title: Successful use of eculizumab in a pediatric patient treated for paroxysmal nocturnal hemoglobinuria.
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.
authors: Department of Pharmacy, Ghent University Hospital, Ghent, Belgium. tiene.bauters@uzgent.be
authors: Tiene
authors: T
authors: Bauters
authors: Victoria
authors: V
authors: Bordon
authors: Hugo
authors: H
authors: Robays
authors: Yves
authors: Y
authors: Benoit
authors: Catharina
authors: C
authors: Dhooge
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000741:Anemia, Aplastic / Q000209:etiology; D000743:Anemia, Hemolytic / Q000209:etiology; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D017707:Erythrocyte Transfusion; D005260:Female; D005492:Folic Acid / Q000627:therapeutic use; D016298:Hematopoietic Cell Growth Factors / Q000627:therapeutic use; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D016130:Immunophenotyping; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D013601:T-Lymphocytes
chemical_list: D000305:Adrenal Cortex Hormones; D061067:Antibodies, Monoclonal, Humanized; D000961:Antilymphocyte Serum; D016298:Hematopoietic Cell Growth Factors; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D005492:Folic Acid; C481642:eculizumab