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Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán

2014, Number 1

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Rev Invest Clin 2014; 66 (1)

Paroxysmal nocturnal hemoglobinuria in México: a 30-year, single institution experience

Hernández-Reyes J, González-Ramírez MP, Martagón-Herrera NÁ, Rosales-Durón AD, Ruiz-Delgado GJ, Ruiz-Argüelles GJ

Language: English
References: 30
Page: 12-16
PDF size: 145.08 Kb.


ABSTRACT

Background. Paroxysmal nocturnal hemoglobinuria (PNH) stems from chronic, complement-mediated, intravascular hemolysis, which results in anemia, hemoglobinuria, fatigue, and other hemolysis-related disabling symptoms. Novel diagnostic methods have led into an increased identification of the disease. Aims. To analyze the salient features of patients with PNH identified in a single institution in México, in a 30-year period. Material and methods. The records of 31 patients with PNH identified between 1984 and 2013 were reviewed; 20 females. Median age was 39 years, range 5 to 88. Patients were followed for periods of 0.5 to 221 months, median 46 months. Results. Most patients (97%) presented peripheral blood cytopenias, 11 (35%) presented a thrombotic episode, whereas 4 (13%) showed hemolytic anemia. No thrombotic episode was fatal. In the cytopenic group, 4 patients with hemolysis were included and in the patients with the hemolytic variant the red blood cell destruction process was continuous while not paroxysmal. Anemia was recorded in 30 individuals; median hemoglobin levels were 8.5 g/dL, range 3.7 to 12.8. Leukopenia was present in 18 individuals; median white blood cell count was 3.3 x 109/L, range 1.6 to 10.8, whereas thrombocytopenia was present in 18 subjects; median platelet count was 67 x 109/L, range 6 to 546. Pancytopenia was present in 15 patients. Hemoglobinuria was recorded in 12 patients and low free haptoglobin levels coupled with increased lactic dehydrogenase levels, consonant with hemolysis in 4 patients. Conclusions. In México the cytopenic variants are considerably more common than either the hemolytic or the thrombotic variants of the disease, this being particularly relevant since only the hemolytic variants of PNH are the ones which show a good response to the complement-blocking therapy employed nowadays in the treatment of the disease.


REFERENCES

  1. Risitano AM. Paroxysmal nocturnal hemoglobinuria and the complement system: recent insights and novel anticomplement strategies. Adv Exp Med Biol 2013; 735: 155-72.

  2. Ruiz-Argüelles GJ, Morales-Aceves R, Labardini J, Kraus- Weisman A. Hemoglobinuria paroxística nocturna. Experiencia de 30 años en el Instituto Nacional de la Nutrición (México). Sangre (Barc) 1981; 26: 463-70.

  3. Góngora-Biachi RA, Khoury-Mancebo L, Dillman E, Hurtado- Monroy R, González-Llaven J. Relation between the hemolytic system and cell variants in paroxysmal nocturnal hemoglobinuria. Sangre (Barc) 1984; 29: 384-90.

  4. Góngora-Biachi RA, González-Martínez P. Paroxysmal nocturnal hemoglobinuria: current concepts and controversies. Rev Clin Esp 1987; 180: 438-43.

  5. Góngora-Biachi RA, Sosa-Muñoz J, Duarte-Zapata L, Pinto- Escalante D, González-Martínez P, Achach-Asaf J. Nocturnal paroxysmal hemoglobinuria: analysis of 36 cases. Sangre (Barc) 1987; 32: 27-37. Figure 1. Overall survival of the 31 patients with paroxymal nocturnal hemoglobinuria. 0 20 40 60 80 100 120 140 160 180 200 220 Survival in months 1.0 0.9 0.8 0.7 0.6 0.5 0.4 0.3 0.2 0.1 0 Percentage 16 Hernández-Reyes J, et al. PNH in México. Rev Invest Clin 2014; 66 (1): 12-16

  6. Góngora-Biachi RA. El fenómeno hemolítico en la hemoglobinuria paroxística nocturna. Rev Biomed 1990; 1: 159-62.

  7. Góngora-Biachi RA, Gonzalez-Martinez P, Pinto-Escalante D, Ceballos-Quintal JM. Chromosomic findings in patients with paroxysmal nocturnal hemoglobinuria. Int J Hematol 1993; 58: 163-7.

  8. Sánchez-Valle E, Morales-Polanco MR, Gómez-Morales E, Gutiérrez-Alamillo LI, Gutiérrez-Espindola G, Pizzuto- Chávez J. Tratamiento de la hemoglobinuria paroxística nocturna con globulina antilinfocitaria. Rev Invest Clin 1993; 45: 457-61. 9 Gongora-Biachi R, Gonzalez-Martinez P, Gonzalez-Llaven J, et al. Clinical spectrum and prognostic factors in paroxysmal nocturnal hemoglobinuria in Mexico. Rev Invest Clin 1994; 46 (Supl. 1): 187.

  9. Góngora-Biachi RA, González-Martínez P, Sosa-Muñoz J, Castro-Sansores C, Delgado-Lamas JL, Vázquez-Villegas V, Rico-Balzadua G, et al. Natural history of paroxysmal nocturnal hemoglobinuria in adolescents, adults, and children: the Mexican experience. Sangre (Barc) 1997; 42: 171-7.

  10. Góngora-Biachi RA, González-Martínez P. Hemoglobinuria paroxística nocturna: apuntaciones sobre su historia. Rev Biomed 1999; 10: 129-36.

  11. Ruiz-Argüelles GJ, Ramírez-Cisneros FJ, Ruiz-Argüelles A, Pérez-Romano B, Morales Aceves R. Deficiencia de proteínas de superficie ancladas por glucosil-fosfatidil-inositol en células de pacientes mexicanos con hipoplasia medular. Rev Invest Clin 1999; 51: 5-9.

  12. Piedras J, López-Karpovitch X. Flow cytometric analysis of glycosylphosphatidyl-inositol-anchored proteins to assess paroxysmal nocturnal hemoglobinuria clone size. Cytometry 2000; 42: 234-8.

  13. Góngora-Biachi RA. Hemoglobinuria paroxística nocturna: un síndrome de falla medular. Rev Hematol Méx 2004; 5: 67-79.

  14. Vega S, Reyes-Maldonado E, Vela-Ojeda J, Xólotl-Castillo M, Montiel-Cervantes L, Cerecedo-Mercado DA. Estudio ultraestructural de las plaquetas de pacientes con hemoglobinuria paroxística nocturna. Rev Invest Clin 2006; 58: 475-86.

  15. Ferrer-Argote VE, Mendoza-García E, García-García JJ, Collazo-Jaloma J, León-Gonzalez G. Frecuencia de hemoglobinuria paroxística nocturna en pacientes mexicanos con anemia aplásica tratados con andrógenos. Rev Med Hosp Gen Mex 2008; 71: 137-40.

  16. Ruiz-Argüelles GJ, Ruiz-Delgado GJ, Calderón-Meza E, Ruiz-Argüelles A, Garcés-Eisele J. Donor-derived hairy cell leukemia. Leukemia Lymph 2009; 50: 1712-4.

  17. Ceballos-López AA, Saldaña-Vázquez R, Pequeño-Luévano MP, Salazar-Riojas MR, Méndez-Ramírez N, Reyes-López MA, Gómez-Almaguer D. Detección de clonas de hemoglobinuria paroxística nocturna por citofluorometría de cuatro colores en un laboratorio de hematología del Noreste de México. Rev Hematol Méx 2012; 13: 95-8.

  18. Sutherland DR, Acton E, Keeney M, Davis BH, Illingworth A. Use of CD157 in FLAER-based assays for high-sensitivity PNH granulocyte and PNH monocyte detection. Cytometry B Clin Cytom 2013. Doi: 10.1002/cyto.b.21111 [Epub ahead of print].

  19. Pu JJ, Brodsky RA. Paroxysmal nocturnal hemoglobinuria from bench to bedside. Clin Transl Sci 2011; 4(3): 219-24. Doi: 10.1111/j.1752-8062.2011.00262.x.

  20. Gupta R, Pandey P, Choudhry R, Kashyap R, Mehrotra M, Naseem S, Nityanand S. A prospective comparison of four techniques for diagnosis of paroxysmal nocturnal hemoglobinuria. Int J Lab Hematol 2007; 29: 119-26.

  21. Sutherland DR, Kuek N, Azcona-Olivera J, Anderson T, Acton E, Barth D, Keeney M. Use of a FLAER-based WBC assay in the primary screening of PNH clones. Am J Clin Pathol 2009; 132: 564-72.

  22. Hillmen P, Muus P, Röth A, Elebute MO, Risitano AM, Schrezenmeier H, Szer J, Browne P, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol 2013; 162: 62-73.

  23. Gómez-Almaguer D, Jaime-Pérez JC, Ruiz-Argüelles GJ. Antibodies in the treatment of aplastic anemia. Arch Immunol Ther Exp (Warsz) 2012 [Epub ahead of print].

  24. Jaime-Pérez JC, Ruiz-Argüelles GJ, Gómez-Almaguer D. Haematopoietic stem cell transplantation to treat aplastic anemia. Expert Opin Biol Ther 2005; 5: 617-26.

  25. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995; 333; 1253-7.

  26. Socie G, Mary JY, de Gramont A, Rio B, Leporrier M, Rose C, Heudler P, et al. Paroxysmal nocturnal hemoglobinuria: Long term follow up and prognostic factors. Lancet 1996; 348: 573-7.

  27. Rosse WF, Nishimura J. Clinical manifestations of paroxysmal nocturnal hemoglobinuria: Present State and future problems. Int J Hematol 2003; 77: 107-12.

  28. Ruiz-Argüelles GJ. Treatment of patients with PNH. Blood 1983; 61: 610.

  29. Ruiz-Argüelles GJ, González-Estrada S, Garcés-Eisele J, Ruiz- Argüelles A. Primary thrombophilia in México I: A prospective study. Am J Hematol 1999; 60: 1-5.

  30. Ruiz-Argüelles GJ, Garcés-Eisele J, Camacho-Alarcón C, Reyes- Nuñez V, Moncada-González B, Valdés-Tapia P, León- Montes N, et al. Primary thrombophilia in Mexico IX: The glycoprotein IIIa PLA1/A2 polymorphism is not associated with the sticky platelet syndrome phenotype. Clin Appl Thromb Hemost 2012 [Epub ahead of prin




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