Anemia aplásica em adultos

Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014 Oct 30;124(18):2775-83.Texto completo  Resumo

Bluteau O, Sebert M, Leblanc T, et al. A landscape of germ line mutations in a cohort of inherited bone marrow failure patients. Blood. 2018 Feb 15;131(7):717-32. Resumo

Kordasti S, Costantini B, Seidl T, et al. Deep phenotyping of Tregs identifies an immune signature for idiopathic aplastic anemia and predicts response to treatment. Blood. 2016 Sep 1;128(9):1193-205.Texto completo  Resumo

Killick SB, Bown N, Cavenagh J, et al; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207.Texto completo  Resumo

Bacigalupo A, Socié G, Hamladji RM, et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica. 2015 May;100(5):696-702.Texto completo  Resumo

1. International Agranulocytosis and Aplastic Anemia Study Group. Incidence of aplastic anemia: the relevance of diagnostic criteria. Blood. 1987 Dec;70(6):1718-21. Resumo

2. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19.Texto completo  Resumo

3. Young NS. Acquired aplastic anemia. Ann Intern Med. 2002 Apr 2;136(7):534-46.Texto completo  Resumo

4. Tischkowitz MD, Hodgson SV. Fanconi anaemia. J Med Genet. 2003 Jan;40(1):1-10.Texto completo  Resumo

5. Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014 Oct 30;124(18):2775-83.Texto completo  Resumo

6. Ruggero D, Shimamura A. Marrow failure: a window into ribosome biology. Blood. 2014 Oct 30;124(18):2784-92.Texto completo  Resumo

7. Spinner MA, Sanchez LA, Hsu AP, et al. GATA2 deficiency: a protean disorder of hematopoiesis, lymphatics, and immunity. Blood. 2014 Feb 6;123(6):809-21.Texto completo  Resumo

8. Dickinson RE, Milne P, Jardine L, et al. The evolution of cellular deficiency in GATA2 mutation. Blood. 2014 Feb 6;123(6):863-74.Texto completo  Resumo

9. Vaht K, Göransson M, Carlson K, et al. Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000-2011. Haematologica. 2017 Oct;102(10):1683-90.Texto completo  Resumo

10. Brodsky RA, Jones RJ. Aplastic anaemia. Lancet. 2005 May 7-13;365(9471):1647-56. Resumo

11. Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92.Texto completo  Resumo

12. Kojima S. Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol. 2017 Apr;10(4):277-9.Texto completo  Resumo

13. Liang J, Yagasaki H, Kamachi Y, et al. Mutations in telomerase catalytic protein in Japanese children with aplastic anemia. Haematologica. 2006 May;91(5):656-8.Texto completo  Resumo

14. Marsh JCW, Gutierrez-Rodrigues F, Cooper J, et al. Heterozygous RTEL1 variants in bone marrow failure and myeloid neoplasms. Blood Adv. 2018 Jan 4;2(1):36-48.Texto completo  Resumo

15. Cardoso SR, Ellison ACM, Walne AJ, et al. Myelodysplasia and liver disease extend the spectrum of RTEL1 related telomeropathies. Haematologica. 2017 Aug;102(8):e293-6.Texto completo  Resumo

16. Bluteau O, Sebert M, Leblanc T, et al. A landscape of germ line mutations in a cohort of inherited bone marrow failure patients. Blood. 2018 Feb 15;131(7):717-32. Resumo

17. Dufour C. How I manage patients with Fanconi anaemia. Br J Haematol. 2017 Jul;178(1):32-47. Resumo

18. Avenoso D, Marsh JCW, Potter V, et al. SARS-CoV-2 infection in aplastic anemia. Haematologica. 2022 Feb 1;107(2):541-3.Texto completo  Resumo

19. Kawaguchi T, Nakakuma H. New insights into molecular pathogenesis of bone marrow failure in paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2007 Jul;86(1):27-32. Resumo

20. Ganapathi KA, Townsley DM, Hsu AP, et al. GATA2 deficiency-associated bone marrow disorder differs from idiopathic aplastic anemia. Blood. 2015 Jan 1;125(1):56-70.Texto completo  Resumo

21. Kordasti S, Marsh J, Al-Khan S, et al. Functional characterization of CD4+ T cells in aplastic anemia. Blood. 2012 Mar 1;119(9):2033-43.Texto completo  Resumo

22. Solomou EE, Rezvani K, Mielke S, et al. Deficient CD4+ CD25+ FOXP3+ T regulatory cells in acquired aplastic anemia. Blood. 2007 Sep 1;110(5):1603-6.Texto completo  Resumo

23. de Latour RP, Visconte V, Takaku T, et al. Th17 immune responses contribute to the pathophysiology of aplastic anemia. Blood. 2010 Nov 18;116(20):4175-84.Texto completo  Resumo

24. Shi J, Ge M, Lu S, et al. Intrinsic impairment of CD4(+)CD25(+) regulatory T cells in acquired aplastic anemia. Blood. 2012 Aug 23;120(8):1624-32.Texto completo  Resumo

25. Kordasti S, Costantini B, Seidl T, et al. Deep phenotyping of Tregs identifies an immune signature for idiopathic aplastic anemia and predicts response to treatment. Blood. 2016 Sep 1;128(9):1193-205.Texto completo  Resumo

26. Shimamura A. Inherited bone marrow failure syndromes: molecular features. Hematology Am Soc Hematol Educ Program. 2006:63-71.Texto completo  Resumo

27. Calado RT, Young NS. Telomere diseases. N Engl J Med. 2009 Dec 10;361(24):2353-65.Texto completo  Resumo

28. Choudhry VP, Gupta S, Gupta M, et al. Pregnancy associated aplastic anemia - a series of 10 cases with review of literature. Hematology. 2002 Aug;7(4):233-8. Resumo

29. Antic M, Lautenschlager S, Itin PH. Eosinophilic fasciitis 30 years after - what do we really know? Report of 11 patients and review of the literature. Dermatology. 2006;213(2):93-101. Resumo

30. Killick SB, Bown N, Cavenagh J, et al; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207.Texto completo  Resumo

31. Barrett J, Saunthararajah Y, Molldrem J. Myelodysplastic syndrome and aplastic anemia: distinct entities or diseases linked by a common pathophysiology? Semin Hematol. 2000 Jan;37(1):15-29. Resumo

32. Kulagin A, Lisukov I, Ivanova M, et al. Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two-centre prospective study. Br J Haematol. 2014 Feb;164(4):546-54. Resumo

33. Bono E, McLornan D, Travaglino E, et al. Clinical, histopathological and molecular characterization of hypoplastic myelodysplastic syndrome. Leukemia. 2019 Oct;33(10):2495-505.Texto completo  Resumo

34. Klingemann HG, Storb R, Sanders J, et al. Acute lymphoblastic leukaemia after bone marrow transplantation for aplastic anaemia. Br J Haematol. 1986 May;63(1):47-50. Resumo

35. Samarasinghe S, Veys P, Vora A, et al. Paediatric amendment to adult BSH Guidelines for aplastic anaemia. Br J Haematol. 2018 Jan;180(2):201-5.Texto completo  Resumo

36. Camitta BM, Thomas ED, Nathan DG, et al. Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality. Blood. 1976 Jul;48(1):63-70.Texto completo  Resumo

37. Bacigalupo A, Hows J, Gluckman E, et al. Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA working party. Br J Haematol. 1988 Oct;70(2):177-82. Resumo

38. Marsh J, Schrezenmeier H, Marin P, et al. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999 Apr 1;93(7):2191-5.Texto completo  Resumo

39. Frickhofen N, Rosenfeld SJ. Immunosuppressive treatment of aplastic anemia with antithymocyte globulin and cyclosporine. Semin Hematol. 2000 Jan;37(1):56-68. Resumo

40. Camitta BM. A controlled prospective trial of antithoracic duct lymphocyte globulin (ATDLG) for treatment of severe aplastic anemia. Prog Clin Biol Res. 1984;148:239-47. Resumo

41. Champlin R, Ho W, Gale RP. Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med. 1983 Jan 20;308(3):113-8. Resumo

42. Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. The German Aplastic Anemia Study Group. N Engl J Med. 1991 May 9;324(19):1297-304.Texto completo  Resumo

43. Rosenfeld S, Follmann D, Nuñez O, et al. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003 Mar 5;289(9):1130-5.Texto completo  Resumo

44. Frickhofen N, Heimpel H, Kaltwasser JP, et al; German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003 Feb 15;101(4):1236-42.Texto completo  Resumo

45. Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022 Jan 6;386(1):11-23. Resumo

46. Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017 Apr 20;376(16):1540-50.Texto completo  Resumo

47. European Blood and Marrow Transplant Group, Severe Aplastic Anaemia Working Party. Rabbit ATG for aplastic anaemia treatment: a backward step? Lancet. 2011 Nov 26;378(9806):1831-3. Resumo

48. Bacigalupo A, Locatelli F, Lanino E, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005 Dec;36(11):947-50. Resumo

49. Deeg HJ, O'Donnell M, Tolar J, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006 Sep 1;108(5):1485-91.Texto completo  Resumo

50. Armand P, Antin JH. Allogeneic stem cell transplantation for aplastic anemia. Biol Blood Marrow Transplant. 2007 May;13(5):505-16.Texto completo  Resumo

51. Marsh JC, Gupta V, Lim Z, et al. Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft versus host disease after allogeneic stem cell transplantation for acquired aplastic anemia. Blood. 2011 Aug 25;118(8):2351-7.Texto completo  Resumo

52. Bacigalupo A, Socie G, Lanino E, et al; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA working party. Haematologica. 2010 Jun;95(6):976-82.Texto completo  Resumo

53. Scheinberg P, Nunez O, Weinstein B, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8.Texto completo  Resumo

54. Marsh J, Socie G, Tichelli A, et al, on behalf of the EBMT Severe Aplastic Anaemia (SAA) Working Party. Prospective phase II pilot study of rabbit antithymocyte globulin with ciclosporin for patients with acquired aplastic anaemia and matched pair analysis with patients treated with horse ATG and ciclosporin. EBMT 2011 physicians' abstracts: no.208. Bone Marrow Transplant. 2011;46(Suppl 1):S30.Texto completo

55. Afable MG 2nd, Shaik M, Sugimoto Y, et al. Efficacy of rabbit antithymocyte globulin in severe aplastic anemia. Haematologica. 2011 Sep;96(9):1269-75.Texto completo  Resumo

56. Eapen M. Allogeneic transplantation for aplastic anemia. Hematology. 2012 Apr;17(suppl 1):S15-7. Resumo

57. Olnes MJ, Scheinberg P, Calvo KR, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9.Texto completo  Resumo

58. Desmond R, Townsley DM, Dumitriu B, et al. Eltrombopag restores tri-lineage hematopoiesis in refractory severe aplastic anemia which can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. Resumo

59. Lengline E, Drenou B, Peterlin P, et al. Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Haematologica. 2018 Feb;103(2):212-20.Texto completo  Resumo

60. Jaime-Pérez JC, Colunga-Pedraza PR, Gómez-Ramírez CD, et al. Danazol as first-line therapy for aplastic anemia. Ann Hematol. 2011 May;90(5):523-7. Resumo

61. Townsley DM, Dumitriu B, Liu D, et al. Danazol treatment for telomere diseases. N Engl J Med. 2016 May 19;374(20):1922-31.Texto completo  Resumo

62. Dokal IS. Inherited aplastic anaemia/bone marrow failure syndromes. In: Hoffbrand AV, Catovsky D, Tuddenham EG, et al, eds. Postgraduate haematology. 6th ed. Oxford, UK: Wiley-Blackwell; 2010.

63. Killick SB, Win N, Marsh JC, et al. Pilot study of HLA alloimmunization after transfusion with pre-storage leucodepleted blood products in aplastic anaemia. Br J Haematol. 1997 Jun;97(3):677-84. Resumo

64. Quillen K, Wong E, Scheinberg P, et al. Granulocyte transfusions in severe aplastic anemia: an eleven-year experience. Haematologica. 2009 Dec;94(12):1661-8.Texto completo  Resumo

65. Shah FT, Porter JB, Sadasivam N, et al. Guidelines for the monitoring and management of iron overload in patients with haemoglobinopathies and rare anaemias. Br J Haematol. 2022 Jan;196(2):336-50.Texto completo  Resumo

66. Mao P, Zhu Z, Wang H, et al. Sustained and stable hematopoietic donor-recipient mixed chimerism after unrelated cord blood transplantation for adult patients with severe aplastic anemia. Eur J Haematol. 2005 Nov;75(5):430-5. Resumo

67. Ohga S, Ichino K, Goto K, et al. Unrelated donor cord blood transplantation for childhood severe aplastic anemia after a modified conditioning. Pediatr Transplant. 2006 Jun;10(4):497-500. Resumo

68. Motwani J, Lawson SE, Darbyshire PJ. Successful HSCT using nonradiotherapy-based conditioning regimens and alternative donors in patients with Fanconi anaemia - experience in a single UK centre. Bone Marrow Transplant. 2005 Sep;36(5):405-10. Resumo

69. Vibhakar R, Radhi M, Rumelhart S, et al. Successful unrelated umbilical cord blood transplantation in children with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2005 Nov;36(10):855-61. Resumo

70. Brodsky RA, Luznik L, Bolaños-Meade J, et al. Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases. Bone Marrow Transplant. 2008 Oct;42(8):523-7.Texto completo  Resumo

71. Clay J, Kulasekararaj AG, Potter V, et al. Nonmyeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2014 Nov;20(11):1711-6.Texto completo  Resumo

72. Bacigalupo A, Sica S. Alternative donor transplants for severe aplastic anemia: current experience. Semin Hematol. 2016 Apr;53(2):115-9. Resumo

73. Bacigalupo A, Socié G, Hamladji RM, et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica. 2015 May;100(5):696-702.Texto completo  Resumo

74. Grimaldi F, Potter V, Perez-Abellan P, et al. Mixed T cell chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia using an alemtuzumab-containing regimen is shaped by persistence of recipient CD8 T cells. Biol Blood Marrow Transplant. 2017 Feb;23(2):293-9.Texto completo  Resumo

75. Valdez JM, Scheinberg P, Nunez O, et al. Decreased infection-related mortality and improved survival in severe aplastic anemia in the past two decades. Clin Infect Dis. 2011 Mar 15;52(6):726-35. Resumo

76. Socié G, Rosenfeld S, Frickhofen N, et al. Late clonal diseases of treated aplastic anemia. Semin Hematol. 2000 Jan;37(1):91-101. Resumo

77. Kulasekararaj AG, Jiang J, Smith AE, et al. Somatic mutations identify a subgroup of aplastic anemia patients who progress to myelodysplastic syndrome. Blood. 2014 Oct 23;124(17):2698-704.Texto completo  Resumo

78. Socié G, Henry-Amar M, Bacigalupo A, et al. Malignant tumors occurring after treatment of aplastic anemia. European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party. N Engl J Med. 1993 Oct 14;329(16):1152-7.Texto completo  Resumo

79. Kiem HP, McDonald GB, Myerson D, et al. Marrow transplantation for hepatitis-associated aplastic anemia: a follow-up of long-term survivors. Biol Blood Marrow Transplant. 1996 May;2(2):93-9. Resumo

80. Anasetti C, Doney KC, Storb R, et al. Marrow transplantation for severe aplastic anemia. Long-term outcome in fifty "untransfused" patients. Ann Intern Med. 1986 Apr;104(4):461-6. Resumo

81. Sanders JE, Woolfrey AE, Carpenter PA, et al. Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemia. Blood. 2011 Aug 4;118(5):1421-8.Texto completo  Resumo

82. Eapen M, Ramsay NK, Mertens AC, et al. Late outcomes after bone marrow transplant for aplastic anaemia. Br J Haematol. 2000 Dec;111(3):754-60. Resumo

83. Deeg HJ, Socié G, Schoch G, et al. Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood. 1996 Jan 1;87(1):386-92.Texto completo  Resumo

84. Marsh JC, Zomas A, Hows JM, et al. Avascular necrosis after treatment of aplastic anaemia with antilymphocyte globulin and high-dose methylprednisolone. Br J Haematol. 1993 Aug;84(4):731-5. Resumo