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Eculizumab reduces Paroxysmal nocturnal hemoglobinuria complications and improves survival

First long term study showing the clinical benefits after 8 years of continued treatment

July 7th 2011

Hemolysis (Image from www.alexionpharm.com/)

Allogenic bone marrow transplantation is the only curative therapy for Paroxysmal nocturnal hemoglobinuria (PNH). Now, an article published recently in the Blood magazine shows that eculizumab, a monoclonal antibody, dramatically alters the natural course of PNH. The new therapeutical approach reduces symptoms and disease complications and improves survival to a similar level to that of the general population.

PHN is an acquired clonal hematopoietic disorder caused by a mutation in the PIGA gene, which takes part in the production of an important protein found on the membrane of blood cells. Without this protein, the GPI anchor, is altered the cell membranes cannot anchor two important proteins for the regulation of the immune system. As a result they are destroyed by the immunological machinery supposed to protect them.

The premature destruction of red blood cells results in hemolytic anemia, which can cause signs and symptoms such as fatigue, weakness, abnormally pale skin, shortness of breath, and an increased heart rate. People with PHN may also be prone to infections due to a deficiency of white blood cells or suffer problems in the blood clotting process due to abnormal platelets. The risk of developing cancer in blood-forming cells such as leukaemia is also increased in these patients.

A work conducted by researchers from the Leeds Teaching Hospitals (UK) is the first long term study showing the clinical benefits after 8 years of continued treatment with eculizumab. The results were recently published in the Blood journal, with Dr. RJ Kelly as its first author and Dr. P. Hillmen as the last one. They evaluated 79 consecutive patients treated with eculizumab in Leeds between May 2002 and July 2010. The survival of patients treated with eculizumab was not different from age- and sex-matched normal controls but was significantly better than 30 similar patients managed before eculizumab.

The new treatment, commercialized by Alexion Pharmaceuticals, also reduced the thrombosis from 5.6 events per 100 patient-years before starting eculizumab to 0.8 events per 100 patient-years on eculizumab. It also reduced in a 74% the transfusion requirement, and up to 66% of the patients on eculizumab for more than 12 months achieved transfusion independence. In summary, eculizumab dramatically alters the natural course of PNH, reducing symptoms and disease complications as well as improving survival to a similar level to that of the general population.

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