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De novo “Classic” Paroxysmal Nocturnal Hemoglobinuria (PNH) (Marchiafava-Micheli Syndrome)

  • Lisa Pleyer
  • Richard Greil

Abstract

PNH, although a benign clonal stem cell myelopathy, is included in the myeloproliferative disorders by some [1]. PNH is mainly a disease of adults, but can be present in adolescence or childhood on rare occasions [2]. Overall both genders are affected in approximately equivalent numbers. However, some Asian studies report a strong male preponderance [3]. The median age at diagnosis is 30 years in Caucasians and 45 years in Asian patients [4].

Keywords

Paroxysmal Nocturnal Hemoglobinuria Bone Marrow Failure Paroxysmal Nocturnal Hemoglobinuria Clone Paroxysmal Nocturnal Hemoglobinuria Cell Paroxysmal Nocturnal Hemoglobinuria Clone Size 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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References

  1. [1]
    Parker CJ (2007) The pathophysiology of paroxysmal nocturnal hemoglobinuria. Exp Hematol 35: 523–533PubMedGoogle Scholar
  2. [2]
    Ware RE, Hall SE, Rosse WF (1991) Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence. N Engl J Med 325: 991–996PubMedGoogle Scholar
  3. [3]
    Le XF, Yang TY, Yang XY, Wang XM (1990) Characteristics of paroxysmal nocturnal hemoglobinuria in China. Clinical analysis of 476 cases. Chin Med J (Engl) 103: 885–889Google Scholar
  4. [4]
    Nishimura J, Kanakura Y, Ware RE et al. (2004) Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore) 83: 193–207Google Scholar
  5. [5]
    Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV (1995) Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 333: 1253–1258PubMedGoogle Scholar
  6. [6]
    Socie G, Mary JY, de Gramont A et al. (1996) Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet 348: 573–577PubMedGoogle Scholar
  7. [7]
    Brodsky RA, Hu R (2006) PIG-A mutations in paroxysmal nocturnal hemoglobinuria and in normal hematopoiesis. Leuk Lymphoma 47: 1215–1221PubMedGoogle Scholar
  8. [8]
    Brodsky RA (2008) Advances in the diagnosis and therapy of paroxysmal nocturnal hemoglobinuria. Blood Rev 22: 65–74PubMedGoogle Scholar
  9. [9]
    Nishimura J, Ware RE, Burnette A et al. (2002) The hematopoietic defect in PNH is not due to defective stroma, but is due to defective progenitor cells. Blood Cells Mol Dis 29: 159–167PubMedGoogle Scholar
  10. [10]
    Navenot JM, Muller JY, Blanchard D (1998) Investigation of the survival of paroxysmal nocturnal hemoglobinuria red cells through the immunophenotyping of reticulocytes. Transfusion 38: 337–342PubMedGoogle Scholar
  11. [11]
    Josten KM, Tooze JA, Borthwick-Clarke C, Gordon-Smith EC, Rutherford TR (1991) Acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria: studies on clonality. Blood 78: 3162–3167PubMedGoogle Scholar
  12. [12]
    Endo M, Ware RE, Vreeke TM et al. (1996) Molecular basis of the heterogeneity of expression of glycosyl phosphatidylinositol anchored proteins in paroxysmal nocturnal hemoglobinuria. Blood 87: 2546–2557PubMedGoogle Scholar
  13. [13]
    Ishiyama K, Chuhjo T, Wang H, Yachie A, Omine M, Nakao S (2003) Polyclonal hematopoiesis maintained in patients with bone marrow failure harboring a minor population of paroxysmal nocturnal hemoglobinuria-type cells. Blood 102: 1211–1216PubMedGoogle Scholar
  14. [14]
    Mortazavi Y, Merk B, McIntosh J, Marsh JC, Schrezenmeier H, Rutherford TR (2003) The spectrum of PIG-A gene mutations in aplastic anemia/paroxysmal nocturnal hemoglobinuria (AA/PNH): a high incidence of multiple mutations and evidence of a mutational hot spot. Blood 101: 2833–2841PubMedGoogle Scholar
  15. [15]
    Merry AH, Rawlinson VI, Uchikawa M, Daha MR, Sim RB (1989) Studies on the sensitivity to complement-mediated lysis of erythrocytes (Inab phenotype) with a deficiency of DAF (decay accelerating factor). Br J Haematol 73: 248–253PubMedGoogle Scholar
  16. [16]
    Araten DJ, Nafa K, Pakdeesuwan K, Luzzatto L (1999) Clonal populations of hematopoietic cells with paroxysmal nocturnal hemoglobinuria genotype and phenotype are present in normal individuals. Proc Natl Acad Sci USA 96: 5209–5214PubMedGoogle Scholar
  17. [17]
    Chen G, Kirby M, Zeng W, Young NS, Maciejewski JP (2002) Superior growth of glycophosphatidy linositol-anchored protein-deficient progenitor cells in vitro is due to the higher apoptotic rate of progenitors with normal phenotype in vivo. Exp Hematol 30: 774–782PubMedGoogle Scholar
  18. [18]
    Elebute MO, Rizzo S, Tooze JA, Marsh JC, Gordon-Smith EC, Gibson FM (2003) Evaluation of the haemopoietic reservoir in de novo haemolytic paroxysmal nocturnal haemoglobinuria. Br J Haematol 123: 552–560PubMedGoogle Scholar
  19. [19]
    Luzzatto L, Bessler M, Rotoli B (1997) Somatic mutations in paroxysmal nocturnal hemoglobinuria: a blessing in disguise? Cell 88: 1–4PubMedGoogle Scholar
  20. [20]
    Moyo VM, Mukhina GL, Garrett ES, Brodsky RA (2004) Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. Br J Haematol 126: 133–138PubMedGoogle Scholar
  21. [21]
    Crosby WH (1953) Paroxysmal nocturnal hemoglobinuria: relation of the clinical manifestations to underlying pathogenic mechanisms. Blood 8: 769–812PubMedGoogle Scholar
  22. [22]
    Hansch G, Hammer C, Jiji R, Rother U, Shin M (1983) Lysis of paroxysmal nocturnal hemoglobinuria erythrocytes by acid-activated serum. Immunobiology 164: 118–126PubMedGoogle Scholar
  23. [23]
    Clark DA, Butler SA, Braren V, Hartmann RC, Jenkins DE Jr. (1981) The kidneys in paroxysmal nocturnal hemoglobinuria. Blood 57: 83–89PubMedGoogle Scholar
  24. [24]
    Morris CR, Kato GJ, Poljakovic M et al. (2005) Dysregulated arginine metabolism, hemolysis-associated pulmonary hypertension, and mortality in sickle cell disease. JAMA 294: 81–90PubMedGoogle Scholar
  25. [25]
    Sogo N, Magid KS, Shaw CA, Webb DJ, Megson IL (2000) Inhibition of human platelet aggregation by nitric oxide donor drugs: relative contribution of cGMP-independent mechanisms. Biochem Biophys Res Commun 279: 412–419PubMedGoogle Scholar
  26. [26]
    Wiedmer T, Hall SE, Ortel TL, Kane WH, Rosse WF, Sims PJ (1993) Complement-induced vesiculation and exposure of membrane prothrombinase sites in platelets of paroxysmal nocturnal hemoglobinuria. Blood 82: 1192–1196PubMedGoogle Scholar
  27. [27]
    Ninomiya H, Hasegawa Y, Nagasawa T, Abe T (1997) Excess soluble urokinase-type plasminogen activator receptor in the plasma of patients with paroxysmal nocturnal hemoglobinuria inhibits cell-associated fibrinolytic activity. Int J Hematol 65: 285–291PubMedGoogle Scholar
  28. [28]
    Liebman HA, Feinstein DI (2003) Thrombosis in patients with paroxysmal noctural hemoglobinuria is associated with markedly elevated plasma levels of leukocyte-derived tissue factor. Thromb Res 111: 235–238PubMedGoogle Scholar
  29. [29]
    Rother RP, Bell L, Hillmen P, Gladwin MT (2005) The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA 293: 1653–1662PubMedGoogle Scholar
  30. [30]
    Erhart SM, Cole DJ, Patel PM, Drummond JC, Burhop KE (2000) Effect of alpha-alpha diaspirin crosslinked hemoglobin (DCLHb) on the potency of sodium nitroprusside and nitroglycerine to decrease blood pressure in rats: a dose-response study. Artif Cells Blood Substit Immobil Biotechnol 28: 385–396PubMedGoogle Scholar
  31. [31]
    Nelin LD, Nash HE, Chicoine LG (2001) Cytokine treatment increases arginine metabolism and uptake in bovine pulmonary arterial endothelial cells. Am J Physiol Lung Cell Mol Physiol 281: L1232–L1239PubMedGoogle Scholar
  32. [32]
    King NE, Rothenberg ME, Zimmermann N (2004) Arginine in asthma and lung inflammation. J Nutr 134: 2830S–2836SPubMedGoogle Scholar
  33. [33]
    Mansoor MA, Seljeflot I, Arnesen H et al. (2004) Endothelial cell adhesion molecules in healthy adults during acute hyperhomocysteinemia and mild hypertriglyceridemia. Clin Biochem 37: 408–414PubMedGoogle Scholar
  34. [34]
    Schutt C, Schilling T, Grunwald U et al. (1995) Human monocytes lacking the membrane-bound form of the bacterial lipopolysaccharide (LPS) receptor CD14 can mount an LPS-induced oxidative burst response mediated by a soluble form of CD14. Res Immunol 146: 339–350PubMedGoogle Scholar
  35. [35]
    Ruggiero G, Terrazzano G, Becchimanzi C et al. (2004) GPI-defective monocytes from paroxysmal nocturnal hemoglobinuria patients show impaired in vitro dendritic cell differentiation. J Leukoc Biol 76: 634–640PubMedGoogle Scholar
  36. [36]
    Suzuki K, Watanabe T, Sakurai S et al. (1999) A novel glycosylphosphatidyl inositol-anchored protein on human leukocytes: a possible role for regulation of neutrophil adherence and migration. J Immunol 162: 4277–4284PubMedGoogle Scholar
  37. [37]
    Cacciapuoti C, Terrazzano G, Barone L et al. (2007) Glycosyl-phosphatidyl-inositol-defective granulocytes from paroxysmal nocturnal haemoglobinuria patients show increased bacterial ingestion but reduced respiratory burst induction. Am J Hematol 82: 98–107PubMedGoogle Scholar
  38. [38]
    Schubert J, Stroehmann A, Scholz C, Schmidt RE (1995) Glycosylphosphatidylinositol (GPI)-anchored surface antigens in the allogeneic activation of T cells. Clin Exp Immunol 102: 199–203PubMedGoogle Scholar
  39. [39]
    Richards SJ, Morgan GJ, Hillmen P (1999) Analysis of T cells in paroxysmal nocturnal hemoglobinuria provides direct evidence that thymic T-cell production declines with age. Blood 94: 2790–2799PubMedGoogle Scholar
  40. [40]
    Schubert J, Uciechowski P, Zielinska-Skowronek M, Tietjen C, Leo R, Schmidt RE (1992) Differences in activation of normal and glycosylphosphatidylinositol-negative lymphocytes derived from patients with paroxysmal nocturnal hemoglobinuria. J Immunol 148: 3814–3819PubMedGoogle Scholar
  41. [41]
    Terrazzano G, Sica M, Becchimanzi C et al. (2005) T cells from paroxysmal nocturnal haemoglobinuria (PNH) patients show an altered CD40-dependent pathway. J Leukoc Biol 78: 27–36PubMedGoogle Scholar
  42. [42]
    Richards SJ, Morgan GJ, Hillmen P (2000) Immunophenotypic analysis of B cells in PNH: insights into the generation of circulating naive and memory B cells. Blood 96: 3522–3528PubMedGoogle Scholar
  43. [43]
    Nakakuma H, Nagakura S, Kawaguchi T et al. (1994) Persistence of affected T lymphocytes in long-term clinical remission in paroxysmal nocturnal hemoglobinuria. Blood 84: 3925–3928PubMedGoogle Scholar
  44. [44]
    Telen MJ, Rosse WF, Parker CJ, Moulds MK, Moulds JJ (1990) Evidence that several high-frequency human blood group antigens reside on phosphatidylinositol-linked erythrocyte membrane proteins. Blood 75: 1404–1407PubMedGoogle Scholar
  45. [45]
    Telen MJ (1995) Glycosyl phosphatidylinositol-linked blood group antigens and paroxysmal nocturnal hemoglobinuria. Transfus Clin Biol 2: 277–290PubMedGoogle Scholar
  46. [46]
    Rosse WF, Nishimura J, (2003) Clinical manifestations of paroxysmal nocturnal hemoglobinuria: present state and future problems. Int J Hematol 77: 113–120PubMedGoogle Scholar
  47. [47]
    Nakakuma H, Hidaka M, Nagakura S et al. (1995) Expression of cryptantigen Th on paroxysmal nocturnal hemoglobinuria erythrocytes in association with a hemolytic exacerbation. J Clin Invest 96: 201–206PubMedGoogle Scholar
  48. [48]
    Nakakuma H (1996) Mechanism of intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH). Am J Hematol 53: 22–29PubMedGoogle Scholar
  49. [49]
    Nakakuma H, Kawaguchi T (1996) Paroxysmal nocturnal hemoglobinuria (PNH): mechanism of intravascular hemolysis. Crit Rev Oncol Hematol 24: 213–229PubMedGoogle Scholar
  50. [50]
    Taylor SR, Hashida Y, Jaffe R (1989) Venous thrombosis and endothelial proliferation in paroxysmal nocturnal hemoglobinuria. Pediatr Pathol 9: 757–764PubMedGoogle Scholar
  51. [51]
    Zimmerman D, Bell WR (1980) Venous thrombosis and splenic rupture in paroxysmal nocturnal hemoglobinuria. Am J Med 68: 275–279PubMedGoogle Scholar
  52. [52]
    Dunphy CH, Sotelo-Avila C, Luisiri A, Chu JY (1994) Paroxysmal nocturnal hemoglobinuria associated with venous thrombosis and papillary endothelial hyperplasia presenting as ulcerated duodenal mass. Arch Pathol Lab Med 118: 837–839PubMedGoogle Scholar
  53. [53]
    Hill A, Richards SJ, Hillmen P (2007) Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol 137: 181–192PubMedGoogle Scholar
  54. [54]
    Richards SJ, Norfolk DR, Swirsky DM, Hillmen P (1998) Lymphocyte subset analysis and glycosylphosphatidylinositol phenotype in patients with paroxysmal nocturnal hemoglobinuria. Blood 92: 1799–1806PubMedGoogle Scholar
  55. [55]
    Tseng JE, Hall SE, Howard TA, Ware RE (1995) Phenotypic and functional analysis of lymphocytes in paroxysmal nocturnal hemoglobinuria. Am J Hematol 50: 244–253PubMedGoogle Scholar
  56. [56]
    Maciejewski JP, Young NS, Yu M, Anderson SM, Sloand EM (1996) Analysis of the expression of glycosylphosphatidylinositol anchored proteins on platelets from patients with paroxysmal nocturnal hemoglobinuria. Thromb Res 83: 433–447PubMedGoogle Scholar
  57. [57]
    Lewis SM, Dacie JV (1965) Neutrophil (leucocyte) alkaline phosphatase in paroxysmal nocturnal haemoglobinuria. Br J Haematol 11: 549–556PubMedGoogle Scholar
  58. [58]
    Auditore JV, Hartmann RC (1959) Paroxysmal nocturnal hemoglobinuria. II. Erythrocyte acetylcholinesterase defect. Am J Med 27: 401–410PubMedGoogle Scholar
  59. [59]
    Auditore JV, Hartmann RC, Flexner JM, Balchum OJ (1960) The erythrocyte acetylcholinesterase enzyme in paroxysmal nocturnal hemoglobinuria. Arch Pathol 69: 534–543PubMedGoogle Scholar
  60. [60]
    Bobolis KA, Moulds JJ, Telen MJ (1992) Isolation of the JMH antigen on a novel phosphatidylinositol-linked human membrane protein. Blood 79: 1574–1581PubMedGoogle Scholar
  61. [61]
    Hartmann RC, Jenkins DE Jr, Arnold AB (1970) Diagnostic specificity of sucrose hemolysis test for paroxysmal nocturnal hemoglobinuria. Blood 35: 462–475PubMedGoogle Scholar
  62. [62]
    Rosse WF, Logue GL, Adams J, Crookston JH (1974) Mechanisms of immune lysis of the red cells in hereditary erythroblastic multinuclearity with a positive acidified serum test and paroxysmal nocturnal hemoglobinuria. J Clin Invest 53: 31–43PubMedGoogle Scholar
  63. [63]
    Crookston JH, Crookston MC, Burnie KL et al. (1969) Hereditary erythroblastic multinuclearity associated with a positive acidified-serum test: a type of congenital dyserythropoietic anaemia. Br J Haematol 17: 11–26PubMedGoogle Scholar
  64. [64]
    Crookston JH, Crookston MC, Rosse WF (1972) Red-cell abnormalities in HEMPAS (hereditary erythroblastic multinuclearity with a positive acidified-serum test). Br J Haematol 23: Suppl-91Google Scholar
  65. [65]
    Verwilghen RL, Lewis SM, Dacie JV, Crookston JH, Crookston MC (1973) Hempas: congenital dyserythropoietic anaemia (type II). Q J Med 42: 257–278PubMedGoogle Scholar
  66. [66]
    Telen MJ, Green AM (1989) The Inab phenotype: characterization of the membrane protein and complement regulatory defect. Blood 74: 437–441PubMedGoogle Scholar
  67. [67]
    Holguin MH, Martin CB, Bernshaw NJ, Parker CJ (1992) Analysis of the effects of activation of the alternative pathway of complement on erythrocytes with an isolated deficiency of decay accelerating factor. J Immunol 148: 498–502PubMedGoogle Scholar
  68. [68]
    Shichishima T, Saitoh Y, Terasawa T, Noji H, Kai T, Maruyama Y (1999) Complement sensitivity of erythrocytes in a patient with inherited complete deficiency of CD59 or with the Inab phenotype. Br J Haematol 104: 303–306PubMedGoogle Scholar
  69. [69]
    Matthes T, Tullen E, Poole J et al. (2002) Acquired and transient RBC CD55 deficiency (Inab phenotype) and anti-IFC. Transfusion 42: 1448–1457PubMedGoogle Scholar
  70. [70]
    Yamaguchi M, Machii T, Azenishi Y et al. (2000) Detection of small populations of CD59-deficient erythrocytes in patients with aplastic anemia or myelodysplastic syndrome and normal individuals. Blood Cells Mol Dis 26: 247–254PubMedGoogle Scholar
  71. [71]
    Hu R, Mukhina GL, Piantadosi S, Barber JP, Jones RJ, Brodsky RA (2005) PIG-A mutations in normal hematopoiesis. Blood 105: 3848–3854PubMedGoogle Scholar
  72. [72]
    Rawstron AC, Rollinson SJ, Richards S et al. (1999) The PNH phenotype cells that emerge in most patients after CAMPATH-1H therapy are present prior to treatment. Br J Haematol 107: 148–153PubMedGoogle Scholar
  73. [73]
    Fracchiolla NS, Barcellini W, Bianchi P, Motta M, Fermo E, Cortelezzi A (2001) Biological and molecular characterization of PNH-like lymphocytes emerging after Campath-1H therapy. Br J Haematol 112: 969–971PubMedGoogle Scholar
  74. [74]
    Brett SJ, Baxter G, Cooper H et al. (1996) Emergence of CD52-, glycosylphosphatidylinositol-anchor-deficient lymphocytes in rheumatoid arthritis patients following Campath-1H treatment. Int Immunol 8: 325–334PubMedGoogle Scholar
  75. [75]
    Hertenstein B, Wagner B, Bunjes D et al. (1995) Emergence of CD52-, phosphatidylinositolglycan-anchor-deficient T lymphocytes after in vivo application of Campath-1H for refractory B-cell non-Hodgkin lymphoma. Blood 86: 1487–1492PubMedGoogle Scholar
  76. [76]
    Taylor VC, Sims M, Brett S, Field MC (1997) Antibody selection against CD52 produces a paroxysmal nocturnal haemoglobinuria phenotype in human lymphocytes by a novel mechanism. Biochem J 322 (Pt 3): 919–925PubMedGoogle Scholar
  77. [77]
    Lagneaux L, Delforge A, Dorval C, Bron D, Stryckmans P (1993) Excessive production of transforming growth factor-beta by bone marrow stromal cells in B-cell chronic lymphocytic leukemia inhibits growth of hematopoietic precursors and interleukin-6 production. Blood 82: 2379–2385PubMedGoogle Scholar
  78. [78]
    Araten DJ, Swirsky D, Karadimitris A et al. (2001) Cytogenetic and morphological abnormalities in paroxysmal nocturnal haemoglobinuria. Br J Haematol 115: 360–368PubMedGoogle Scholar
  79. [79]
    Inoue N, Izui-Sarumaru T, Murakami Y et al. (2006) Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH). Blood 108: 4232–4236PubMedGoogle Scholar
  80. [80]
    Parlier V, Tiainen M, Beris P, Miescher PA, Knuutila S, Jotterand BM (1992) Trisomy 8 detection in granulomonocytic, erythrocytic and megakaryocytic lineages by chromosomal in situ suppression hybridization in a case of refractory anaemia with ringed sideroblasts complicating the course of paroxysmal nocturnal haemoglobinuria. Br J Haematol 81: 296–304PubMedGoogle Scholar
  81. [81]
    Longo L, Bessler M, Beris P, Swirsky D, Luzzatto L (1994) Myelodysplasia in a patient with pre-existing paroxysmal nocturnal haemoglobinuria: a clonal disease originating from within a clonal disease. Br J Haematol 87: 401–403PubMedGoogle Scholar
  82. [82]
    Parker C, Omine M, Richards S et al. (2005) Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 106: 3699–3709PubMedGoogle Scholar
  83. [83]
    Hall C, Richards S, Hillmen P (2003) Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood 102: 3587–3591PubMedGoogle Scholar
  84. [84]
    Sholar PW, Bell WR (1985) Thrombolytic therapy for inferior vena cava thrombosis in paroxysmal nocturnal hemoglobinuria. Ann Intern Med 103: 539–541PubMedGoogle Scholar
  85. [85]
    Griffith JF, Mahmoud AE, Cooper S, Elias E, West RJ, Olliff SP (1996) Radiological intervention in Budd-Chiari syndrome: techniques and outcome in 18 patients. Clin Radiol 51: 775–784PubMedGoogle Scholar
  86. [86]
    Ray JG, Burows RF, Ginsberg JS, Burrows EA (2000) Paroxysmal nocturnal hemoglobinuria and the risk of venous thrombosis: reviewand recommendations for management of the pregnant and nonpregnant patient. Haemostasis 30: 103–117PubMedGoogle Scholar
  87. [87]
    Rosse WF (1997) Paroxysmal nocturnal hemoglobinuria as a molecular disease. Medicine (Baltimore) 76: 63–93Google Scholar
  88. [88]
    Hartmann RC, Jenkins DE Jr, McKee LC, Heyssel RM (1966) Paroxysmal nocturnal hemoglobinuria: clinical and laboratory studies relating to iron metabolism and therapy with androgen and iron. Medicine (Baltimore) 45: 331–363Google Scholar
  89. [89]
    Firkin F, Goldberg H, Firkin BG (1968) Glucocorticoid management of paroxysmal nocturnal haemoglobinuria. Australas Ann Med 17: 127–134PubMedGoogle Scholar
  90. [90]
    Jacobs P, Wood L (1986) Paroxysmal nocturnal haemoglobinuria and pregnancy. Lancet 2: 1099PubMedGoogle Scholar
  91. [91]
    de Gramont A, Krulik M, Debray J (1987) Paroxysmal nocturnal haemoglobinuria and pregnancy. Lancet 1: 868PubMedGoogle Scholar
  92. [92]
    Beresford CH, Gudex DJ, Symmans WA (1986) Paroxysmal nocturnal haemoglobinuria and pregnancy. Lancet 2: 1396–1397PubMedGoogle Scholar
  93. [93]
    Brodsky RA, Young NS, Antonioli E et al. (2007) Multicenter phase III study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 111(4): 1840–1847PubMedGoogle Scholar
  94. [94]
    Hillmen P, Young NS, Schubert J et al. (2006) The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 355: 1233–1243PubMedGoogle Scholar
  95. [95]
    Ross SC, Densen P (1984) Complement deficiency states and infection: epidemiology, pathogenesis and consequences of neisserial and other infections in an immune deficiency. Medicine (Baltimore) 63: 243–273Google Scholar
  96. [96]
    Hill A, Ridley SH, Esser D et al. (2006) Protection of erythrocytes from human complement-mediated lysis by membrane-targeted recombinant soluble CD59: a new approach to PNH therapy. Blood 107: 2131–2137PubMedGoogle Scholar
  97. [97]
    Fraser DA, Harris CL, Williams AS et al. (2003) Generation of a recombinant, membrane-targeted form of the complement regulator CD59: activity in vitro and in vivo. J Biol Chem 278: 48921–48927PubMedGoogle Scholar
  98. [98]
    Rother RP, Rollins SA, Mennone J et al. (1994) Expression of recombinant transmembrane CD59 in paroxysmal nocturnal hemoglobinuria B cells confers resistance to human complement. Blood 84: 2604–2611PubMedGoogle Scholar
  99. [99]
    Sloand EM, Maciejewski JP, Dunn D et al. (1998) Correction of the PNH defect by GPI-anchored protein transfer. Blood 92: 4439–4445PubMedGoogle Scholar
  100. [100]
    Harris CL, Williams AS, Linton SM, Morgan BP (2002) Coupling complement regulators to immunoglobulin domains generates effective anti-complement reagents with extended half-life in vivo. Clin Exp Immunol 129: 198–207PubMedGoogle Scholar
  101. [101]
    Harris CL, Hughes CE, Williams AS et al. (2003) Generation of anti-complement “prodrugs”: cleavable reagents for specific delivery of complement regulators to disease sites. J Biol Chem 278: 36068–36076PubMedGoogle Scholar
  102. [102]
    Paquette RL, Yoshimura R, Veiseh C, Kunkel L, Gajewski J, Rosen PJ (1997) Clinical characteristics predict response to antithymocyte globulin in paroxysmal nocturnal haemoglobinuria. Br J Haematol 96: 92–97PubMedGoogle Scholar
  103. [103]
    Ebenbichler CF, Wurzner R, Sandhofer AD, Niederwieser D, Dierich MP, Patsch JR (1996) Anti-thymocyte globulin treatment of a patient for paroxysmal nocturnal haemoglobinuriaaplastic anaemia syndrome: complement activation and transient decrease of the PNH clone. Immunobiology 196: 513–521PubMedGoogle Scholar
  104. [104]
    Stoppa AM, Vey N, Sainty D et al. (1996) Correction of aplastic anaemia complicating paroxysmal nocturnal haemoglobinuria: absence of eradication of the PNH clone and dependence of response on cyclosporin A administration. Br J Haematol 93: 42–44PubMedGoogle Scholar
  105. [105]
    van Kamp H, van Imhoff GW, de Wolf JT, Smit JW, Halie MR, Vellenga E (1995) The effect of cyclosporine on haematological parameters in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol 89: 79–82PubMedGoogle Scholar
  106. [106]
    Antin JH, Ginsburg D, Smith BR, Nathan DG, Orkin SH, Rappeport JM (1985) Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria: eradication of the PNH clone and documentation of complete lymphohematopoietic engraftment. Blood 66: 1247–1250PubMedGoogle Scholar
  107. [107]
    Kawahara K, Witherspoon RP, Storb R (1992) Marrow transplantation for paroxysmal nocturnal hemoglobinuria. Am J Hematol 39: 283–288PubMedGoogle Scholar
  108. [108]
    Saso R, Marsh J, Cevreska L et al. (1999) Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br J Haematol 104: 392–396PubMedGoogle Scholar
  109. [109]
    Raiola AM, Van Lint MT, Lamparelli T et al. (2000) Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria. Haematologica 85: 59–62PubMedGoogle Scholar
  110. [110]
    Woodard P, Wang W, Pitts N et al. (2001) Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria. Bone Marrow Transplant 27: 589–592PubMedGoogle Scholar
  111. [111]
    Szer J, Deeg HJ, Witherspoon RP et al. (1984) Long-term survival after marrow transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia. Ann Intern Med 101: 193–195PubMedGoogle Scholar
  112. [112]
    Kolb HJ, Holler E, Bender-Gotze C et al. (1989) Myeloablative conditioning for marrow transplantation in myelodysplastic syndromes and paroxysmal nocturnal haemoglobinuria. Bone Marrow Transplant 4: 29–34PubMedGoogle Scholar
  113. [113]
    Bemba M, Guardiola P, Garderet L et al. (1999) Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria. Br J Haematol 105: 366–368PubMedGoogle Scholar
  114. [114]
    Cho SG, Lim J, Kim Y et al. (2001) Conditioning with high-dose cyclophosphamide may not be sufficient to provide a long-term remission of paroxysmal nocturnal hemoglobinuria following syngeneic peripheral blood stem cell transplantation. Bone Marrow Transplant 28: 987–988PubMedGoogle Scholar
  115. [115]
    Lee JL, Lee JH, Lee JH et al. (2003) Allogeneic hematopoietic cell transplantation for paroxysmal nocturnal hemoglobinuria. Eur J Haematol 71: 114–118PubMedGoogle Scholar
  116. [116]
    Markiewicz M, Wojciechowska M, Wylezol I et al. (2005) First two successful unrelated donor bone marrow transplantations for paroxysmal nocturnal hemoglobinuria in Poland. Ann Transplant 10: 26–30PubMedGoogle Scholar
  117. [117]
    Endo M, Beatty PG, Vreeke TM, Wittwer CT, Singh SP, Parker CJ (1996) Syngeneic bone marrow transplantation without conditioning in a patient with paroxysmal nocturnal hemoglobinuria: in vivo evidence that the mutant stem cells have a survival advantage. Blood 88: 742–750PubMedGoogle Scholar
  118. [118]
    Nafa K, Bessler M, Deeg HJ, Luzzatto L (1998) New somatic mutation in the PIG-A gene emerges at relapse of paroxysmal nocturnal hemoglobinuria. Blood 92: 3422–3427PubMedGoogle Scholar
  119. [119]
    Braren V, Jenkins DE Jr, Phythyon JM, Hartmann RC, Clark DA (1981) Perioperative management of patients with paroxysmal nocturnal hemoglobinuria. Surg Gynecol Obstet 153: 515–520PubMedGoogle Scholar
  120. [120]
    Lightfoot T (2002) Drug-induced hemolytic anemia. Transfus Med Q (American Red Cross, Greater Alleghenies Region) (4)Google Scholar
  121. [121]
    Rodriguez LF, Smolik LM, Zbehlik AJ (1994) Benzocaine-induced methemoglobinemia: report of a severe reaction and review of the literature. Ann Pharmacother 28: 643–649PubMedGoogle Scholar
  122. [122]
    Karim A, Ahmed S, Siddiqui R, Mattana J (2001) Methemoglobinemia complicating topical lidocaine used during endoscopic procedures. Am J Med 111: 150–153PubMedGoogle Scholar
  123. [123]
    Fincher ME, Campbell HT (1989) Methemoglobinemia and hemolytic anemia after phenazopyridine hydrochloride (Pyridium) administration in end-stage renal disease. South Med J 82: 372–374PubMedGoogle Scholar
  124. [124]
    de Torres JP, Strom JA, Jaber BL, Hendra KP (2002) Hemodialysis-associated methemoglobinemia in acute renal failure. Am J Kidney Dis 39: 1307–1309PubMedGoogle Scholar
  125. [125]
    Bjorge L, Ernst P, Haram KO (2003) Paroxysmal nocturnal hemoglobinuria in pregnancy. Acta Obstet Gynecol Scand 82: 1067–1071PubMedGoogle Scholar
  126. [126]
    Frakes JT, Burmeister RE, Giliberti JJ (1976) Pregnancy in a patient with paroxysmal nocturnal hemoglobinuria. Obstet Gynecol 47: 22S–24SPubMedGoogle Scholar
  127. [127]
    Lange JG, Griever GE, Brand A, van Roosmalen J (1998) Paroxysmal nocturnal hemoglobinuria in pregnancy. Ned Tijdschr Geneeskd 142: 2308–2311PubMedGoogle Scholar
  128. [128]
    Solal-Celigny P, Tertian G, Fernandez H et al. (1988) Pregnancy and paroxysmal nocturnal hemoglobinuria. Arch Intern Med 148: 593–595PubMedGoogle Scholar
  129. [129]
    Svigos JM, Norman J (1994) Paroxysmal nocturnal haemoglobinuria and pregnancy. Aust N Z J Obstet Gynaecol 34: 104–106PubMedGoogle Scholar
  130. [130]
    Takagi H, Imai A, Kawabata I, Sumi H, Shiraki S, Tamaya T (1989) A good outcome pregnancy in a patient with paroxysmal nocturnal hemoglobinuria. J Med 20: 163–170PubMedGoogle Scholar
  131. [131]
    Heilmann L, Siekmann U, Ludwig H (1980) Paroxysmal nocturnal haemoglobinuria (PNH) and pregnancy (author’s transl). Geburtshilfe Frauenheilkd 40: 682–687PubMedGoogle Scholar
  132. [132]
    Buisson MP, Quereux C, Palot M, Pignon B, Wahl P (1991) Nocturnal paroxysmal hemoglobinuria disclosed during pregnancy. Apropos of a case. J Gynecol Obstet Biol Reprod (Paris) 20: 83–86Google Scholar
  133. [133]
    Fieni S, Bonfanti L, Gramellini D, Benassi L, Delsignore R (2006) Clinical management of paroxysmal nocturnal hemoglobinuria in pregnancy: a case report and updated review. Obstet Gynecol Surv 61: 593–601PubMedGoogle Scholar
  134. [134]
    Bais J, Pel M, von dem BA, van der LH (1994) Pregnancy and paroxysmal nocturnal hemoglobinuria. Eur J Obstet Gynecol Reprod Biol 53: 211–214PubMedGoogle Scholar
  135. [135]
    Fernandez H, Pons JC, Solal-Celigny P, Tertian G, Tchernia G, Papiernik E (1987) Marchiafava-Micheli syndrome and pregnancy. J Gynecol Obstet Biol Reprod (Paris) 16: 909–913Google Scholar
  136. [136]
    Chen ML, Yu CH, Chang FM, Kuo PL (2006) Paroxysmal nocturnal hemoglobinuria superimposed with preeclampsia. Taiwan J Obstet Gynecol 45: 276–278PubMedGoogle Scholar
  137. [137]
    Imai A, Takagi H, Kawabata I, Sumi H, Shiraki S, Tamaya T (1989) Successful pregnancy in a patient with paroxysmal nocturnal hemoglobinuria: case report. Arch Gynecol Obstet 246: 121–124PubMedGoogle Scholar
  138. [138]
    Blickstein D, Blickstein I (2002) The risk of fetal loss associated with Warfarin anticoagulation. Int J Gynaecol Obstet 78: 221–225PubMedGoogle Scholar
  139. [139]
    Murphy JF, O’Riordan J, Newcombe RG, Coles EC, Pearson JF (1986) Relation of haemoglobin levels in first and second trimesters to outcome of pregnancy. Lancet 1: 992–995PubMedGoogle Scholar
  140. [140]
    Stocche RM, Garcia LV, Klamt JG (2001) Labor analgesia in a patient with paroxysmal nocturnal hemoglobinuria with thrombocytopenia. Reg Anesth Pain Med 26: 79–82PubMedGoogle Scholar
  141. [141]
    Kjaer K, Comerford M, Gadalla F (2004) General anesthesia for cesarean delivery in a patient with paroxysmal nocturnal hemoglobinuria and thrombocytopenia. Anesth Analg 98: 1471–1472, tablePubMedGoogle Scholar

Copyright information

© Springer-Verlag/Wien 2010

Authors and Affiliations

  • Lisa Pleyer
    • 1
  • Richard Greil
    • 1
  1. 1.Universitätsklinik für Innere Medizin IIIParacelsus Medizinische PrivatuniversitätSalzburgAustria

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