Uso de Eculizumab en pacientes adultos con hemoglobinuria paroxística nocturna: Revisión sistemática

Abstract

Introduction: Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare disease associated with high morbidity and mortality. The introduction of Eculizumab has improved patient prognosis by reducing hemolysis and the risk of thrombosis; however, this treatment is not curative, requires regular infusions, and may cause adverse effects. Objective: To evaluate the use of Eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria. Methodology: Studies in English published between 2019 and 2024 were included, excluding reviews and animal studies. A structured search strategy was employed in the Web of Science, Scopus, and PubMed databases. Article selection was conducted using Rayyan, and the risk of bias was evaluated using the GRADEpro tool. Results: A total of 66.1% of patients treated with Eculizumab avoided transfusions, and a 49.4% decrease in LDH levels, a key marker of hemolysis, was observed. Additionally, 23.4% of patients achieved a complete hematologic response after 12 months of treatment. Discussion: The use of Eculizumab significantly improves quality of life and reduces thromboembolic events in patients with PNH. Nonetheless, limitations persist, such as recurrent hemolysis and fatigue. Conclusions: Eculizumab reduces the need for transfusions and helps normalize LDH levels, which contributes to reducing fatigue and improving overall patient well-being. Regarding adverse effects, the most notable include headache, an increased risk of infections, dyspnea, and nausea, among others