The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria.

  title={The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria.},
  author={Peter Hillmen and Nam Sang Young and Joerg Schubert and Robert A Brodsky and G{\'e}rard Soci{\'e} and Petra Muus and Alexander R{\"o}th and Jeffrey Szer and Modupe O. Elebute and Ryotaro Nakamura and Paul Leduc Browne and Antonio M Risitano and Anita V. Hill and Hubert Schrezenmeier and Chieh-Lin Fu and Jaroslaw Maciejewski and Scott A. Rollins and Christopher F. Mojcik and Russell P. Rother and Lucio Luzzatto},
  journal={The New England journal of medicine},
  volume={355 12},
BACKGROUND We tested the safety and efficacy of eculizumab, a humanized monoclonal antibody against terminal complement protein C5 that inhibits terminal complement activation, in patients with paroxysmal nocturnal hemoglobinuria (PNH). METHODS We conducted a double-blind, randomized, placebo-controlled, multicenter, phase 3 trial. Patients received either placebo or eculizumab intravenously; eculizumab was given at a dose of 600 mg weekly for 4 weeks, followed 1 week later by a 900-mg dose… CONTINUE READING
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