Aplastic anemia in adults

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

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.[Abstract][Full Text]

3. Young NS. Acquired aplastic anemia. Ann Intern Med. 2002 Apr 2;136(7):534-46.[Abstract][Full Text]

4. Tischkowitz MD, Hodgson SV. Fanconi anaemia. J Med Genet. 2003 Jan;40(1):1-10.[Abstract][Full Text]

5. Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014 Oct 30;124(18):2775-83.[Abstract][Full Text]

6. Ruggero D, Shimamura A. Marrow failure: a window into ribosome biology. Blood. 2014 Oct 30;124(18):2784-92.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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

11. Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92.[Abstract][Full Text]

12. Kojima S. Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol. 2017 Apr;10(4):277-9.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract]

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

18. Avenoso D, Marsh JCW, Potter V, et al. SARS-CoV-2 infection in aplastic anemia. Haematologica. 2022 Feb 1;107(2):541-3.[Abstract][Full Text]

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.[Abstract]

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.[Abstract][Full Text]

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. [Abstract][Full Text]

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. [Abstract][Full Text]

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. [Abstract][Full Text]

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.[Abstract][Full Text]

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. [Abstract][Full Text]

26. Shimamura A. Inherited bone marrow failure syndromes: molecular features. Hematology Am Soc Hematol Educ Program. 2006:63-71.[Abstract][Full Text]

27. Calado RT, Young NS. Telomere diseases. N Engl J Med. 2009 Dec 10;361(24):2353-65.[Abstract][Full Text]

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.[Abstract]

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.[Abstract]

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. [Abstract][Full Text]

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.[Abstract]

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.[Abstract]

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract][Full Text]

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

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.[Abstract]

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.[Abstract]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract]

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. [Abstract][Full Text]

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.[Abstract]

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.[Abstract]

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.[Abstract][Full Text]

50. Armand P, Antin JH. Allogeneic stem cell transplantation for aplastic anemia. Biol Blood Marrow Transplant. 2007 May;13(5):505-16.[Abstract][Full Text]

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. [Abstract][Full Text]

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.[Abstract][Full Text]

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. [Abstract][Full Text]

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.[Full Text]

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.[Abstract][Full Text]

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

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract][Full Text]

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. [Abstract]

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. [Abstract][Full Text]

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. [Abstract]

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract]

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.[Abstract]

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.[Abstract]

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. [Abstract][Full Text]

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.[Abstract][Full Text]

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

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. [Abstract][Full Text]

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. [Abstract][Full Text]

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.[Abstract]

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

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.[Abstract][Full Text]

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract]

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.[Abstract][Full Text]

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.[Abstract]

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.[Abstract][Full Text]

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.[Abstract]