WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals, Inc. (ALXN), Saturday said patients with paroxysmal nocturnal hemoglobinuria or PNH who have late-stage renal impairment had a significantly worse overall survival rate compared with PNH patients without renal impairment.
Alexion presented the data this weekend at the 16th Congress of the European Hematology Association or EHA in London, which included studies on mortality in PNH patients with renal impairment and long-term outcomes with Soliris (eculizumab) therapy.
PNH is an ultra-rare, life-threatening blood disorder in which uncontrolled activation of the complement system causes the chronic destruction of red blood cells (hemolysis). Soliris, a first-in-class terminal complement inhibitor, has been approved for the treatment of patients with PNH.
In 301 patients enrolled in the National Data Registry in South Korea, 16 percent of patients had a history or presence of late-stage renal impairment, and these patients accounted for 35 percent of patient deaths in the registry.
Jin Seok Kim, Division of Hematology, Severance Hospital, Yonsei University College of Medicine, Seoul, Korea. 'This study confirms the connection between renal impairment and PNH, and provides a strong rationale for the effective treatment of hemolysis, the underlying cause of organ damage in patients with PNH.'
In another presentation of long-term outcomes, Alexion reported long-term data from all 195 patients who participated in the Soliris PNH clinical trials and extension studies. Overall survival with Soliris therapy was 97.6 percent at three years and was maintained through 5.5 years.
Alexion said the results were consistent with data published in the journal Blood earlier this year.
The current study also showed a reduction of thromboembolic events (TE) from 52 pre-treatment events to 10 events during Soliris therapy. These results were previously presented at the 52nd American Society of Hematology Annual Meeting.
In another study presented at EHA, investigators showed that patients with no history of transfusion demonstrated substantial disease burden despite their transfusion status.
In two patients with no history of transfusion, Alexion reported evidence of significant clinical disease burden at baseline, including chronic hemolysis and renal disease. In both patients, Soliris treatment resulted in substantial reductions in LDH (a measure of hemolysis), clinically meaningful improvement in fatigue and quality of life, and reduction of CKD.
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