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| |  Eculizumab Effective in Paroxysmal Nocturnal Haemoglobinuria: Presented at EHA By Jill Stein VIENNA, AUSTRIA -- June 12, 2007 -- The terminal complement inhibitor eculizumab (SolirisTM) reduces haemolysis and transfusion requirements in a heterogeneous population of patients with paroxysmal nocturnal haemoglobinuria (PNH), according to data presented here at the 12th Congress of the European Hematology Association (EHA). Hubert Schrezenmeier, MD, director, Institute of Clinical Transfusion Medicine and Immunogenetics, University Hospital, Ulm, Germany, reported the results of an open-label, non–placebo-controlled, phase 3 trial in 97 patients who were treated with intravenous eculizumab. Eculizumab was given at a dose of 600 mg weekly for 4 weeks followed a week later by a 900-mg dose and then 900 mg every other week for a total of 52 weeks. The study cohort included a broad population of patients with PNH, including patients with severe thrombocytopenia and/or minimal transfusion requirements. The primary outcome measure was a reduction in haemolysis as measured by the surrogate endpoint of lactate dehydrogenase (LDH). Results showed that LDH decreased by 87% from a median level of 2,051 IU/L at baseline to 269 IU/L at 52 weeks (P <.001). The normal LDH range is 103 to 223 IU/L. "This finding is important because it means that treatment is associated with a return to normal or near normal red blood cell survival," Dr. Schrezenmeier noted in his presentation on June 9th. Control of haemolysis resulted in an improvement in anaemia as evidenced by a significant decrease in transfusions from a median of 8 packed red blood cell units per patient during the 12 months prior to treatment to 0 units per patient during the 12-month treatment period (P <.001). The investigators also documented a significant improvement in fatigue and global health status using widely validated quality-of-life instruments. Also, 51% of patients became transfusion-independent during the entire 12-month treatment period (P <.001). Most adverse events were mild or moderate and not considered to result from eculizumab treatment, according to the researchers. Eculizumab is the first compound that is specifically approved by the US Food and Drug Administration for the treatment of PNH. Current unapproved treatments do not address red blood cell haemolysis, the underlying cause of PNH. Such treatments involve routine blood transfusions, nonspecific immunosuppressive therapy, and, rarely, bone marrow transplant. Alexion Pharmaceuticals, Inc. provided funding for this study, which was conducted at 33 sites in the United States, Canada, Europe, and Australia.
[Presentation title: Safety and Efficacy of the Terminal Complement Inhibitor Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria: SHEPHERD Phase III Clinical Study Results. Abstract 378]
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