title: Effective eculizumab therapy followed by BMT in a boy with paroxysmal nocturnal hemoglobinuria.
abstract: 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.
authors: Department of Pediatrics, School of Medicine, University of Yamanashi, Yamanashi, Japan.
authors: Hiroko
authors: H
authors: Oshiro
authors: Kumiko
authors: K
authors: Goi
authors: Koshi
authors: K
authors: Akahane
authors: Takeshi
authors: T
authors: Inukai
authors: Kanji
authors: K
authors: Sugita
mesh_terms: D000741:Anemia, Aplastic / Q000150:complications / Q000628:therapy*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D003131:Combined Modality Therapy; D051056:Complement Inactivating Agents / Q000627:therapeutic use*; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000628:therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use; D008297:Male
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D051056:Complement Inactivating Agents; D007166:Immunosuppressive Agents; C481642:eculizumab
keywords: bone marrow transplantation; eculizumab; paroxysmal nocturnal hemoglobinuria