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


  • Ricardo Parente Garcia Vieira Faculdade de Medicina do ABC Hospital Regional do Cariri
  • Lucas Parente Alencar Hospital Regional do Cariri- Juazeiro do Norte/Ce-Brazil
  • George Nilton Nunes Mendes
  • Natália Parente Alencar
  • Francisco Rômulo Patrício de Sá
  • Hermes Melo Teixeira Batista
  • Jully Graziela Coelho Campos Couto
  • José Lucas Souza Ramos
  • Thiáskara Ramile Caldas Leite
  • Italla Maria Pinheiro Bezerra
  • Luiz Carlos de Abreu



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, 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.

Author Biographies

Ricardo Parente Garcia Vieira, Faculdade de Medicina do ABC Hospital Regional do Cariri

Laboratory of design study and  scientific writing of the FMABC

Lucas Parente Alencar, Hospital Regional do Cariri- Juazeiro do Norte/Ce-Brazil

Department of internal medicine


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 – 4996

Arruda MMA, et al. Hemoglobinúria paroxística noturna: da fisiopatologia ao tratamento. Rev Assoc Med Bras. 2010; 56: 214-21

Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014; 124(18): 2804 – 281

Parker C, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinúria. Blood. 2005; 106(12): 3699 – 3709

Brodsky RA. How I treat paroxysmal nocturnal hemoglobinúria. Blood. 2009; 113(26): 6522 – 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. Importância e vantagem da citometria de fluxo frente aos testes de triagem no diagnostico da Hemoglobinúria Paroxística Noturna. Rev. Bras. Hematol. Hemoter. 2006; 28(4) 275-279

Nomura ML, et al. Hemoglobinúria paroxística noturna e gravidez. . RBGO. 2004; 26(7):579-82






Internal Medicine & Hospital Medicine

Most read articles by the same author(s)

1 2 3 4 5 > >>