Paroxysmal nocturnal hemoglobinuria

References

Key articles

Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. ASH Education Book 2016;2016(1):208-16.Full text  Abstract

National Organization for Rare Disorders. NORD rare disease report: paroxysmal nocturnal hemoglobinuria. January 2023 [internet publication].Full text

Dezern AE, Borowitz MJ. ICCS/ESCCA Consensus Guidelines to detect GPI‐deficient cells in Paroxysmal Nocturnal Hemoglobinuria (PNH) and related Disorders Part 1 – Clinical Utility. Cytometry B Clin Cytom. 2018;94(1):16-22.Full text  Abstract

Reference articles

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2. Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. ASH Education Book 2016;2016(1):208-16.Full text  Abstract

3. Cançado RD, Araújo ADS, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021 Jul-Sep;43(3):341-8.Full text  Abstract

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20. Poulou LS, Vakrinos G, Pomoni A, et al. Stroke in paroxysmal nocturnal haemoglobinuria: patterns of disease and outcome. Thromb Haemost. 2007 Sep;98(3):699-701. Abstract

21. Dezern AE, Borowitz MJ. ICCS/ESCCA Consensus Guidelines to detect GPI‐deficient cells in Paroxysmal Nocturnal Hemoglobinuria (PNH) and related Disorders Part 1 – Clinical Utility. Cytometry B Clin Cytom. 2018;94(1):16-22.Full text  Abstract

22. Brodsky RA, Mukhina GL, Li S, et al. Improved detection and characterization of paroxysmal nocturnal hemoglobinuria using fluorescent aerolysin. Am J Clin Pathol. 000 Sep;114(3):459-66.Full text  Abstract

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24. Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005 Dec 1;106(12):3699-709.Full text  Abstract

25. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43.Full text  Abstract

26. Hillmen P, 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 Feb 5;350(6):552-9.Full text  Abstract

27. Martí-Carvajal AJ, Anand V, Cardona AF, et al. Eculizumab for treating patients with paroxysmal nocturnal hemoglobinuria. Cochrane Database Syst Rev. 2014;(10):CD010340.Full text  Abstract

28. Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011 Jun 23;117(25):6786-92.Full text  Abstract

29. Hillmen P, Muus P, Röth A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013 Jul;162(1):62-73.Full text  Abstract

30. Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019 Feb 7;133(6):530-9.Full text  Abstract

31. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021 Mar 18;384(11):1028-37.Full text  Abstract

32. de Latour RP, Szer J, Weitz IC, et al. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. Lancet Haematol. 2022 Sep;9(9):e648-59. Abstract

33. Danilov AV, Smith H, Craigo S, et al. Paroxysmal nocturnal hemoglobinuria (PNH) and pregnancy in the era of eculizumab. Leuk Res. 2009 Jun;33(6):e4-5. Abstract

34. Kelly R, Arnold L, Richards S, et al. The management of pregnancy in paroxysmal nocturnal haemoglobinuria on long term eculizumab. Br J Haematol. 2009 Jun;33(6):e4-5. Abstract

35. Kelly RJ, Höchsmann B, Szer J, et al. Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 015 Sep 10;373(11):1032-9. Abstract

36. Griffin M, Munir T. Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician’s guide. Ther Adv Hematol. 2017;8(3):119–126. Abstract

37. Balleari E, Gatti AM, Mareni C, et al. Recombinant human erythropoietin for long-term treatment of anemia in paroxysmal nocturnal hemoglobinuria. Haematologica. 1996 Mar-Apr;81(2):143-7.Full text  Abstract

38. Bourantas K. High-dose recombinant human erythropoietin and low-dose corticosteroids for treatment of anemia in paroxysmal nocturnal hemoglobinuria. Acta Haematol. 1994;91(2):62-5. Abstract

39. Hill A, Richards SJ, Rother RP, et al. Erythropoietin treatment during complement inhibition with eculizumab in a patient with paroxysmal nocturnal hemoglobinuria. Haematologica. 2007 Mar;92(3):e31-3.Full text  Abstract

40. Regnier L. Erythropoietin used in renal failure complicated by paroxysmal nocturnal hemoglobinuria. ANNA J. 1989 Dec;16(7):512-3. Abstract

41. Rosse WF, Gutterman LA. The effect of iron therapy in paroxysmal nocturnal hemoglobinuria. Blood. 1970 Nov;36(5):559-65.Full text  Abstract

42. Rosse WF. Transfusion in paroxysmal nocturnal hemoglobinuria: to wash or not to wash? Transfusion. 1989 Oct;29(8):663-4. Abstract

43. Kuo GP, Brodsky RA, Kim HS. Catheter-directed thrombolysis and thrombectomy for the Budd-Chiari syndrome in paroxysmal nocturnal hemoglobinuria in three patients. J Vasc Interv Radiol. 2006 Feb;17(2 Pt 1):383-7. Abstract

44. Hillmen P, Muus P, Dührsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8.Full text  Abstract

45. Hall C, Richards S, Hillmen P. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003 Nov 15;102(10):3587-91.Full text  Abstract

46. te Raa GD, Ribbert LS, Snijder RJ, et al. Treatment options in massive pulmonary embolism during pregnancy; a case-report and review of literature. Thromb Res. 2009 May;124(1):1-5. Abstract

47. Bjorge L, Ernst P, Haram KO. Paroxysmal nocturnal hemoglobinuria in pregnancy. Acta Obstet Gynecol Scand. 2003 Dec;82(12):1067-71. Abstract

48. Ray JG, Burows RF, Ginsberg JS, et al. Paroxysmal nocturnal hemoglobinuria and the risk of venous thrombosis: review and recommendations for management of the pregnant and nonpregnant patient. Haemostasis. 2000 May-Jun;30(3):103-17. Abstract

49. Young NS. Paroxysmal nocturnal hemoglobinuria; current issues in pathophysiology and treatment. Curr Hematol Rep. 2005 Mar;4(2):103-9. Abstract

50. Lee JL, Lee JH, Lee JH, et al. Allogeneic hematopoietic cell transplantation for paroxysmal nocturnal hemoglobinuria. Eur J Haematol. 2003 Aug;71(2):114-8. Abstract

51. Antin JH, Ginsburg D, Smith BR, et al. Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria: eradication of the PNH clone and documentation of complete lymphohematopoietic engraftment. Blood. 1985 Dec;66(6):1247-50.Full text  Abstract

52. Kawahara K, Witherspoon RP, Storb R. Marrow transplantation for paroxysmal nocturnal hemoglobinuria. Am J Hematol. 1992 Apr;39(4):283-8. Abstract

53. Peffault de Latour R, Roeth A, Kulasekararaj A, et al. Oral monotherapy with iptacopan, a proximal complement inhibitor of factor B, has superior efficacy to intravenous terminal complement inhibition with standard of care cculizumab or ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: results from the randomized, active-comparator-controlled, open-label, multicenter, phase III apply-PNH study. Blood. 2022;140(suppl 2):LBA-2.Full text

54. Röth A, He G, Brodsky​ A, et al. S181: the phase III, randomized COMMODORE 2 trial: results from a multicenter study of crovalimab vs eculizumab in paroxysmal nocturnal hemoglobinuria (PNH) patients naive to complement inhibitors. Hemasphere. 2023 Aug; 7(suppl): e72750f1.Full text

55. Kulasekararaj AG, Risitano AM, Maciejewski JP, et al. Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood. 2021 Nov 18;138(20):1928-38.Full text  Abstract

56. Senzolo M, Cholongitas EC, Patch D, et al. Update on the classification, assessment of prognosis and therapy of Budd-Chiari syndrome. Nat Clin Pract Gastroenterol Hepatol. 2005 Apr;2(4):182-90.Full text  Abstract

57. Ziakas PD, Poulou LS, Rokas GI, et al. Thrombosis in paroxysmal nocturnal hemoglobinuria: sites, risks, outcome: an overview. J Thromb Haemost. 2007 Mar;5(3):642-5.Full text  Abstract

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