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- aggregation classification "A1".
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- aggregation date "2012".
- aggregation format "application/pdf".
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- aggregation isPartOf urn:issn:1077-4114.
- aggregation language "eng".
- aggregation rights "I have transferred the copyright for this publication to the publisher".
- aggregation subject "Medicine and Health Sciences".
- aggregation title "Successful use of eculizumab in a pediatric patient treated for paroxysmal nocturnal hemoglobinuria".
- aggregation abstract "Paroxysmal 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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- aggregation issue "8".
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- aggregation volume "34".
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