
• Day 2: Platelets 250. Starts feeling a little better. No pheresis. No dialysis.
• Day 3: Platelets 260. Still shows schistocytes. Urine output starts to improve. Lots of energy. Feeling good.
• Day 4: Platelets 281. RBC increases. Creatinine 2.8. Urine output continues to improve but there is unexpected whole red blood cells in urine. No schistocytes.
• Day 5: PLT 254. Creatinine decreases. BP coming down. Feeling great. No more red cells in urine. Electrolytes start becoming stable. Ins & Outs match.
• April 29: 2nd dose of Soliris. No dialysis or pheresis for 1 week. PLT 232. Kidney function stable. Creatinine 2.4. Dialysis catheter removed. Released from hospital.
• May 5: 3rd dose of Soliris. PLT 261 Creatinine 2.1. Back in school and feeling great.
• May 12: 4th dose of Soliris PLT 330 (highest ever). Creatinine 1.9. BP good.
• May 19: PLTs down to 190. Creatinine 1.5. Feeling great. No signs of schistocytes. No explainable reason for PLT drop.
• June 2: PLT 384 (another record high) Creatinine 1.6. HCT 35.0. No pheresis or dialysis since April. BP meds have been reduced.
Anna is on Plasma exchange, our dr. is waiting for study be available. She told us they are writing protocol for kids age and it should be ready to go in few month. Anna also on BP medications and iron supplements. She used to get IV Iron, but we stopped finally. She never been on dyalasys and her kidney functions are 0.5-0.6. We still waiting on her genetic testing.
Below is a copy of an article I read about the upcoming clinical trials of Soliris. Hope it's legal for me to post here!!! :)

New England Journal of Medicine Publishes Case Reports on the Investigational Use of Soliris (eculizumab) in Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
Alexion To Begin Clinical Trials of Soliris® in aHUS Patients
CHESHIRE, Conn.--(BUSINESS WIRE)--Jan 29, 2009 - Two separate case reports published today in the New England Journal of Medicine (NEJM) examine the investigational use of Soliris ® (eculizumab), a terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), in patients with a rare and severe inflammatory disease called atypical Hemolytic Uremic Syndrome (aHUS). In both cases, physicians observed a significant reduction in the destruction of red blood cells, reduced platelet consumption and improved kidney function following Soliris therapy.

Separately, Alexion announced today that it is currently initiating clinical trials of eculizumab in patients with aHUS. Soliris is approved in the United States, European Union, and Canada for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).
Atypical Hemolytic Uremic Syndrome is characterized by hemolysis, thrombocytopenia and clotting of blood vessels (microangiopathy), particularly in the kidney and brain, often progressing to end-stage kidney disease. Like PNH, aHUS is caused by a deficiency in normally occurring complement inhibitors. Typically, patients with aHUS have genetic mutations in one of several complement inhibitor proteins that lead to uncontrolled complement activation. Excessive complement activation may contribute to severe inflammation of the blood vessels and blood clotting through the activation of white blood cells, platelets, and the endothelial cell lining of blood vessels. (1)
The prognosis for patients with aHUS is poor. Approximately 70% of patients with the most common mutation experience chronic renal insufficiency, chronic dialysis, or death by one year after the first clinical episode. (2) Following kidney transplantation, recurrent aHUS causes kidney transplant failure in approximately 62 - 88% of patients. (3)
Case Reports
In a case report submitted to the NEJM by Ralph A. Gruppo, M.D., Director of the Comprehensive Hemophilia and Thrombosis Center at the Cincinnati Children's Hospital Medical Center, an 18-month-old infant was admitted to the hospital following a fourth clinically severe relapse of congenital aHUS. During this episode, the patient did not respond to daily plasmapheresis, a procedure whereby proteins are removed from the blood by circulating the patient's blood through a machine. When the patient's condition deteriorated further, the physician administered Soliris. Complete blockade of terminal complement was observed in this patient, and hematologic and renal improvement began within 48 hours after initiation of treatment. Plasmapheresis was discontinued within the first week of eculizumab treatment and clinical remission was sustained throughout the 60 day observation period. The report further notes that eculizumab therapy is ongoing for over four months, to date, with sustained disease remission and no further plasma therapy intervention.
“It is well known that aHUS is a devastating disease without effective treatment options,” explained Dr. Gruppo, lead author of the report. “Further, after clinical worsening, there may be virtually nothing a physician can do to prevent further kidney damage and eventual kidney failure. Based on this initial and very limited experience, further studies of terminal complement inhibition with eculizumab are warranted.”
In a second case reported in the same issue of the NEJM by Dr. Jens Nuernberger of the Department of Nephrology at University Duisburg-Essen in Essen, Germany, a 37-year-old woman with a history of kidney failure due to aHUS and loss of her first kidney transplant due to recurrent aHUS, was admitted to the hospital with progressive and severe aHUS shortly after her second kidney transplant. The patient's aHUS condition continued to clinically worsen despite extensive plasma treatments, indicating a high probability that the second kidney transplant would fail. After the administration of eculizumab, hemolysis quickly resolved, platelet count rebounded and kidney transplant function recovered. The patient's renal graft function has remained stable.
Eculizumab appeared to be well tolerated in these two patients, with safety observations that have been consistent with those reported from controlled trials with eculizumab in patients with PNH.
"There is a profound need to improve the management of aHUS. We are encouraged by the initial clinical experience with eculizumab in a very limited number of aHUS patients, and we are undertaking prospective clinical trials to investigate the role of complement inhibition in this condition," said Leonard Bell, M.D., Chief Executive Officer of Alexion.
Upcoming Clinical Studies
Alexion is currently initiating four prospective, open-label clinical studies of eculizumab as a treatment for patients with aHUS in North America and multiple European countries: two studies of patients who are plasma therapy sensitive (one in adults and one in adolescents) and two studies of patients who are plasma therapy resistant (one in adults and one in adolescents). Information on the trials can be requested by e-mail using the address clinicaltrials@alxn.com, or by visiting the Alexion website at www.alexionpharma.com and clicking on the clinical trials link. The trials also will be posted to the www.clinicaltrials.gov website maintained by the U.S. National Institutes of Health
Another article about the drug.
Attachments:
Hi Phyllis,
In speaking with a rep. from Soliris, they are considering lowering the age of trial participants. I had originally heard that age 12 and older was part of the study's parameters, so in my phone conversation I raised age as a concern since many aHUS kids are diagnosed at an early age. Apparently, they have reconsidered the age issue so you (or your doctor) may wish to check directly with Alexion Pharmaceuticals on that point. Skyler seemed to have no problem obtaining the drug once we decided to go that route. Please keep in mind that our family is merely reporting out on Skyler's treatment for any interested parties wondering how Soliris is working for a factor H atypical HUS child. We'll keep you posted on Skyler's lab values here as well as any advancements or treatment glitches - stay tuned....

Phyllis Ann Talbot said:
Hi there - we are also watching all developments with Soliris. Currently we are on PD and doing well. We don't qualify for the first round of studies on Soliris both b/c of age and also the fact that we are on dialysis. So Hyde would need to do Soliris in conjunction with a kidney transplant.

We most likely won't be able to wait long enough to be in an actual trial - and have discussed requesting a compassionate waiver with our dr's - but while he's stable - we are going to wait to see how much other information comes out before pursuing other options.
Thanks for posting these, Heather!

Heather Still said:
Below is a copy of an article I read about the upcoming clinical trials of Soliris. Hope it's legal for me to post here!!! :)

New England Journal of Medicine Publishes Case Reports on the Investigational Use of Soliris (eculizumab) in Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
Alexion To Begin Clinical Trials of Soliris® in aHUS Patients
CHESHIRE, Conn.--(BUSINESS WIRE)--Jan 29, 2009 - Two separate case reports published today in the New England Journal of Medicine (NEJM) examine the investigational use of Soliris ® (eculizumab), a terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), in patients with a rare and severe inflammatory disease called atypical Hemolytic Uremic Syndrome (aHUS). In both cases, physicians observed a significant reduction in the destruction of red blood cells, reduced platelet consumption and improved kidney function following Soliris therapy.

Separately, Alexion announced today that it is currently initiating clinical trials of eculizumab in patients with aHUS. Soliris is approved in the United States, European Union, and Canada for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).
Atypical Hemolytic Uremic Syndrome is characterized by hemolysis, thrombocytopenia and clotting of blood vessels (microangiopathy), particularly in the kidney and brain, often progressing to end-stage kidney disease. Like PNH, aHUS is caused by a deficiency in normally occurring complement inhibitors. Typically, patients with aHUS have genetic mutations in one of several complement inhibitor proteins that lead to uncontrolled complement activation. Excessive complement activation may contribute to severe inflammation of the blood vessels and blood clotting through the activation of white blood cells, platelets, and the endothelial cell lining of blood vessels. (1)
The prognosis for patients with aHUS is poor. Approximately 70% of patients with the most common mutation experience chronic renal insufficiency, chronic dialysis, or death by one year after the first clinical episode. (2) Following kidney transplantation, recurrent aHUS causes kidney transplant failure in approximately 62 - 88% of patients. (3)
Case Reports
In a case report submitted to the NEJM by Ralph A. Gruppo, M.D., Director of the Comprehensive Hemophilia and Thrombosis Center at the Cincinnati Children's Hospital Medical Center, an 18-month-old infant was admitted to the hospital following a fourth clinically severe relapse of congenital aHUS. During this episode, the patient did not respond to daily plasmapheresis, a procedure whereby proteins are removed from the blood by circulating the patient's blood through a machine. When the patient's condition deteriorated further, the physician administered Soliris. Complete blockade of terminal complement was observed in this patient, and hematologic and renal improvement began within 48 hours after initiation of treatment. Plasmapheresis was discontinued within the first week of eculizumab treatment and clinical remission was sustained throughout the 60 day observation period. The report further notes that eculizumab therapy is ongoing for over four months, to date, with sustained disease remission and no further plasma therapy intervention.
“It is well known that aHUS is a devastating disease without effective treatment options,” explained Dr. Gruppo, lead author of the report. “Further, after clinical worsening, there may be virtually nothing a physician can do to prevent further kidney damage and eventual kidney failure. Based on this initial and very limited experience, further studies of terminal complement inhibition with eculizumab are warranted.”
In a second case reported in the same issue of the NEJM by Dr. Jens Nuernberger of the Department of Nephrology at University Duisburg-Essen in Essen, Germany, a 37-year-old woman with a history of kidney failure due to aHUS and loss of her first kidney transplant due to recurrent aHUS, was admitted to the hospital with progressive and severe aHUS shortly after her second kidney transplant. The patient's aHUS condition continued to clinically worsen despite extensive plasma treatments, indicating a high probability that the second kidney transplant would fail. After the administration of eculizumab, hemolysis quickly resolved, platelet count rebounded and kidney transplant function recovered. The patient's renal graft function has remained stable.
Eculizumab appeared to be well tolerated in these two patients, with safety observations that have been consistent with those reported from controlled trials with eculizumab in patients with PNH.
"There is a profound need to improve the management of aHUS. We are encouraged by the initial clinical experience with eculizumab in a very limited number of aHUS patients, and we are undertaking prospective clinical trials to investigate the role of complement inhibition in this condition," said Leonard Bell, M.D., Chief Executive Officer of Alexion.
Upcoming Clinical Studies
Alexion is currently initiating four prospective, open-label clinical studies of eculizumab as a treatment for patients with aHUS in North America and multiple European countries: two studies of patients who are plasma therapy sensitive (one in adults and one in adolescents) and two studies of patients who are plasma therapy resistant (one in adults and one in adolescents). Information on the trials can be requested by e-mail using the address clinicaltrials@alxn.com, or by visiting the Alexion website at www.alexionpharma.com and clicking on the clinical trials link. The trials also will be posted to the www.clinicaltrials.gov website maintained by the U.S. National Institutes of Health
Good specific info, Jodi - thanks for sharing!

Jodi Kayler said:
Coen has been receiving Soliris since April 22. For all those who love labs, I’ll try to give a rundown of lab values since the start of Soliris. I may have dates off by a day or two…let me know if you have questions
At the start of Soliris, Coen was hospitalized and getting daily pheresis, IVIG twice a week, hemo dialysis and getting RBC transfusions as needed. Once Soliris is started pheresis and dialysis were stopped completely. Soliris protocol is identical to Skyler's.
• April 22: First dose of Soliris: Platelets 220. Very high BP. Creatinine 3.0.
• Day 2: Platelets 250. Starts feeling a little better. No pheresis. No dialysis.
• Day 3: Platelets 260. Still shows schistocytes. Urine output starts to improve. Lots of energy. Feeling good.
• Day 4: Platelets 281. RBC increases. Creatinine 2.8. Urine output continues to improve but there is unexpected whole red blood cells in urine. No schistocytes.
• Day 5: PLT 254. Creatinine decreases. BP coming down. Feeling great. No more red cells in urine. Electrolytes start becoming stable. Ins & Outs match.
• April 29: 2nd dose of Soliris. No dialysis or pheresis for 1 week. PLT 232. Kidney function stable. Creatinine 2.4. Dialysis catheter removed. Released from hospital.
• May 5: 3rd dose of Soliris. PLT 261 Creatinine 2.1. Back in school and feeling great.
• May 12: 4th dose of Soliris PLT 330 (highest ever). Creatinine 1.9. BP good.
• May 19: PLTs down to 190. Creatinine 1.5. Feeling great. No signs of schistocytes. No explainable reason for PLT drop.
• June 2: PLT 384 (another record high) Creatinine 1.6. HCT 35.0. No pheresis or dialysis since April. BP meds have been reduced.
Our doctors contacted investigators to obtain Soliris info directly, and I'm pleased to report that they had great success with their emails and phone contacts. Our terrific nephrology team doctors spent much time and effort checking into Soliris issues before Skyler began this treatment. Thanks for adding this, Heather.

Heather Still said:
Another article about the drug.
Linda - thanks! yes - our dr I think has been pushing for them to lower the age as well - but they are also not including soliris along with transplants on the first phase of testing - so we mostly likely won't qualify either way. however - we most likely could still be approved - just not under the study - when we decide to move forward. At the moment PD is going well and Hyde's still growing - so we are just going to wait and see what happnens......of course - at any point our hand could be forced depending on what goes on wtih Hyde. We'll see.....

Linda Burke said:
Hi Phyllis,
In speaking with a rep. from Soliris, they are considering lowering the age of trial participants. I had originally heard that age 12 and older was part of the study's parameters, so in my phone conversation I raised age as a concern since many aHUS kids are diagnosed at an early age. Apparently, they have reconsidered the age issue so you (or your doctor) may wish to check directly with Alexion Pharmaceuticals on that point. Skyler seemed to have no problem obtaining the drug once we decided to go that route. Please keep in mind that our family is merely reporting out on Skyler's treatment for any interested parties wondering how Soliris is working for a factor H atypical HUS child. We'll keep you posted on Skyler's lab values here as well as any advancements or treatment glitches - stay tuned....

Phyllis Ann Talbot said:
Hi there - we are also watching all developments with Soliris. Currently we are on PD and doing well. We don't qualify for the first round of studies on Soliris both b/c of age and also the fact that we are on dialysis. So Hyde would need to do Soliris in conjunction with a kidney transplant.

We most likely won't be able to wait long enough to be in an actual trial - and have discussed requesting a compassionate waiver with our dr's - but while he's stable - we are going to wait to see how much other information comes out before pursuing other options.
Latest Labs Update

8th dose of Soliris: Coen's latest labs (as of Tues) continued to stay stable. PLT 384. RBC 12.5. Creatinine 1.5. Protein and albumin levels show continued improvement. Plan is for Coen to continue to receive Soliris every 2 weeks for now. There is clearly a huge difference in how Coen is feeling. More energy, strength, and endurance than he has had ever had previously.

Jodi Kayler said:
Coen has been receiving Soliris since April 22. For all those who love labs, I’ll try to give a rundown of lab values since the start of Soliris. I may have dates off by a day or two…let me know if you have questions
At the start of Soliris, Coen was hospitalized and getting daily pheresis, IVIG twice a week, hemo dialysis and getting RBC transfusions as needed. Once Soliris is started pheresis and dialysis were stopped completely. Soliris protocol is identical to Skyler's.
• April 22: First dose of Soliris: Platelets 220. Very high BP. Creatinine 3.0.
• Day 2: Platelets 250. Starts feeling a little better. No pheresis. No dialysis.
• Day 3: Platelets 260. Still shows schistocytes. Urine output starts to improve. Lots of energy. Feeling good.
• Day 4: Platelets 281. RBC increases. Creatinine 2.8. Urine output continues to improve but there is unexpected whole red blood cells in urine. No schistocytes.
• Day 5: PLT 254. Creatinine decreases. BP coming down. Feeling great. No more red cells in urine. Electrolytes start becoming stable. Ins & Outs match.
• April 29: 2nd dose of Soliris. No dialysis or pheresis for 1 week. PLT 232. Kidney function stable. Creatinine 2.4. Dialysis catheter removed. Released from hospital.
• May 5: 3rd dose of Soliris. PLT 261 Creatinine 2.1. Back in school and feeling great.
• May 12: 4th dose of Soliris PLT 330 (highest ever). Creatinine 1.9. BP good.
• May 19: PLTs down to 190. Creatinine 1.5. Feeling great. No signs of schistocytes. No explainable reason for PLT drop.
• June 2: PLT 384 (another record high) Creatinine 1.6. HCT 35.0. No pheresis or dialysis since April. BP meds have been reduced.

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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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