[Paroxysmal nocturnal hemoglobinuria therapy with eculizumab: Spanish experience]. / Tratamiento de la hemoglobinuria paroxística nocturna con eculizumab: experiencia en España.

ABSTRACT

BACKGROUND AND OBJECTIVES: 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. MATERIAL AND 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.