Paroxismal nocturnal hemoglobinuria: New treatments and general guidelines for diagnosis;Hemoglobinuria paroxística nocturna: nuevos tratamientos y recomendaciones generales para su diagnóstico

Medicina Clinica

Volumn 134, Issue 8, 2010, Pages 369-374

  Morado, Marta ^a   Subirá, Dolores ^b   López Rubio, Montserrat ^c  

^a HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

^b Hospital UniversitarioFundación Jiménez Díaz   (Spain)

^c HOSPITAL UNIVERSITARIO PRÍNCIPE DE ASTURIAS   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ANDROGEN; ECULIZUMAB; ERYTHROPOIETIN; GLUCOCORTICOID;

ARTICLE; ASTHENIA; BUDD CHIARI SYNDROME; DYSPNEA; ERECTILE DYSFUNCTION; GRANULOCYTOSIS; HEMOLYSIS; HEMOLYTIC ANEMIA; HUMAN; MORBIDITY; MORTALITY; MYELODYSPLASTIC SYNDROME; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PRACTICE GUIDELINE; PULMONARY HYPERTENSION;

HEMOGLOBINURIA, PAROXYSMAL; HUMANS; PRACTICE GUIDELINES AS TOPIC;

EID: 77949282295     PISSN: 00257753     EISSN: 15788989     Source Type: Journal    
DOI: 10.1016/j.medcli.2009.09.026     Document Type: Article

References (33)

1. Parker C., Omine M., Richards S., Nishimura J., Bessler M., Ware R., et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 106 (2005) 3699-3709
2. Hernández-Campo P.M., Almeida J., López A., and Orfao A. Hemoglobinuria paroxística nocturna. Med Clin (Barc) 131 (2008) 617-630
3. De Latour R.P., Mary J.Y., Salanoubat C., Terriou L., Etienne G., Mothy M., et al. Paroxysmal nocturnal hemoglobinuria: Natural history of disease subcategories. Blood 112 (2008) 3099-3106
4. Parker C. Ask the hematologist: How do I diagnose and treat PNH?. The Hematologist (2008)
5. Richards S.J., Rawstron A.C., and Hillmen P. Application of flow cytometry to the diagnosis of paroxysmal nocturnal hemoglobinuria. Cytometry B Clin Cytom 42B (2000) 223-233
6. Richards S.J., and Barnett D. The role of flow cytometry in the diagnosis of paroxysmal nocturnal hemoglobinuria in the clinical laboratory. Clin Lab Med 27 (2007) 577-590
7. Brodsky R.A. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R., Benz E., Shattil S., Furie B., and Cohen H. (Eds). Hematology: Basic principles and practice. 5 (2008), Elsevier, Philadelphia 385-394
8. Rother R.P., Bell L., Hillmen P., and Gladwin M.T. The clinical sequelae of intravascular hemolysis and extracelular plasma hemoglobin: A novel mechanism of human disease. JAMA 293 (2005) 1653
9. Sugimori C., Chuhjo T., Feng X., Yamazaki H., Takami A., Teramura M., et al. Minor population of CD55-CD59- blood cells predicts response to immunosupressive therapy and prognosis in patients with aplastic anemia. Blood 107 (2006) 1308-1314
10. Brodsky R.A. Paroxysmal nocturnal hemoglobinuria: Stem cell and clonality. Hematology Am Soc Hematol Educ Program 1 (2008) 111-115
11. Luzzatto L., and Gianfaldoni G. Recent advances in biological and clinical aspects of paroxysmal nocturnal hemoglobinuria. Int J Hematol 84 (2006) 104-112
12. Tiu R., and Maciesjewski J. Immune pathogenesis of paroxysmal nocturnal hemoglobinuria. Int J Hematol 84 (2006) 113-117
13. Hill A., Hillmen P., Richards S.J., Elebute D., Marsh J.C., Chan J., et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 106 (2005) 2559-2565
14. Hillmen P., Young N.S., Schubert J., Brodsky R.A., Socie G., Muus P., et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 355 (2006) 1233-1243
15. Brodsky R.A., Muus P., Dührsen U., Hill A., Bessler M., Coutre S., et al. Effect of the terminal complement inhibitor eculizumab on patient reported outcomes in paroxysmal nocturnal hemoglobinuria (PNH): Phase III Triumph study results. Blood (ASH Annual Meeting Abstracts) 108 (2006) 3770
16. Brodsky R.A., Young N.S., Antonioli E., Risitano A.M., Schrezenmeier H., Shubert J., et al. Multicentric phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 111 (2008) 1840-1847
17. Parker C. Eculizumab for paroxysmal nocturnal haemoglobinuria. Lancet 373 (2009) 759-767
18. Hillmen P., Muus P., Dührsen U., Risitano A.M., Schubert J., Luzzatto L., et al. Effect on the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 110 (2007) 4123-4128
19. Hillmen P. The role of complement inhibition in PNH. Hematology Am Soc Hematol Educ Program 1 (2008) 116-123
20. Hill A., Ridley S.H., Esser D., Oldroyd R.G., Cullen M.J., Kareclas P., et al. Protection of erythrocytes from human complement-mediated lysis by membrane-targeted recombinant soluble CD59: A new approach to PNH therapy. Blood 107 (2006) 2131-2137
21. Richards S.J., Whitby L., Cullen M.J., Dickinson A.J., Granger V., Reilly J.T., et al. Development and evaluation of a stabilized whole-blood preparation as a process control material for screening of paroxysmal nocturnal hemoglobinuria by flow cytometry. Cytometry B Clin Cytom 79B (2009) 47-55
22. Wang S.A., Pozdyakova O., Jorgensen J.L., Medeiros J., Stachurski D., Anderson M., et al. Detection of paroxysmal nocturnal hemoglobinuria clones in patientswith myelodysplastic syndromes and related bone marrow diseases, with emphasis on diagnostic pitfalls and caveats. Haematologica 94 (2009) 29-37
23. Wang H., Chuhjo T., Yasue S., Omine M., and Nakao S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood 100 (2002) 3897-3902
24. Hernández-Campo P.M., Almeida J., Matarranz S., De Santiago M., Sánchez M.L., and Orfao A. Quantitative analysis of expression of glycosylphosphatidylinositol- anchored proteins during the maturation of different hematopoietic cell compartments of normal bone marrow. Cytometry B Clin Cytom 72B (2007) 34-42
25. Sutherland D.R., Kuek N., Davidson J., Barth D., Chang H., Yeo E., et al. Diagnosing PNH with FLAER and multiparameter flow cytometry. Cytometry B Clin Cytom 72B (2007) 167-177
26. Oelschlaegel U., Besson I., Arnoulet C., Sainty D., Nowak R., Naumann R., et al. A standarized flow cytometric method for screening paroxysmal nocturnal hemoglobinuria (PNH) measuring CD55 and CD59 expression on erythrocytes and granulocytes. Clin Lab Haem 23 (2001) 81-90
27. Hernández-Campo P.M., Martín-Ayuso M., Almeida J., López A., and Orfao A. Comparative analysis of different flow cytometry-based immunophenotypic methods for the analysis of CD59 and CD55 expression on major peripheral blood cell subsets. Cytometry B Clin Cytom 50B (2002) 191-201
28. Hernández-Campo P.M., Almeida J., Sánchez M.L., Malvezzi M., and Orfao A. Normal patterns of expression of glycosylphosphatidylinositol-anchored proteins on different subsets of peripheral blood cells: A frame of reference for the diagnosis of paroxysmal nocturnal hemoglobinuria. Cytometry B Clin Cytom 70B (2006) 71-81
29. Olteanu H., Karandikar N.J., McKenna R.W., and Xu Y. Differential usefulness of various markers in the flow cytometric detection of paroxysmal nocturnal hemoglobinuria in blood and bone marrow. Am J Clin Path 126 (2006) 781-788
30. Richards S.J., Hill A., and Hillmen P. Recent advances in the diagnosis, monitoring and management of patients with paroxysmal nocturnal hemoglobinuria. Cytometry B Clin Cytom 72B (2007) 291-298
31. Araten D.J., Nafa K., Pakdeesuwan K., and Luzzatto L. Clonal populations of hematopoietic cells with paroxysmal nocturnal hemoglobinuria genotype and phenotype are present in normal individuals. Proc Natl Acad Sci 96 (1999) 5209-5214
32. Dunn D., Tanawattanacharioen P., Boccuni P., Nagakura S., Green S.W., Kirby M.R., et al. Paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failure syndromes. Ann Intern Med 131 (1999) 401-408
33. Nakao S., Sugimori S., and Yamakazi H. Clinical significance of a small population of paroxysmal nocturnal hemoglobinuria-type cells in the management of bone marrow failure. Int J Hematol 84 (2006) 118-122