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. May 2024 [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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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. Kulasekararaj A, Lanza F, Arvanitakis A, et al. Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2024 Nov;99(11):2108-17.Full text  Abstract

31. Jang JH, Gomez RD, Bumbea H, et al. A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria. EJHaem. 2022 Dec 20;4(1):26-36.Full text  Abstract

32. 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

33. 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

34. 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

35. Wong R, Fishman J, Wilson K, et al. Comparative effectiveness of pegcetacoplan versus ravulizumab and eculizumab in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria: a matching-adjusted indirect comparison. Adv Ther. 2023 Apr;40(4):1571-89.Full text  Abstract

36. Peffault de Latour R, Röth A, Kulasekararaj AG, et al. Oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2024 Mar 14;390(11):994-1008.Full text  Abstract

37. Notaro R, Luzzatto L. Breakthrough hemolysis in PNH with proximal or terminal complement inhibition. N Engl J Med. 2022 Jul 14;387(2):160-6. Abstract

38. Dingli D, De Castro C III, Koprivnikar J, et al. Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria. Hematology. 2024 Mar 21;29(1):2329030.Full text

39. 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

40. 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

41. Kelly RJ, Höchsmann B, Szer J, et al. Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. Abstract

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

43. 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

44. 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

45. 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

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

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

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

49. 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

50. 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

51. 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

52. 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

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

54. 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

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

56. 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

57. 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

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

59. Röth A, He G, Tong H, et al. Phase 3 randomized COMMODORE 2 trial: crovalimab versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria naive to complement inhibition. Am J Hematol. 2024 Sep;99(9):1768-77.Full text  Abstract

60. Lee JW, Griffin M, Kim JS, et al. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023 Dec;10(12):e955-65. Abstract

61. 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

62. 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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