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Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria
Author(s) -
Reiss Ulrike M.,
Schwartz Jeffrey,
Sakamoto Kathleen M.,
Puthenveetil Geetha,
Ogawa Masayo,
Bedrosian Camille L.,
Ware Russell E.
Publication year - 2014
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.25068
Subject(s) - eculizumab , medicine , paroxysmal nocturnal hemoglobinuria , hemoglobinuria , adverse effect , anemia , pediatrics , gastroenterology , immunology , antibody , complement system
Paroxysmal nocturnal hemoglobinuria (PNH) is rare in children, but represents a similarly serious and chronic condition as in adults. Children with PNH frequently experience complications of chronic hemolysis, recurrent thrombosis, marrow failure, serious infections, abdominal pain, chronic fatigue, and decreased quality of life with reduced survival. The terminal complement inhibitor eculizumab is proven to be effective and safe in adults and approved by the FDA for treatment of PNH.