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>Journals >Revista de Hematología >Year 2015, Issue 1


Góngora-Biachi RA, González-Martínez P, Ceballos-López AA, Rivas-Llamas JR, Rico-Curiel E, Aquino-Salgado JL, López-Karpovitch X, López-Hernández M, Alvarado-Ibarra M, Trejo-Gómora JE, Vélez-Ruelas MA, Oropeza-Martínez MP, Selva-Pallares JE, León-González MG
Mexican consensus on the treatment of the paroxysmal nocturnal hemoglobinuria
Rev Hematol Mex 2015; 16 (1)

Language: Español
References: 66
Page: 70-96
PDF: 552.65 Kb.


Full text




ABSTRACT

Paroxysmal nocturnal hemoglobinuria (PNH) is a consequence of clonal expansion of one or several hematopoietic stem cells that have a somatic mutation in the PIG-A gene, presenting impaired synthesis of the glycosyl-phosphatidylinositol (GPI), and in turn, complete or partial deficiency of CD59 and CD55. These deficiencies produce a higher sensitivity of the PNH cells to the complement biological effect (C). PNH clinical manifestations are primarily related to the hematopoietic system (intravascular hemolysis, inefficient hematopoiesis, thrombophilia), although pulmonary hypertension, chronic kidney disease and smooth muscle dystonia (dysphagia, abdominal pain), chronic fatigue and erectile dysfunction are part of the clinic spectrum of this illness and associated to the chronic depletion of nitric oxide. The association of PNH with aplastic anemia, myelodysplastic syndromes and acute leukemia are events also known to be part of this clinic spectrum. PNH treatment has been basically empirical and based in the experience of the hematologic groups. In the most of the cases, the therapeutic schemes are aimed to reduce the symptoms or prevent the complications. From the advance in the knowledge of the PNH pathogenesis, they have surged more rational therapies for this disease, in particular the treatment of PNH with the use of eculizumab, monoclonal humanized antibody blocking the C C5 portion. This work presents the first version of the Mexican Consensus on the treatment of the Paroxysmal Nocturnal Hemoglobinuria, written by the Mexican PNH & Complement Diseases Study Group.


Key words: paroxysmal nocturnal hemoglobinuria, hemolysis, thrombosis, eculizumab, bone marrow failure syndromes.


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