Paroxysmal Nocturnal Hemoglobinuria: Report of Suboptimal Response to the Use of Eculizumab

Abstract

Introduction: The Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal disorder of one or more stem cells of hematopoietic lineage. It is characterized as a chronic hemolytic anemia in which there is a persistent intravascular hemolysis which is subject to exacerbations due to production of subpopulations of erythrocytes, platelets and granulocytes hypersensitive to complement. Furthermore there has been a high tendency to thrombosis. The anti-complement therapy is the only recognized treatment for this condition. We report a case of PNH with suboptimal response to anti-complement therapy. Method: This is one case report, obtained through data from medical records of a reference hospital, located in the city of Barbalha, Ceará, Brazil. Case report: Patient with PNH, with typical clinical features, however nonspecific from the onset of the clinical picture, especially marked by signs and symptoms of anemic syndrome (asthenia, lethargy palpitations, paleness). Showed present hemolysis markers and negative Coombs test. The flow cytometry closed the diagnosis of paroxysmal nocturnal hemoglobinuria and there was the initiation of the anti-complement therapy using the drug called eculizumab. After one year of treatment, the patient persists with anemia and sporadic episodes of hemoglobinuria, as well as present hemolysis markers. However, she remains free of thromboembolic events and without need for transfusion. Conclusion: The PNH is a rare pathology with high morbidity and mortality if not treated. The anti-complement therapy with eculizumab offers great chance of long-term control of the most harmful consequences of the disease.