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. May 2024 [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. May 2024 [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 Aug 31;348(9027):573-7. 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. 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.Texto completo Resumo
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.Texto completo Resumo
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.Texto completo Resumo
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.Texto completo Resumo
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. Resumo
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.Texto completo Resumo
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.Texto completo Resumo
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. Resumo
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.Texto completo
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. Resumo
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. Resumo
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. Resumo
42. Griffin M, Munir T. Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician’s guide. Ther Adv Hematol. 2017;8(3):119–126. Resumo
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.Texto completo Resumo
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. Resumo
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.Texto completo Resumo
46. Regnier L. Erythropoietin used in renal failure complicated by paroxysmal nocturnal hemoglobinuria. ANNA J. 1989 Dec;16(7):512-3. Resumo
47. Rosse WF, Gutterman LA. The effect of iron therapy in paroxysmal nocturnal hemoglobinuria. Blood. 1970 Nov;36(5):559-65.Texto completo Resumo
48. Rosse WF. Transfusion in paroxysmal nocturnal hemoglobinuria: to wash or not to wash? Transfusion. 1989 Oct;29(8):663-4. Resumo
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. Resumo
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.Texto completo Resumo
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.Texto completo Resumo
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. Resumo
53. Bjorge L, Ernst P, Haram KO. Paroxysmal nocturnal hemoglobinuria in pregnancy. Acta Obstet Gynecol Scand. 2003 Dec;82(12):1067-71. Resumo
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. Resumo
55. Young NS. Paroxysmal nocturnal hemoglobinuria; current issues in pathophysiology and treatment. Curr Hematol Rep. 2005 Mar;4(2):103-9. Resumo
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. Resumo
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.Texto completo Resumo
58. Kawahara K, Witherspoon RP, Storb R. Marrow transplantation for paroxysmal nocturnal hemoglobinuria. Am J Hematol. 1992 Apr;39(4):283-8. Resumo
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.Texto completo Resumo
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. Resumo
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.Texto completo Resumo
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.Texto completo Resumo
O uso deste conteúdo está sujeito ao nosso aviso legal