NISHIMURA Junichi, YAMAMOTO Masaki, KANAKURA Yuzuru ≪Hematology and Oncology≫ “Genetic Variants in C5 and Poor Response to Eculizumab ”

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PublishThe New England Journal of Medicine(2014) doi:10.1056/NEJMoa1311084

Eculizumab is a humanized monoclonal antibody that targets complement
protein C5 and inhibits terminal complement-mediated hemolysis associated with PNH. We identified a C5 polymorphism (c.2654G→A)
in Japanese patients with PNH prevents binding and blockade by eculizumab, while retaining the functional capacity of the mutant C5 to cause hemolysis.
Therefore, the polymorphism in the target protein might be important to consider in patients with a poor
response to the antibody-based treatments for various diseases.