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The New England Journal of Medicine reports data on eculizumab for the treatment of PNH

September 21, 2006

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<0.001). Haemoglobin stabilization was achieved by 49% of eculizumab patients as compared to 0% for placebo (p<0.001). Patients treated with eculizumab also experienced significantly less intravascular hemolysis, fatigue, pain, and shortness of breath, together with improvements in overall health status and functioning.

"PNH is a rare and life-threatening form of haemolytic anaemia where currently there are limited treatment options," said Dr. Peter Hillmen, senior author of the study, lead investigator and chairman of the TRIUMPH steering committee and Consultant Hematologist of the General Infirmary at the University of Leeds, Leeds, UK. "These study results suggest that eculizumab has great potential in providing patients diagnosed with PNH with an effective therapy."

Dr Hillmen also noted, "Patients with PNH have chronic destruction of red blood cells causing both anaemia and the release of free haemoglobin. The need for transfusion is common in patients in order to sustain tolerable levels of haemoglobin and to maintain a certain quality of life. With this study involving eculizumab, we saw improvements in the quality of life and stabilization of haemoglobin levels, and a majority of patients became transfusion independent."

PNH is characterized by severe destruction of red blood cells by the body's complement system (a component of the immune system). Patients with PNH may also experience anaemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark coloured urine, known as haemoglobinuria. Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are the major cause of death. Dr. Hillmen has previously published findings that describe a prevalence in North and East Yorkshire, Northern Lincolnshire and West Yorkshire of approximately 13 people have detectable PNH per million population. Based on that data, it is estimated that there may be approximately 5,000 - 6,000 people in Europe and 3,000 - 5,000 people in North America with PNH.

Study results show eculizumab may be an effective therapy for PNH. The most common adverse events reported in the eculizumab group were headache, nasopharyngitis, back pain, and nausea.

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