Alexion Pharmaceuticals (Nasdaq: ALXN) today announced the presentation of data that underscore the critical need for testing patients at high risk for paroxysmal nocturnal hemoglobinuria (PNH) and new data on the potentially life-threatening, systemic complications in patients with atypical hemolytic uremic syndrome (aHUS). A retrospective analysis of 30 pediatric and adult patients with aHUS showed that the majority of patients experienced severe complications of the disease across multiple organs, despite receiving supportive care, including plasma exchange or plasma infusion. These data underscore the risk for sudden and potentially fatal systemic complications of aHUS.
In a separate analysis of 19 pediatric patients with aHUS, Soliris
(eculizumab) significantly reduced thrombotic microangiopathy, or TMA (the formation of blood clots in small blood vessels throughout the body), leading to improved kidney function and eliminating the need for dialysis in half of patients who previously required it before starting on Soliris therapy.
These data were presented at the 17th Congress of the European Hematology Association (EHA), being held in Amsterdam on June 14-17.
“The research presented at EHA confirms the significant and potentially fatal risks of both PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation, and the urgent need for an accurate diagnosis and rapid treatment in these patients,” said Leonard Bell, M.D., Chief Executive Officer of Alexion.
Systemic Multi-Organ Complications Common in Patients with aHUS
In a poster session on Friday, June 15, researchers presented retrospective data from an analysis of 30 pediatric and adult patients with aHUS prior to receiving Soliris. All 30 patients (100%) showed evidence of kidney impairment. Extra-renal, systemic organ complications were reported in 63% of patients (19 of 30), and extra-renal thrombi were reported in 37% of patients (11 of 30).
These complications spanned the cardiovascular (47% of patients), gastrointestinal (37%) and neurological (20%) systems, and were similarly reported in aHUS patients with or without identified genetic complement mutation.
“While aHUS often has a devastating impact on the kidneys, the TMA process that defines aHUS can also cause progressive and sudden damage across multiple organs, including heart attack, stroke, pancreatitis, deep vein thrombosis and seizures,” said Craig B. Langman, M.D., the Isaac A Abt MD Professor of Kidney Diseases, Head of Kidney Diseases, Feinberg School of Medicine, Northwestern University. “Evidence of TMA or progressive systemic organ involvement should prompt high suspicion of aHUS as a clinical diagnosis, even in the absence of kidney failure.”