New Drugs: Eculizumab

    August 2009 in “ Australian Prescriber ”
    Image of study
    TLDR Eculizumab helps reduce blood transfusions and stabilize hemoglobin but increases infection risk and is expensive.
    Eculizumab was a humanized monoclonal antibody used to treat paroxysmal nocturnal hemoglobinuria by binding to complement protein C5, thereby reducing hemolysis and stabilizing hemoglobin levels. In a study with 87 patients, 49% of those treated with eculizumab had stabilized hemoglobin levels, and 51% did not require blood transfusions, unlike the placebo group, where all needed transfusions. An open-label study with 97 patients showed suppressed hemolytic activity in 89 patients, reducing transfusion needs. However, eculizumab increased susceptibility to meningococcal infections, requiring vaccination before treatment. While it reduced thromboembolism rates, the long-term outcomes and effects on survival were uncertain, and the high cost of the drug was a concern.
    Discuss this study in the Community →