Hemoglobinúria paroxística noturna

Referências

Principais artigos

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

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

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.Texto completo  Resumo

Artigos de referência

1. Rosse W. Paroxysmal nocturnal hemoglobinuria as a molecular disease. Medicine (Baltimore). 1997 Mar;76(2):63-93. Resumo

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

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.Texto completo  Resumo

4. 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 May 21;73(4):703-11. Resumo

5. Young NS. The problem of clonality in aplastic anemia: Dr. Dameshek's riddle, restated. Blood. 1992 Mar 15;79(6):1385-92.Texto completo  Resumo

6. Hall SE, Rosse WF. The use of monoclonal antibodies and flow cytometry in the diagnosis of paroxysmal nocturnal hemoglobinuria. Blood. 1996 Jun 15;87(12):5332-40.Texto completo  Resumo

7. Bat T, Abdelhamid ON, Balasubramanian SK, et al. The evolution of paroxysmal nocturnal haemoglobinuria depends on intensity of immunosuppressive therapy. Br J Haematol. 2018 Sep;182(5):730-3.Texto completo  Resumo

8. Sugimori C, Mochizuki K, Qi Z, et al. Origin and fate of blood cells deficient in glycosylphosphatidylinositol-anchored protein among patients with bone marrow failure. Br J Haematol. 2009;147(1):102-12.Texto completo  Resumo

9. Rother R, Bell L, Hillmen P, et al. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA. 2005 Apr 6;293(13):1653-62.Texto completo  Resumo

10. Clark DA, Butler SA, Braren V, et al. The kidneys in paroxysmal nocturnal hemoglobinuria. Blood. 1981 Jan;57(1):83-9.Texto completo  Resumo

11. Hillmen P, Lewis SM, Bessler M, et al. Natural history of paroxysmal nocturnal haemoglobinuria. New Engl J Med. 1995 Nov 9;333(19):1253-8.Texto completo  Resumo

12. Tichelli A, Gratwohl A, Wursch A, et al. Late haematological complications in severe aplastic anemia. Br J Haematol. 1988 Jul;69(3):413-8. Resumo

13. Fattizzo B, Ireland R, Dunlop A, et al. Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia. Leukemia. 2021 Nov;35(11):3223-31.Texto completo  Resumo

14. Young NS, Maciejewski JP, Sloand E. The enigma of the aplastic anemia/PNH syndrome. In: Omine M, Kinoshita T, eds. Paroxysmal nocturnal hemoglobinuria and related disorders: molecular aspects of pathogenesis. Tokyo, Japan: Springer; 2003:149.

15. de Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008 Oct 15;112(8):3099-106.Texto completo  Resumo

16. Wang SA, Pozdnyakova O, Jorgensen JL, et al. Detection of paroxysmal nocturnal hemoglobinuria clones in patients with myelodysplastic syndromes and related bone marrow diseases, with emphasis on diagnostic pitfalls and caveats. Haematologica. 2009 Jan;94(1):29-37.Texto completo  Resumo

17. Okamoto M, Shichishima T, Noji H, et al. High frequency of several PIG-A mutations in patients with aplastic anemia and myelodysplastic syndrome. Leukemia. 2006 Apr;20(4):627-34. Resumo

18. Nishimura J, 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 May;83(3):193-207. Resumo

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

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. Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

23. Socie G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long term follow-up and prognostic factors. Lancet. 1996;348:573-577. Resumo

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

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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. Resumo

33. Danilov AV, Smith H, Craigo S, et al. Paroxysmal nocturnal hemoglobinuria (PNH) and pregnancy in the era of eculizumab. Leuk Res. 22009 Jun;33(6):e4-5. Resumo

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. Resumo

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. Resumo

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

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.Texto completo  Resumo

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. Resumo

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.Texto completo  Resumo

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

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

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

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. Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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. Resumo

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

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. Resumo

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

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. Resumo

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.Texto completo  Resumo

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

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.Texto completo

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.Texto completo

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.Texto completo  Resumo

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.Texto completo  Resumo

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.Texto completo  Resumo

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