Resumen de Eight years of experience with eculizumab in a case of paroxysmal nocturnal hemoglobinuria
Yared González Pérez, Jesica Nuñez Rodríguez, Rebeca Apiñaniz Apiñaniz, María José Nebot Villacampa, María Eugenia Calvo Aragüete, Alicia Caso González, Raquel Marin Gorricho, Andrea Casajús Navasal
Paroxysmal nocturnal hemoglobinuria is a rare clonal disease of hematopoietic stem cells. These hematopoietic cells present a deficit of surface membrane anchorage proteins such as DAF (Complement Degradation Accelerator Factor, CD55) and MIRL (Membrane Reactive Lysis Inhibitor, CD59). This total or partial absence produces a greater susceptibility of erythrocytes to complement-mediated hemolysis. Eculizumab is a monoclonal antibody indicated in the paroxysmal nocturnal hemoglobinuria, capable of blocking the complement protein C5 avoiding its activation and, therefore, hemolysis. We present the case of a woman diagnosed with paroxysmal nocturnal hemoglobinuria resistant to the use of systemic corticosteroids with a poor quality of life, an important amount of symptoms and hospitalizations in several occasions. After eight years in treatment with eculizumab the patient presents a good drug tolerance, a better quality of life and a decrease in the value of lactate dehydrogenase, stabilization of the value of hemoglobin and a reduction on the number of units of packed red cells transfused
