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

Authors

  • 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

DOI:

https://doi.org/10.3823/1754

Keywords:

paroxysmal nocturnal hemoglobinuria, eculizumab, complement system

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.

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

References

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Published

2015-07-07

Issue

Section

Internal Medicine & Hospital Medicine

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