Eight years of experience with eculizumab in a case of paroxysmal nocturnal hemoglobinuria

• Autores: 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
• Localización: European journal of clinical pharmacy: atención farmacéutica, ISSN 2385-409X, Vol. 21, Nº. 3, 2019, págs. 164-166
• Idioma: inglés
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• Resumen

  • 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