The pathophysiology of acquired aplastic anemia

New England Journal of Medicine

Volumn 336, Issue 19, 1997, Pages 1365-1372

  Young, Neal S ^a   Maciejewski, Jaroslaw ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BENZENE; CYTOKINE; ERYTHROPOIETIN; GAMMA INTERFERON; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; HEMOPOIETIC GROWTH FACTOR; IMMUNOSUPPRESSIVE AGENT; INTERLEUKIN 1; INTERLEUKIN 3; LYMPHOCYTE ANTIBODY; STEM CELL FACTOR; THROMBOPOIETIN; THYMOCYTE ANTIBODY; TUMOR NECROSIS FACTOR;

APLASTIC ANEMIA; BONE MARROW DEPRESSION; CANCER CHEMOTHERAPY; GRAFT VERSUS HOST REACTION; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LYMPHOCYTE; NONHUMAN; PANCYTOPENIA; PATHOPHYSIOLOGY; PRIORITY JOURNAL; RADIATION INJURY; REVIEW; STROMA CELL; VIRUS HEPATITIS;

ANEMIA, APLASTIC; CELL DIVISION; HEMATOPOIESIS; HEMATOPOIETIC STEM CELLS; HEPATITIS, VIRAL, HUMAN; HUMANS; IMMUNOSUPPRESSION;

EID: 0030912049     PISSN: 00284793     EISSN: None     Source Type: Journal    
DOI: 10.1056/NEJM199705083361906     Document Type: Review

References (72)

1. Young NS, Alter BP. Aplastic anemia: acquired and inherited. Philadelphia: W.B. Saunders, 1994.
2. Maciejewski JP, Anderson S, Katevas P, Young NS. Phenotypic and functional analysis of bone marrow progenitor cell compartment in bone marrow failure. Br J Haematol 1994;87:227-34.
3. Scopes J, Bagnara M, Gordon-Smith EC, Ball SE, Gibson FM. Haemopoietic progenitor cells are reduced in apiastic anaemia. Br J Haematol 1994;86:427-30.
4. Maciejewski JP, Selleri C, Sato T, Anderson S, Young NS. A severe and consistent deficit in marrow and circulating primitive hematopoietic cells (long-term culture-initiating cells) in acquired aplastic anemia. Blood 1996;88:1983-91.
5. Schrezenmeier H, Jenal M, Herrmann F, Heimpel H, Raghavachar A. Quantitative analysis of cobblestone area-forming ecus in bone marrow of patients with aplastic anemia by limiting dilution assay. Blood 1996;88:4474-80.
6. Marsh JCW, Chang J, Testa NG, Hows JM, Dexter TM. In vitro assessment of marrow 'stem cell' and stromal cell function in aplastic anaemia. Br J Haematol 1991;78:258-67.
7. Novitzky N, Jacobs P. Immunosuppressive therapy in bone marrow aplasia: the stroma functions normally to support hematopoiesis. Exp Hematol 1995;23:1472-7.
8. Kojima S, Matsuyama T, Kodera Y. Hematopoietic growth factors released by marrow stromal cells from patients with aplastic anemia. Blood 1992;79:2256-61.
9. Gibson FM, Scopes J, Daly S, Ball S, Gordon-Smith EC. Haemopoietic growth factor production by normal and aplastic anaemia stroma in longterm bone marrow culture. Br J Haematol 1995;91:551-61.
10. Stark R, Andre C, Thierry D, Cherel M, Galibert F, Gluckman E. The expression of cytokine and cytokine receptor genes in long-term bone marrow culture in congenital and acquired bone marrow hypoplasias. Br J Haematol 1993;83:560-6.
11. Migliaccio AR, Migliaccio G, Adamson JW, Torok-Storb B. Production of granulocyte colony-stimulating factor and granulocyte/macrophage-colony-stimulating factor after interleukin-1 stimulation of marrow stromal cell cultures from normal or aplastic anemia donors. J Cell Physiol 1992;152:199-206.
12. Tani K, Ozawa K, Ogura H, et al. The production of granulocyte colony-stimulating factor and interleukin 6 by human bone marrow stromal cells in aplastic anemia. Tohoku J Exp Med 1993;169:325-34.
13. Das RE, Milne A, Rowley M, Smith EC, Cotes PM. Serum immunoreactive erythropoietin in patients with idiopathic aplastic and Fanconi's anaemias. Br J Haematol 1992;82:601-7.
14. Emmons RVB, Reid DM, Cohen RL, et al. Human thrombopoietin levels are high when thrombocytopcnia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood 1996;87:4068-71.
15. Watari K, Asano S, Shirafuji N, et al. Serum granulocyte colony-stimulating factor levels in healthy volunteers and patients with various disorders as estimated by enzyme immunoassay. Blood 1989;73:117-22.
16. Devetten MP, Young NS. Hematopoietic growth factors in the patho- physiology and treatment of aplastic anemia. In: Hoelzer D, Ganser A, eds. Cytokines in the treatment of hematopoietic failure. New York: Marcel Dekker (in press).
17. Lyman SD, Seaberg M, Hanna R, et al. Plasma/serum levels of flt3 ligand are low in normal individuals and highly elevated in patients with Fanconi anemia and acquired aplastic anemia. Blood 1995;86:4091-6.
18. Wodnar-Filipowicz A, Yancik S, Moser Y, et al. Levels of soluble stem cell factor in serum of patients with aplastic anemia. Blood 1993;81:3259-64.
19. Young NS, Barren AJ. The treatment of severe acquired aplastic anemia. Blood 1995;85:3367-77.
20. Kaufman DW, Kelly JP, Levy M, Shapiro S. The drug etiology of agranidocytosis and aplastic anemia. Vol. 18 of Monographs in epidemiology and biostatistics. New York: Oxford University Press, 1991.
21. Schattenberg DG, Stillman WS, Gruntmeir JJ, Helm KM, Irons RD, Ross D. Peroxidase activity in murine and human hematopoietic progenitor cells: potential relevance to benzene-induced toxicity. Mol Pharmacol 1994;46:346-51.
22. Watanabe KH, Bois FY, Dajsey JM, Auslander DM, Spear RC. Benzene toxicokinetics in humans: exposure of bone marrow to metabolites. Occup Environ Med 1994;51:414-20.
23. Pathak DN, Levay G, Bodell WJ. DNA adduct formation in the bone marrow of B6C3F1 mice treated with benzene. Carcinogenesis 1995;16:1803-8.
24. Mathé G, Amiel JL, Schwarzenberg L, et al. Bone marrow graft in man after conditioning by anrilymphocytic serum. BMJ 1970;2:131-6.
25. Champlin RE, Feig SA, Sparkes RS, Gale RP. Bone marrow transplantation from identical twins in the treatment of aplastic anaemia: implication for the pathogenesis of the disease. Br J Haematol 1984;56:455-63.
26. Speck B, Gluckman E, Haak HL, van Rood JTJ. Treatment of aplastic anaemia by antilymphocyte globulin with and without allogeneic bonemarrow infusions. Lancet 1977;2:1145-8.
27. Champtfn R, Ho W, Gale RP. Antirhymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med 1983;308:113-8.
28. Marmont AM, Bacigalupo A, Van Lint MT, et al. Treatment of severe aplastic anemia with high-dose methylprednisolorie and antilymphocyte globulin. In: Young NS, Levine AS, Humphries RK, eds. Aplastic anemia: stem cell biology and advances in treatment. Vol. 148 of Progress in clinical and biological research. New York: Alan R. Liss, 1984:271-87,
29. Leonard EM, Raefsky E, Griffith P, Kimball J, Nienhuis AW, Young NS. Cyclosporine therapy of aplastic anaemia, congenital and acquired red cell aplasia. Br J Haematol 1989;72:278-84.
30. Brodsky RA, Sensenbrenner LL, Jones RJ. Complete remission in severe aplastic anemia after high-dose cyclophosphamide without bone marrow transplantation. Blood 1996;87:491-4.
31. Bacigalupo A, Broccia G, Corda G, et al. Antilymphocyte globulin, cyclosporin, and granulocyte colony-stimulating factor in patients with acquired severe aplastic anemia (SAA): a pilot study of the EBMT SAA Working Party. Blood 1995;85:1348-53.
32. Rosenfeld SJ, Kimball J, Vining D, Young NS. Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood 1995;85:3058-65.
33. Sato T, Selleri C, Young NS, Maciejewski JP. Hematopoietic inhibition by interferon-γ is partially mediated through Interferon regulatory factor1. Blood 1995;86:3373-80.
34. Maciejewski JP, Selleri C, Sato T, et al. Nitric oxide suppression of human hematopoiesis in vitro: contribution to inhibitory action of interferonγ and tumor necrosis factor-α. J Clin Invest 1995;96:1085-92.
35. Kagan WA, Ascensao JA, Pahwa RN, et al. Aplastic anemia: presence in human bone marrow of cells that suppress myeJopoiesis, Proc Natl Acad Sci U S A 1976;73:2890-4.
36. Zoumbos NC, Gascon P, Djeu JY, Young NS. Interferon is a mediator of hematopoietk: suppression in aplastic anemia in vitro and possibly in vivo. Proc Natl Acad Sci U S A 1985;82:188-92.
37. Tong J, Bacigalupo A, Piaggio G, Figari O, Sogno G, Marmont A. In vitro response of T cells from aplastic anemia patients to antilymphocyte globulin and phytohemagglutinin: colony-stimulating activity and lymphokine production. Exp Hematol 1991;19:312-6.
38. Selleri C, Sato T, Anderson S, Young NS, Maciejewski JP. Interferonγ and tumor necrosis factor-α suppress both early and late stages of hematopoiesis and induce programmed cell death. J Cell Physiol 1995;165:538-46.
39. Nakao S, Yamaguchi M, Shiobara S, et al. Interferon-gamma gene expression in unstimulatcd bone marrow mononuclear cells predicts a good response to cyclosporine therapy in aplastic anemia. Blood 1992;79:2532-5.
40. Nistico A, Young NS. γ-Interferon gene expression in the bone marrow of patients with aplastic anemia. Ann Intern Med 1994;120:463-9.
41. Maciejewski JP, Hibbs JR, Anderson S, Katevas P, Young NS. Bone marrow and peripheral blood lymphocyte phenotype in patients with bone marrow failure. Exp Hematol 1994;22:1102-10.
42. Platanias L, Gascon P, Bielory L, Griffith P, Nienhuis A, Young N. Lymphocyte phenotype and lymphokines following anri-rbymocyte globulin therapy in patients with aplastic anaemia. Br J Haematol 1987;66:437-43.
43. Laver J, Castro-Malaspina H, Kernan NA, et al. In vitro interferongamma production by cultured T-cells in severe aplastic anaemia: correlation with granulomonopoietic inhibition in patients who respond to antithymocyte globulin. Br J Haematol 1988;69:545-50.
44. Seileri C, Madejewski JP, Sato T, Young NS. Interferon-γ constitutively expressed in the stromal microenvironment of human marrow cultures mediates potent hematopoietic inhibition. Blood 1996;87:4149-57
45. Maciejewski JP, Selleri C, Anderson S, Young NS. Fas antigen expression on CD34+ human marrow cells is induced by interteron gamma and tumor necrosis factor alpha and potentiates cytokine-mediated hematopoietic suppression in vitro. Blood 1995;85:3183-90.
46. Nagafuji K, Shibuya T, Harada M, et al. Functional expression of Fas antigen (CD95) on hematopoietic progenitor cells. Blood 1995;86:883-9.
47. Maciejewski JP, Selleri C, Sato T, Anderson S, Young NS. Increased expression of Fas antigen on bone marrow CD34+ cells of patients with aplastic anaemia. Br J Haematol 1995;91:245-52.
48. Philpott NJ, Scopes J, Marsh JCW, Gordon-Smith EC, Gibson FM. Increased apoptosis in aplastic anemia bone marrow progenitor cells: possible pathophysiologic significance. Exp Hematol 1995;23:1642-8.
49. Nimer SD, Ireland P, Meshkinpour A, Franc M. An increased HLA DR2 frequency is seen in aplastic anemia patients. Blood 1994;84:923-7.
50. Nakao S, Takamatsu H, Chuhjo T, et al. Identification of a specific HLA class II haplotype strongly associated with susceptibility to cyclosporine-dependent aplastic anemia. Blood 1994;84:4257-61.
51. Brown KE, Tisdale J, Dunbar CE, Barrett AJ, Young NS. Hepatitis-associated aplastic anemia. N Engl J Med 1997;336:1059-64.
52. Moebius U, Herrmann F, Hercend T, Meuer SC. Clonal analysis of CD4+/CD8+ T cells in a patient with aplastic anemia. J Clin Invest 1991;87:1567-74.
53. Nakao S, Takamatsu H, Yachie A, et al. Establishment of a CD4+ T cell clone recognizing autologous hematopoietic progenitor cells from a patient with immune-mediated aplastic anemia. Exp Hematof 1995;23:433-8.
54. Anderson KC, Weinstein HJ. Transfusion-associated graft-versus-host disease. N Engl J Med 1990;323:315-21. [Erratum, N Engl J Med 1990;323:1360.]
55. Tzakis AG, Arditi M, Whitington PF, et al. Aplastic anemia complicating orthotopic liver transplantation for non-A, non-B hepatitis. N Engl J Med 1988;319:393-6.
56. Issaragrisil S, Kaufman DW, Anderson TE, et al. An association of aplastic anaemia in Thailand with low socioeconomic status. Br J Haematol 1995-91:80-4.
57. Issaragrisil S, Chansung K, Kauftnan D, et al. Aplastic anemia in Thailand and occupational exposures: associations with grain farming and solvents. Am J Epidemiol (in press).
58. Issaragrisil S, Kaufman DW, Young NS. The epidemiology of acquired aplastic anemia (AA) in Thailand. Blood 1995;86:Suppl l:478a. abstract.
59. Issaragrisil S, Sriratanasatavorn C, Piankijagurn A, et al. Incidence of aplastic anemia in Bangkok. Blood 1991;77:2166-8.
60. Bacigalupo A, Aetiology of severe aplastic anaemia and outcome after allogeneic bone marrow transplantation or immunosuppression therapy. Eut J Haematol 1996;57:Suppl 60:16-9.
61. Corzo D, Yunis JJ, Salazar M, et al. The major histocompatibility complex region marked by HSP70-1 and HSP70-2 variants is associated with clozapine-induced agranulocytosis in two different ethnic groups. Blood 1995;86:3835-40.
62. Tamai H, Sudo T, Kirnura A, et al. Association between the DRBI * 08032 histocompatibility antigen and methimazole-induced agranulocytosis in Japanese patients with Graves disease. Ann Intern Med 1996;124:490-4.
63. de PJanque MM, Bacigalupo A, Würsch A, et al. Long-term follow-up of severe aplastic anaemia patients treated with antithymocyte globulin. Br J Haematol 1989;73:121-6.
64. Socié G, Henry-Amar M, Bacigalupo A, et al. Malignant tumors occurring after treatment of apfastic anemia. N Engl J Med 1993;329:1152-7.
65. Young NS. The problem of clonality in aplastic anemia: Dr. Damcshek's riddle, restated. Blood 1992;79;1385-92.
66. Molldrem J, Stetler-Stevenson M, Mavroudis D, Young NS, Barrett AJ. Antithymocyte globulin (ATG) abrogates cytopenias in patients with myelodysplastic syndrome. Blood 1996;88:Suppl l:454a. abstract.
67. White JR, Josten KM, Chopra R, et al. Absence of N-RAS point mutations in peripheral blood cells of patients with aplastic anaemia and paroxysmal nocturnal haemoglobinurea. Br J Haematol 1995;91:921-3.
68. Kinoshita T, Inoue N, Takeda J. Role of phosphatidylinositol-linked proteins in paroxysmal nocturnal hemoglobinuria pathogenesis. Annu Rev Med 1996;47:1-10.
69. Kawagoe K, Kitamura D, Okabe M, et al. Glycosylphosphatidylinositol-anchor-defkicnt mice: implications for clonal dominance of mutant cells in paroxysmal nocturnal hemoglobinuria. Blood 1996;87:3600-6.
70. - hematopoiesis is reconstituted following intercellular transfer of GPI-anchored proteins. Proc Natl Acad Sci U S A 1996;93:7938-43.
71. Bessler M, Mason P, Hillmen P, Luzzatto L. Somatic mutations and cellular selection in paroxysmal nocturnal haemoglobinuria. Lancet 1994;343:951-3.
72. -, phosphatidylinositolglycan-anchor-deficient T lymphocytes after in vivo application of Campath-1 H for refractory B-cell non-Hodgkin lymphoma. Blood 1995;86:1487-92.