Paroxysmal Nocturnal Hemoglobinuria: Report of Suboptimal Response to the Use of Eculizumab
Keywords:paroxysmal nocturnal hemoglobinuria, eculizumab, complement system
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, CearÃ¡, 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.
Luzzato, L, Notaro R. Paroxysmal Nocturnal Haemoglobinuria. In: Principles and practice of hematology. Handin, R.I.; Lux, S.E.; Stosser, T.P. 2 ed, Lippincott Williams 8 Wilkins, 2003-2004. Chapter 10, p. 328-32.
De Latour RP, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008; 112(8): 3099-3106
Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013; 121(25): 4985 â€“ 4996
Arruda MMA, et al. HemoglobinÃºria paroxÃstica noturna: da fisiopatologia ao tratamento. Rev Assoc Med Bras. 2010; 56: 214-21
Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014; 124(18): 2804 â€“ 281
Parker C, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinÃºria. Blood. 2005; 106(12): 3699 â€“ 3709
Brodsky RA. How I treat paroxysmal nocturnal hemoglobinÃºria. Blood. 2009; 113(26): 6522 â€“ 6527
Mahmood A, Sharif MA, Murtaza B. . Paroxysmal Nocturnal Hemoglobinuria. Journal of the Collage of Physicians and Surgeons Pakistan. 2008; 18(8): 512-14
Modesto TM, et al. ImportÃ¢ncia e vantagem da citometria de fluxo frente aos testes de triagem no diagnostico da HemoglobinÃºria ParoxÃstica Noturna. Rev. Bras. Hematol. Hemoter. 2006; 28(4) 275-279
Nomura ML, et al. HemoglobinÃºria paroxÃstica noturna e gravidez. . RBGO. 2004; 26(7):579-82
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