Aplastic anaemia in adults

Kulasekararaj A, Cavenagh J, Dokal I, et al. Guidelines for the diagnosis and management of adult aplastic anaemia: a British Society for Haematology Guideline. Br J Haematol. 2024 Mar;204(3):784-804.Full text  Abstract

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

1. Kulasekararaj A, Cavenagh J, Dokal I, et al. Guidelines for the diagnosis and management of adult aplastic anaemia: a British Society for Haematology Guideline. Br J Haematol. 2024 Mar;204(3):784-804.Full text  Abstract

2. Furlong E, Carter T. Aplastic anaemia: current concepts in diagnosis and management. J Paediatr Child Health. 2020 Jul;56(7):1023-8. Abstract

3. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19.Full text  Abstract

4. Young NS. Acquired aplastic anemia. Ann Intern Med. 2002 Apr 2;136(7):534-46. Abstract

5. Gonnot M, Neumann F, Huet F, et al. Hepatitis-associated aplastic anemia. J Pediatr Gastroenterol Nutr. 2022 Nov 1;75(5):553-5. Abstract

6. Jaime-Pérez JC, González-Treviño M, Gómez-Almaguer D. Pregnancy-associated aplastic anemia: a case-based review. Expert Rev Hematol. 2021 Feb;14(2):175-84. Abstract

7. Moreno OM, Paredes AC, Suarez-Obando F, et al. An update on fanconi anemia: clinical, cytogenetic and molecular approaches (review). Biomed Rep. 2021 Sep;15(3):74.Full text  Abstract

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

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

10. 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.Full text  Abstract

11. Dickinson RE, Milne P, Jardine L, et al. The evolution of cellular deficiency in GATA2 mutation. Blood. 2014 Feb 6;123(6):863-74.Full text  Abstract

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

13. 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.Full text  Abstract

14. Montané E, Ibáñez L, Vidal X, et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica. 2008 Apr;93(4):518-23.Full text  Abstract

15. Vallejo C, Rosell A, Xicoy B, et al. A multicentre ambispective observational study into the incidence and clinical management of aplastic anaemia in Spain (IMAS study). Ann Hematol. 2024 Mar;103(3):705-13. Abstract

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

17. Norasetthada L, Wongkhantee S, Chaipokam J, et al. Adult aplastic anemia in Thailand: incidence and treatment outcome from a prospective nationwide population-based study. Ann Hematol. 2021 Oct;100(10):2443-52.Full text  Abstract

18. Li SS, Hsu YT, Chang C, et al. Incidence and treatment outcome of aplastic anemia in Taiwan-real-world data from single-institute experience and a nationwide population-based database. Ann Hematol. 2019 Jan;98(1):29-39. Abstract

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

20. 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.Full text  Abstract

21. 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.Full text  Abstract

22. 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.Full text  Abstract

23. 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

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

25. Park M. Overview of inherited bone marrow failure syndromes. Blood Res. 2022 Apr 30;57(s1):49-54.Full text  Abstract

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

27. DeZern AE, Churpek JE. Approach to the diagnosis of aplastic anemia. Blood Adv. 2021 Jun 22;5(12):2660-71.Full text  Abstract

28. Niraj J, Färkkilä A, D'Andrea AD. The fanconi anemia pathway in cancer. Annu Rev Cancer Biol. 2019 Mar;3:457-78.Full text  Abstract

29. Tummala H, Walne A, Dokal I. The biology and management of dyskeratosis congenita and related disorders of telomeres. Expert Rev Hematol. 2022 Aug;15(8):685-96.Full text  Abstract

30. Nelson AS, Myers KC. Diagnosis, treatment, and molecular pathology of Shwachman-Diamond syndrome. Hematol Oncol Clin North Am. 2018 Aug;32(4):687-700. Abstract

31. 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.Full text  Abstract

32. 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.Full text  Abstract

33. 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.Full text  Abstract

34. 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.Full text  Abstract

35. 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.Full text  Abstract

36. 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.Full text  Abstract

37. Giudice V, Selleri C. Aplastic anemia: pathophysiology. Semin Hematol. 2022 Jan;59(1):13-20. Abstract

38. Shimamura A. Inherited bone marrow failure syndromes: molecular features. Hematology Am Soc Hematol Educ Program. 2006:63-71.Full text  Abstract

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

40. Locasciulli A, Bacigalupo A, Bruno B, et al. Hepatitis-associated aplastic anaemia: epidemiology and treatment results obtained in Europe. A report of The EBMT aplastic anaemia working party. Br J Haematol. 2010 Jun;149(6):890-5. Abstract

41. Anderson E, Shah B, Davidson A, et al. Lessons learned from bone marrow failure in systemic lupus erythematosus: case reports and review of the literature. Semin Arthritis Rheum. 2018 Aug;48(1):90-104.Full text  Abstract

42. Gendron N, de Fontbrune FS, Guyard A, et al. Aplastic anemia related to thymoma: a survey on behalf of the French reference center of aplastic anemia and a review of the literature. Haematologica. 2020 Jul;105(7):e333-6.Full text  Abstract

43. 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

44. Salmeron G, Patey N, de Latour RP, et al. Coeliac disease and aplastic anaemia: a specific entity? Br J Haematol. 2009 Jun;146(1):122-4.Full text

45. Arber DA, Orazi A, Hasserjian RP, et al. International Consensus Classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022 Sep 15;140(11):1200-28.Full text  Abstract

46. 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

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

48. Babushok DV, DeZern AE, de Castro CM, et al. Modified Delphi panel consensus recommendations for management of severe aplastic anemia. Blood Adv. 2024 Aug 13;8(15):3946-60.Full text  Abstract

49. Li J, Li X, Cai L, et al. Prognostic value of pre-treatment PNH clone among the patients with aplastic anemia: a meta-analysis. Hematology. 2023 Dec;28(1):2204617.Full text  Abstract

50. 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

51. 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.Full text  Abstract

52. 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

53. Fattizzo B, Gurnari C, Cassanello G, et al. Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study. Leukemia. 2023 Dec;37(12):2479-85.Full text  Abstract

54. Calado RT, Clé DV. Treatment of inherited bone marrow failure syndromes beyond transplantation. Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):96-101.Full text  Abstract

55. 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.Full text  Abstract

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

57. 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

58. 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

59. 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.Full text  Abstract

60. 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.Full text  Abstract

61. 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.Full text  Abstract

62. Fan X, Desmond R, Winkler T, et al. Eltrombopag for patients with moderate aplastic anemia or uni-lineage cytopenias. Blood Adv. 2020 Apr 28;4(8):1700-10.Full text  Abstract

63. Bejanyan N, Kim S, Hebert KM, et al. Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Adv. 2019 Oct 22;3(20):3123-31.Full text  Abstract

64. 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

65. 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.Full text  Abstract

66. 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

67. Scheinberg P, Finelli C, Montaňo-Figueroa EH, et al. Activity and safety of eltrombopag in combination with cyclosporin A as first‑line treatment of adults with severe aplastic anaemia (SOAR): a phase 2, single-arm study. Lancet Haematol. 2024 Mar;11(3):e206-15. Abstract

68. 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

69. 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.Full text  Abstract

70. Iftikhar R, Chaudhry QUN, Anwer F, et al. Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies. Blood Rev. 2021 May;47:100772. Abstract

71. 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.Full text  Abstract

72. 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.Full text  Abstract

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

74. 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.Full text  Abstract

75. 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

76. 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.Full text  Abstract

77. Marsh JC, Kulasekararaj AG. Management of the refractory aplastic anemia patient: what are the options? Blood. 2013 Nov 21;122(22):3561-7.Full text  Abstract

78. Pagliuca S, Kulasekararaj AG, Eikema DJ, et al. Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. 2024 Mar 1;109(3):765-76.Full text  Abstract

79. 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

80. Townsley DM, Dumitriu B, Liu D, et al. Danazol treatment for telomere diseases. N Engl J Med. 2016 May 19;374(20):1922-31.Full text  Abstract

81. 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

82. 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.Full text  Abstract

83. 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.Full text  Abstract

84. 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.Full text  Abstract

85. 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

86. 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.Full text  Abstract

87. Vaht K, Göransson M, Carlson K, et al. Low response rate to ATG-based immunosuppressive therapy in very severe aplastic anaemia - a Swedish nationwide cohort study. Eur J Haematol. 2018 Jun;100(6):613-20. Abstract

88. Peffault de Latour R, Tabrizi R, Marcais A, et al. Nationwide survey on the use of horse antithymocyte globulins (ATGAM) in patients with acquired aplastic anemia: a report on behalf of the French Reference Center for aplastic anemia. Am J Hematol. 2018 May;93(5):635-42.Full text  Abstract

89. Scheinberg P, Rios O, Scheinberg P, et al. Prolonged cyclosporine administration after antithymocyte globulin delays but does not prevent relapse in severe aplastic anemia. Am J Hematol. 2014 Jun;89(6):571-4.Full text  Abstract

90. 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

91. 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

92. 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

93. 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

94. Childs RW, Tian X, Vo P, et al. Combined haploidentical and cord blood transplantation for refractory severe aplastic anaemia and hypoplastic myelodysplastic syndrome. Br J Haematol. 2021 Jun;193(5):951-60.Full text  Abstract

95. McCurdy SR, Luznik L. How we perform haploidentical stem cell transplantation with posttransplant cyclophosphamide. Blood. 2019 Nov 21;134(21):1802-1810.Full text  Abstract

96. ElGohary G, El Fakih R, de Latour R, et al. Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant. 2020 Oct;55(10):1906-17. Abstract

97. DeZern AE, Zahurak ML, Symons HJ, et al. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 2020 Apr 28;4(8):1770-9.Full text  Abstract

98. Prata PH, Eikema DJ, Afansyev B, et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. Bone Marrow Transplant. 2020 Jun;55(6):1050-8. Abstract

99. DeZern AE, Eapen M, Wu J, et al. Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial. Lancet Haematol. 2022 Sep;9(9):e660-9.Full text  Abstract

100. DeZern AE, Zahurak M, Symons HJ, et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023 Jun 22;141(25):3031-8.Full text  Abstract

101. Montoro J, Eikema DJ, Tuffnell J, et al. Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT. Blood. 2024 Jul 18;144(3):323-3.Full text  Abstract

102. Patel BJ, Barot SV, Kuzmanovic T, et al. Distinctive and common features of moderate aplastic anaemia. Br J Haematol. 2020 Jun;189(5):967-75.Full text  Abstract

103. Nakamura R, Patel BA, Kim S, et al. Conditional survival and standardized mortality ratios of patients with severe aplastic anemia surviving at least one year after hematopoietic cell transplantation or immunosuppressive therapy. Haematologica. 2023 Dec 1;108(12):3298-307.Full text  Abstract

104. 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.Full text  Abstract

105. 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.Full text  Abstract

106. Alotaibi H, Aljurf M, de Latour R, et al. Upfront alternative donor transplant versus immunosuppressive therapy in patients with severe aplastic anemia who lack a fully HLA-matched related donor: systematic review and meta-analysis of retrospective studies, on behalf of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Transplant Cell Ther. 2022 Feb;28(2):105.e1-7.Full text  Abstract

107. 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

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

109. 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.Full text  Abstract

110. 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.Full text  Abstract

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

112. Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-56.Full text

113. Piekarska A, Pawelec K, Szmigielska-Kapłon A, et al. The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults. Front Immunol. 2024;15:1378432.Full text  Abstract

114. 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.Full text  Abstract

115. 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

116. 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.Full text  Abstract

117. 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