• J. Pediatr. Hematol. Oncol. · Nov 2012

    Successful use of eculizumab in a pediatric patient treated for paroxysmal nocturnal hemoglobinuria.

    • Tiene Bauters, Victoria Bordon, Hugo Robays, Yves Benoit, and Catharina Dhooge.
    • Department of Pharmacy, Ghent University Hospital, Ghent, Belgium. tiene.bauters@uzgent.be
    • J. Pediatr. Hematol. Oncol. 2012 Nov 1; 34 (8): e346-8.

    AbstractParoxysmal nocturnal hemoglobinuria (PNH) is a rare, debilitating life-threatening clonal hematopoietic stem cell disease. The clinical manifestations of PNH are usually seen in adulthood and are very rarely reported in children. Eculizumab, a humanized monoclonal antibody targeting and preventing cleavage of the terminal complement protein C5, has become the "gold standard" of treatment for hemolysis or significant disease-related complications in patients with PNH. Although eculizumab is not licensed for use in pediatrics, we report a young PNH patient with bone marrow failure and severe episodes of hemolytic anemia who was treated successfully with eculizumab for >18 months.

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