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Effective eculizumab therapy followed by BMT in a boy with paroxysmal nocturnal hemoglobinuria
Author(s) -
Oshiro Hiroko,
Goi Kumiko,
Akahane Koshi,
Inukai Takeshi,
Sugita Kanji
Publication year - 2015
Publication title -
pediatrics international
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.49
H-Index - 63
eISSN - 1442-200X
pISSN - 1328-8067
DOI - 10.1111/ped.12522
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , eculizumab , hemoglobinuria , immunology , pediatrics , antibody , hemolysis , complement system
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.