Rare Disease Report

New Data Shows Soliris Provides Reduction in Post-Transplant Dialysis in aHUS Patients

NOVEMBER 22, 2016
Andrew Black
New data on Alexion’s Soliris presented at the 2016 annual meeting of the American Society of Nephrology (ASN) in Chicago shows encouraging results for post-transplant dialysis in atypical Hemolytic Uremic Syndrome (aHUS) patients.

About the study

Researcher’s goal of the study was to evaluate the timing of Soliris treatment and the need for dialysis in patients diagnosed with aHUS.
 
147 Patients enrolled in the Global aHUS Registry who have had a kidney transplant and were treated with Soliris were evaluated by the rate of dialysis. Researchers studied and compared all patients who started on Soliris therapy before or at the time of the transplant (n=69, median age 34 at current transplant, including patients receiving non-labeled dosing) to those who started on Soliris after the transplant (n=78, median age 35).
 
The researchers also performed a sub-analysis on the patients who continued to receive labeled dosing of Soliris initiated prior to transplant (n=53) compared to those who initiated and continued to receive labeled dosing of Soliris after the transplant (n=65).

Results

6 patients out of 69 who were on Soliris therapy before transplant required dialysis vs 28 out of 78 who initiated Soliris treatment post-transplant. Patients who started Soliris post-transplant saw an increase in need of dialysis.

Sub-analysis results

4 out of 53 patients receiving labeled dosing, who started Soliris therapy before transplant required dialysis, vs 23 out of 65 patients who initiated Soliris treatment post-transplant, indicating a nearly three-fold increase in likelihood of dialysis in the patients initiating Soliris treatment post-transplant.
 
In both analysis, there was an increase of dialysis in patients initiating Soliris treatment post-transplant rather than those who started treatment prior.

About aHUS

Atypical hemolytic-uremic syndrome (aHUS) is a systemic disease that primarily affects the kidneys. The rare condition is associated with genetic mutations or polymorphisms that result in chronic, uncontrolled activation of the alternative complement pathway. The dysregulated alternative complement pathway activation causes blood clot (thrombi) formation in the glomerular capillaries (groups of small blood vessels of the kidneys) and thickening of the glomerular capillary walls. 

Symptoms of aHUS most often include:
  • Nausea and vomiting.
  • Confusion.
  • shortness of breath (dyspnea)
  • Fatigue.
  • Anemia (low red blood cell/platelet count in the blood)
  • Thrombocytopenia (low platelet count in the blood)
 
 


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