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Leeds University study shows eculizumab may be an effective therapy for PNH
Sep 21, 2006, 20:23, Reviewed by: Dr. Rashmi Yadav
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"These study results suggest that eculizumab has great potential in providing patients diagnosed with PNH with an effective therapy."
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By Euro RSCG Life NRP ,
A study led by Dr Peter Hillmen of the Leeds Teaching Hospitals NHS Trust, relating to an uncommon and severe haemolytic anaemia known as paroxysmal nocturnal haemoglobinuria (PNH), was published in the current issue of The New England Journal of Medicine. In the Phase III efficacy study, TRIUMPH, 87 patients were treated at 34 sites in the U.S., Canada, Europe, and Australia. The data showed clinically significant improvements in anaemia and the quality of life for patients with PNH.
The study was a double-blind, randomized, placebo-controlled trial that tested the safety and efficacy of eculizumab, a humanized monoclonal antibody against complement protein C5, that inhibits terminal complement activation. Patients received either placebo or eculizumab intravenously. Eculizumab treatment significantly improved anaemia in patients as both primary endpoints were achieved, including median transfusion rate and haemoglobin stabilization over six months. The median transfusion rate was reduced from 10 units per patient with placebo to 0 units per patient with eculizumab (p<0<0 
- The results are published in the current issue of The New England Journal of Medicine
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