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Paroxysmal Nocturnal Hemoglobinuria

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References

  1. Dacie JV, Lewis SM. Paroxysmal nocturnal hemoglobinuria: clinical manifestations, haematology, and nature of the disease. Ser Haematol. 1972;5:3–23.

    PubMed  CAS  Google Scholar 

  2. Rosse WF. A short history of paroxysmal nocturnal hemoglobinuria: how we came to understand its natural history. In: Omine M, Kinoshita T, eds. Paroxysmal Nocturnal Hemoglobinuria and Related Disorders: Molecular Aspects of Pathogenesis. Tokyo: Springer; 2003:71–83.

    Chapter  Google Scholar 

  3. Nicholson-Weller A, March JP, Rosenfeld SI, Austen KF. Affected erythrocytes of patients with paroxysmal nocturnal hemoglobinuria are deficient in the complement regulatory protein, decay accelerating factor. Proc Natl Acad Sci USA. 1983;80:5066–5070.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  4. Holguin MH, Fredrick LR, Bernshaw NJ, Wilcox LA, Parker CJ. Isolation and characterization of a membrane protein from normal human erythrocytes that inhibits reactive lysis of the erythrocytes of paroxysmal nocturnal hemoglobinuria. J Clin Invest. 1989;84:7–17.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  5. Parker CJ. Historical aspects of paroxysmal nocturnal haemoglobinuria: ‘defining the disease’. Br J Haematol. 2002;117:3–22.

    Article  PubMed  Google Scholar 

  6. Takeda J, Miyata T, Kawagoe K, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993;73:703–711.

    Article  CAS  PubMed  Google Scholar 

  7. Nagakura S, Ishihara S, Dunn DE, et al. Decreased susceptibility of leukemic cells with PIG-A mutation to natural killer cells in vitro. Blood. 2002;100:1031–1037.

    Article  CAS  PubMed  Google Scholar 

  8. Murakami Y, Kosaka H, Maeda Y, et al. Inefficient response of T lymphocytes to glycosylphosphatidylinositol anchor-negative cells: implications for paroxysmal nocturnal hemoglobinuria. Blood. 2002;100:4116–4122.

    Article  CAS  PubMed  Google Scholar 

  9. Wang H, Chuhjo T, Yasue S, Omine M, Nakao S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobin- uria-type cells in bone marrow failure syndrome. Blood. 2002;100:3897–3902.

    Article  CAS  PubMed  Google Scholar 

  10. Inoue N, Murakami Y, Kinoshita T. Molecular genetics of paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2003;77:107–112.

    Article  CAS  PubMed  Google Scholar 

  11. Araten DJ, Nafa K, Pakdeesuwan K, Luzzatto L. Clonal populations of hematopoietic cells with paroxysmal nocturnal hemoglobinuria genotype and phenotype are present in normal individuals. Proc Natl Acad Sci USA. 1999;96: 5209–5214.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  12. Inoue N, Izui T, Kuwayama M, et al. A possible intrinsic mechanism for clonal expansion of PNH abnormal cells. In: Omine M, Kinoshita T, eds. Paroxysmal Nocturnal Hemoglobinuria and Related Disorders: Molecular Aspects of Pathogenesis. Tokyo: Springer; 2003:117–126.

    Chapter  Google Scholar 

  13. Lewis SM, Dacie JV. The aplastic anaemia-paroxysmal nocturnal haemoglobinuria syndrome. Br J Haematol. 1967;13:236–251.

    Article  CAS  PubMed  Google Scholar 

  14. Tichelli A, Gratwohl A, Wursch A, Nissen C, Speck B. Late haematological complications in severe aplastic anaemia. Br J Haematol 1988;69:413–418.

    Article  CAS  PubMed  Google Scholar 

  15. Nishimura J, Murakami Y, Kinoshita T. Paroxysmal nocturnal hemoglobinuria: an acquired genetic disease. Am J Hematol. 1999;62:175–182.

    Article  CAS  PubMed  Google Scholar 

  16. Griscelli-Bennaceur A, Gluckman E, Scrobohaci ML, et al. Aplastic anemia and paroxysmal nocturnal hemoglobinuria: search for a pathogenetic link. Blood. 1995;85:1354–1363.

    PubMed  CAS  Google Scholar 

  17. Schrezenmeier H, Hertenstein B, Wagner B, Raghavachar A, Heimpel H. A pathogenetic link between aplastic anemia and proxysmal nocturnal hemoglobinuria is suggested by a high frequency of aplastic anemia patients with a deficiency of phos- phatidylinositol glycan anchored proteins. Exp Hematol. 1995;23:81–87.

    PubMed  CAS  Google Scholar 

  18. Azenishi Y, Ueda E, Machii T, et al. CD59-deficient blood cells and PIG-A gene abnormalities in Japanese patients with aplastic anaemia. Br J Haematol. 1999;104:523–529.

    Article  CAS  PubMed  Google Scholar 

  19. Luzzatto L, Nafa K. Genetics of PNH. In: Young NS, Moss J, eds. Paroxysmal Nocturnal Hemoglobinuria and the Glycosylphos- phatidylinositol-Linked Proteins. New York, NY: Academic Press, 2000:21–47.

    Google Scholar 

  20. Parker CJ, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria: Recommendations of the International PNH Interest Group. Blood. In press.

  21. Iwanaga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal hemoglobinuria clones in patients with myelodysplastic syndromes. Br J Haematol. 1998;102:465–474.

    Article  CAS  PubMed  Google Scholar 

  22. Dunn DE, Tanawatthnacharoen P, Boccuni P, et al. Paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failure syndromes. Ann Inter Med. 1999;132:401–408.

    Article  Google Scholar 

  23. Feng X, Chuhjo T, Sugimori C, et al. Diazepam-binding inhibitor- related protein 1: a candidate autoantigen in acquired aplastic anemia patients harboring a minor population of paroxysmal nocturnal hemoglobinuria-type cells. Blood. 2004;104:2425–2431.

    Article  CAS  PubMed  Google Scholar 

  24. Bemba M, Guardiola P, Garderet L, et al. Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria. Br J Haematol. 1999;105:366–368.

    Article  CAS  PubMed  Google Scholar 

  25. Raiola AM, Van Lint MT, Lamparelli T, et al. Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria. Haematologica. 2000;85:59–62.

    PubMed  CAS  Google Scholar 

  26. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal haemoglobinuria. N Engl J Med. 1995;333:1253–1258.

    Article  CAS  PubMed  Google Scholar 

  27. Socie G, Rosenfeld S, Frickhofen N, Gluckman E, Tichelli A. Late clonal diseases of treated aplastic anemia. Semin Hematol. 2000;37:91–101.

    Article  CAS  PubMed  Google Scholar 

  28. Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003;101:1236–1242.

    Article  CAS  PubMed  Google Scholar 

  29. Wang H, Chuhjo T, Yasue S, Omine M, Nakao S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100:3897–3902.

    Article  CAS  PubMed  Google Scholar 

  30. Maciejewski JP, Follmann D, Nakamura R, et al. Incresased frequency of HLA-DR2 in patients with paroxysmal nocturnal hemoglobinuria and the PNH/aplastic anemia syndrome. Blood. 2001;98:3515–3519.

    Article  Google Scholar 

  31. Meyers G, Parker CJ. Management issues in paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2003;77:125–132.

    Article  PubMed  Google Scholar 

  32. Hall C, Richards S, Hillmen P. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003;102:3587–3591.

    Article  CAS  PubMed  Google Scholar 

  33. Nishimura JI, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore). 2004;83:193–207.

    Article  Google Scholar 

  34. Fujioka S, Asai T. Prognostic features of paroxysmal nocturnal hemoglobinuria in Japan. Nippon Ketsueki Gakkai Zasshi. 1989;52:1386–1394.

    PubMed  CAS  Google Scholar 

  35. Socie G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet. 1996;348:573–577.

    Article  CAS  PubMed  Google Scholar 

  36. Nishimura J, Phillips KL, Ware RE, et al. Efficient retrovirus-mediated PIG-A gene transfer and stable restoration of GPI-anchored protein expression in cells with the PNH phenotype. Blood. 2002;99:2748–2751.

    Article  CAS  Google Scholar 

  37. Parker CJ. An overview of the development of specific inhibitors of complement: opportunities for therapy of paroxysmal nocturnal hemoglobinuria. In Omine M, Kinoshita T, eds. Paroxysmal Nocturnal Hemoglobinuria and Related Disorders: Molecular Aspects of Pathogenesis. Tokyo: Springer; 2003:11–23.

    Chapter  Google Scholar 

  38. Hillmen PV, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004;350:552–559.

    Article  CAS  PubMed  Google Scholar 

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Authors and Affiliations

  1. Division of Hematology, Internal Medicine, Showa University Fujigaoka Hospital, 1-30 Fujigaoka, Aoba-ku, 227-8501, Yokohama city, Japan

    Mitsuhiro Omine

  2. Department of Immunoregulation, Research Institute for Microbial Diseases, Osaka University, Osaka, Japan

    Taroh Kinoshita

  3. Department of Hematology/Oncology, Wakayama Medical University, Wakayama, Japan

    Hideki Nakakuma

  4. Experimental hematology and hematopoiesis section, Taussig Cancer Center, Cleveland, OH, USA

    Jaroslaw P.Maciejewski

  5. Division of Hematology, University of Utah School of Medicine, Salt Lake City, UT, USA

    Charles J. Parker

  6. Hématologie, Greffe de moelle Hôpital, St Louis, Paris, France

    Gérard Socié

Authors
  1. Mitsuhiro Omine
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  2. Taroh Kinoshita
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  3. Hideki Nakakuma
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  4. Jaroslaw P.Maciejewski
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  5. Charles J. Parker
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  6. Gérard Socié
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Corresponding author

Correspondence to Mitsuhiro Omine.

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Omine, M., Kinoshita, T., Nakakuma, H. et al. Paroxysmal Nocturnal Hemoglobinuria. Int J Hematol 82, 417–421 (2005). https://doi.org/10.1532/IJH97.05140

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  • DOI: https://doi.org/10.1532/IJH97.05140

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