title: [Paroxysmal nocturnal hemoglobinuria therapy with eculizumab: Spanish experience].
abstract: OBJECTIVE
  Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal disease characterized by complement-mediated hemolysis, bone marrow failure and thrombosis. Eculizumab is a humanized monoclonal antibody that blocks the cytolytic component of the complement system by binding to complement C5.
  
  
  METHODS
  We report the results of eculizumab treatment in 25 PNH patients from different centers in Spain. Statistical analysis was perfomed with a SPSS v15.0 software.
  
  
  RESULTS
  Fifty-eight per cent of the patients achieved transfusional independence after a median of 14 months. Transfusion requirements were reduced in 60% of the remaining cases. Fatigue resolved in 96% of the patients and smooth muscle dystony-related symptoms in all cases. A single case of treatment-related infection was observed.
  
  
  CONCLUSIONS
  Eculizumab controls effectively hemolysis and greatly improves clinical symptoms. The drug is safe and well tolerated, without significant adverse effects except meningococcal infection. Patients with suboptimal response to treatment must be assessed for bone marrow insufficiency and extravascular haemolysis.
authors.affiliation: Servicio de Hematología y Hemoterapia, Hospital Universitario Príncipe de Asturias, Madrid, España. montserratlr@terra.es
authors.forename: Montserrat
authors.initials: M
authors.lastname: López Rubio
authors.forename: Marta
authors.initials: M
authors.lastname: Morado
authors.forename: Ana
authors.initials: A
authors.lastname: Gaya
authors.forename: Dora
authors.initials: D
authors.lastname: Alonso Rosa
authors.forename: Emilio
authors.initials: E
authors.lastname: Ojeda
authors.forename: Juan Antonio
authors.initials: JA
authors.lastname: Muñoz
authors.forename: Begoña
authors.initials: B
authors.lastname: Pérez de Mendiguren
authors.forename: María Dolores
authors.initials: MD
authors.lastname: Monteagudo
authors.forename: José María
authors.initials: JM
authors.lastname: Durán
authors.forename: Rosa María
authors.initials: RM
authors.lastname: Fisac
authors.forename: Damián
authors.initials: D
authors.lastname: Moreno
authors.forename: Ana María
authors.initials: AM
authors.lastname: Villegas
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D013030:Spain; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab