12:40AM Alexion Pharma presents data show majority of patients with aHUS experienced systemic multi-organ complications prior to treatment (ALXN) 93.31 : Co announces the presentation of data that underscore the critical need for testing patients at high risk for paroxysmal nocturnal hemoglobinuria and new data on the potentially life-threatening, systemic complications in patients with atypical hemolytic uremic syndrome. 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.1 In a separate analysis of 19 pediatric patients with aHUS, Soliris significantly reduced thrombotic microangiopathy, or TMA, leading to improved kidney function and eliminating the need for dialysis in half of patients who previously required it before starting on Soliris therapy. 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).1 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.
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