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abstractpubmed· Abstract 2015· item PMID:25868955

Effective eculizumab therapy followed by BMT in a boy with paroxysmal nocturnal hemoglobinuria. A 9-year-old boy with paroxysmal nocturnal hemoglobinuria/aplastic anemia syndrome (PNH/AA) developed hemolytic crisis after receiving immunosuppressive therapy. Eculizumab dramatically relieved the signs and symptoms and then he safely underwent unrelated bone marrow transplantation, suggesting the feasibility and effectiveness of eculizumab before stem cell transplantation in children with PNH/AA in hemolytic crisis.