-- Global
Many health conditions that damage kidney function can severely affect a patient's quality of life and progress to end-stage renal disease (ESRD) and premature death. One such disease is atypical hemolytic uremic syndrome (aHUS), a chronic, life-threatening ultra-rare disease that can progressively damage vital organs, including the kidney. Patients with aHUS experience kidney damage and kidney failure leading to ESRD.1 Sixty-five percent of patients with aHUS require kidney dialysis, have permanent kidney damage or die within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI).2,3
"At the age of 28, I was experiencing renal failure but my doctors were
not able to pinpoint the cause of it," says
"We know first-hand the importance of the disease awareness efforts
taking part on
In addition to its work in aHUS, Alexion is conducting investigational studies in kidney transplant patients who are at elevated risk for antibody mediated rejection, or other kidney transplant patients who are at elevated risk for delayed graft function.
For more information about
About aHUS
aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.4,5 Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.4,6 Sixty-five percent of all patients with aHUS require kidney dialysis, have permanent kidney damage or die within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI).2,3 The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate in these TMA patients.7
aHUS affects both children and adults.8 Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.8
About Alexion
[ALXN-G]
References
1. |
Noris M, Bresin E, Mele C, et al. Atypical Hemolytic-Uremic
Syndrome. 2007 Nov 16 [Updated 2011 Mar 10]. In: Pagon RA, Bird
TD, Dolan CR, et al., editors. GeneReviews™ [Internet]. |
|
2. |
Caprioli J, Noris M, Brioschi S, et al. The impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006;108:1267-1269. |
|
3. |
Loirat C, Garnier A, Sellier-Leclerc AL, Kwon T. Plasmatherapy in atypical hemolytic uremic syndrome. Semin Thromb Hemost. 2010;36:673-81. |
|
4. |
Definition from the |
|
5. |
Benz K, |
|
6. |
Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-96. |
|
7. |
Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23. |
|
8. |
Bresin E, Daina E, Noris M, et al. Outcome of renal
transplantation in patients with non- |
|
Executive
Director, Corporate Communications
or
Director, Corporate Communications
Source:
News Provided by Acquire Media