Immunosuppression for myelodysplastic syndrome: How bench to bedside to bench research led to success

Hematology/Oncology Clinics of North America

Volumn 24, Issue 2, 2010, Pages 331-341

  Sloand, Elaine M ^a   Barrett, A J ^a  

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

Author keywords

IFN ; Immunosuppression; Myelodysplastic syndrome; Tumor necrosis factor

Indexed keywords

CYCLOSPORIN A; FAS LIGAND; FLICE INHIBITORY PROTEIN; GAMMA INTERFERON; HLA DR15 ANTIGEN; LENALIDOMIDE; METHYLPREDNISOLONE; OXYMETHOLONE; PROTEIN BCL 2; SURVIVIN; T LYMPHOCYTE RECEPTOR; THALIDOMIDE; THYMOCYTE ANTIBODY; TUMOR NECROSIS FACTOR ALPHA; TUMOR NECROSIS FACTOR ALPHA ANTIBODY; TUMOR NECROSIS FACTOR ALPHA RECEPTOR; TUMOR NECROSIS FACTOR RELATED APOPTOSIS INDUCING LIGAND; TUMOR NECROSIS FACTOR RELATED APOPTOSIS INDUCING LIGAND RECEPTOR 3; WT1 PROTEIN;

APLASTIC ANEMIA; APOPTOSIS; CD4+ CD25+ T LYMPHOCYTE; CLINICAL TRIAL; CYTOKINE PRODUCTION; CYTOPENIA; CYTOTOXIC T LYMPHOCYTE; DISEASE ASSOCIATION; DOWN REGULATION; DRUG DOSAGE FORM COMPARISON; DRUG RESPONSE; HEMATOPOIESIS; HUMAN; IMMUNE DYSREGULATION; IMMUNOSUPPRESSIVE TREATMENT; KIDNEY FAILURE; LUPUS VULGARIS; MYELODYSPLASTIC SYNDROME; NEUROLOGIC DISEASE; PRIORITY JOURNAL; RAYNAUD PHENOMENON; REFRACTORY ANEMIA; REVIEW; RHEUMATIC POLYMYALGIA; RHEUMATOID ARTHRITIS; TRISOMY 8; UNSPECIFIED SIDE EFFECT; UPREGULATION;

ADULT; AGED; AGED, 80 AND OVER; ANTILYMPHOCYTE SERUM; APOPTOSIS; BIOMEDICAL RESEARCH; BONE MARROW; CLINICAL MEDICINE; CLINICAL TRIALS AS TOPIC; CYCLOSPORINE; CYTOKINES; FEMALE; HUMANS; IMMUNOSUPPRESSION; IMMUNOSUPPRESSIVE AGENTS; MALE; MIDDLE AGED; MYELODYSPLASTIC SYNDROMES; T-LYMPHOCYTES; TUMOR NECROSIS FACTOR-ALPHA;

EID: 77950527409     PISSN: 08898588     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.hoc.2010.02.009     Document Type: Review

References (66)

1. Allampallam K., Shetty V., Mundle S., et al. Biological significance of proliferation, apoptosis, cytokines, and monocyte/macrophage cells in bone marrow biopsies of 145 patients with myelodysplastic syndrome. Int J Hematol 2002, 75:289-297.
2. Allampallam K., Shetty V.T., Raza A. Cytokines and MDS. Cancer Treat Res 2001, 108:93-100.
3. Kitagawa M., Saito I., Kuwata T., et al. Overexpression of tumor necrosis factor (TNF)-alpha and interferon (IFN)-gamma by bone marrow cells from patients with myelodysplastic syndromes. Leukemia 1997, 11:2049-2054.
4. Koike M., Ishiyama T., Tomoyasu S., et al. Spontaneous cytokine overproduction by peripheral blood mononuclear cells from patients with myelodysplastic syndromes and aplastic anemia. Leuk Res 1995, 19:639-644.
5. Zang D.Y., Goodwin R.G., Loken M.R., et al. Expression of tumor necrosis factor-related apoptosis-inducing ligand, Apo2L, and its receptors in myelodysplastic syndrome: effects on in vitro hemopoiesis. Blood 2001, 98:3058-3065.
6. Deeg H.J., Gotlib J., Beckham C., et al. Soluble TNF receptor fusion protein (etanercept) for the treatment of myelodysplastic syndrome: a pilot study. Leukemia 2002, 16:162-164.
7. Bouscary D., De Vos J., Guesnu M., et al. Fas/Apo-1 (CD95) expression and apoptosis in patients with myelodysplastic syndromes. Leukemia 1997, 11:839-845.
8. Kitagawa M., Yamaguchi S., Takahashi M., et al. Localization of Fas and Fas ligand in bone marrow cells demonstrating myelodysplasia. Leukemia 1998, 12:486-492.
9. Lepelley P., Grardel N., Emy O., et al. Fas/APO-1 (CD95) expression in myelodysplastic syndromes. Leuk Lymphoma 1998, 30:307-312.
10. Gupta P., Niehans G.A., LeRoy S.C., et al. Fas ligand expression in the bone marrow in myelodysplastic syndromes correlates with FAB subtype and anemia, and predicts survival. Leukemia 1999, 13:44-53.
11. Claessens Y.E., Bouscary D., Dupont J.M., et al. In vitro proliferation and differentiation of erythroid progenitors from patients with myelodysplastic syndromes: evidence for Fas-dependent apoptosis. Blood 2002, 99:1594-1601.
12. Mathe G., Schwarzenberg L. Treatment of bone marrow aplasia by mismatched bone marrow transplantation after conditioning with antilymphocyte globulin-long-term results. Transplant Proc 1976, 8:595-602.
13. Gluckman E., Devergie A., Poros A., et al. Results of immunosuppression in 170 cases of severe aplastic anaemia. Report of the European Group of bone marrow transplant (EGBMT). Br J Haematol 1982, 51:541-550.
14. Molldrem J.J., Jiang Y.Z., Stetler-Stevenson M., et al. Haematological response of patients with myelodysplastic syndrome to antithymocyte globulin is associated with a loss of lymphocyte-mediated inhibition of CFU-GM and alterations in T cell receptor Vbeta profiles. Br J Haematol 1998, 102:1314-1322.
15. Sloand E.M., Wu C.O., Greenberg P., et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol 2008, 26(15):2505-2511.
16. Maciejewski J.P., Sloand E.M., Nunez O., et al. Recombinant humanized anti-IL-2 receptor antibody (daclizumab) produces responses in patients with moderate aplastic anemia. Blood 2003, 102:3584-3586.
17. Kitagawa M., Takahashi M., Yamaguchi S., et al. Expression of inducible nitric oxide synthase (NOS) in bone marrow cells of myelodysplastic syndromes. Leukemia 1999, 13:699-703.
18. Molnar L., Berki T., Hussain A., et al. Detection of TNFα expression in the bone marrow and determination of TNFα production of peripheral blood mononuclear cells in myelodysplastic syndrome. Pathol Oncol Res 2000, 6:18-23.
19. Verhoef G.E., Schouwer P., Ceuppens J.L., et al. Measurement of serum cytokine levels in patients with myelodysplastic syndrome. Leukemia 1992, 6:1268-1272.
20. Deeg H.J., Appelbaum F.R. Hemopoietic stem cell transplantation for myelodysplastic syndrome. Curr Opin Oncol 2000, 12:116-120.
21. Stifter G., Heiss S., Gastl G., et al. Over-expression of tumor necrosis factor-alpha in bone marrow biopsies from patients with myelodysplastic syndromes: relationship to anemia and prognosis. Eur J Haematol 2005, 75:485-491.
22. Deeg H.J., Beckham C., Loken M.R., et al. Negative regulators of hemopoiesis and stroma function in patients with myelodysplastic syndrome. Leuk Lymphoma 2000, 37:405-414.
23. Flores-Figueroa E., Gutierrez-Espindola G., Montesinos J.J., et al. In vitro characterization of hematopoietic microenvironment cells from patients with myelodysplastic syndrome. Leuk Res 2002, 26:677-686.
24. Tauro S., Hepburn M.D., Peddie C.M., et al. Functional disturbance of marrow stromal microenvironment in the myelodysplastic syndromes. Leukemia 2002, 16:785-790.
25. Maciejewski J., Selleri C., Anderson S., et al. Fas antigen expression on CD34+ human marrow cells is induced by interferon gamma and tumor necrosis factor alpha and potentiates cytokine-mediated hematopoietic suppression in vitro. Blood 1995, 85:3183-3190.
26. Benesch M., Platzbecker U., Ward J., et al. Expression of FLIP(Long) and FLIP(Short) in bone marrow mononuclear and CD34+ cells in patients with myelodysplastic syndrome: correlation with apoptosis. Leukemia 2003, 17:2460-2466.
27. Kochenderfer J.N., Kobayashi S., Wieder E.D., et al. Loss of T lymphocyte clonal dominance in patients with myelodysplastic syndrome responsive to immunosuppression. Blood 2002, 100:3639-3645.
28. Zang Y.C., Hong J., Rivera V.M., et al. Human anti-idiotypic T cells induced by TCR peptides corresponding to a common CDR3 sequence motif in myelin basic protein-reactive T cells. Int Immunol 2003, 15:1073-1080.
29. Melenhorst J.J., Eniafe R., Follmann D., et al. Molecular and flow cytometric characterization of the CD4 and CD8 T cell repertoire in patients with myelodysplastic syndrome. Br J Haematol 2002, 119:97-105.
30. Epperson D.E., Nakamura R., Saunthararajah Y., et al. Oligoclonal T cell expansion in myelodysplastic syndrome: evidence for an autoimmune process. Leuk Res 2001, 25:1075-1083.
31. Sloand E.M., Kim S., Fuhrer M., et al. Fas-mediated apoptosis is important in regulating cell replication and death in trisomy 8 hematopoietic cells but not in cells with other cytogenetic abnormalities. Blood 2002, 100:4427-4432.
32. Sloand E.M., Pfannes L., Chen G., et al. CD34 cells from patients with trisomy 8 myelodysplastic syndrome (MDS) express early apoptotic markers but avoid programmed cell death by up-regulation of antiapoptotic proteins. Blood 2007, 109:2399-2405.
33. Chen G., Zeng W., Miyazato A., et al. Distinctive gene expression profiles of CD34 cells from patients with myelodysplastic syndrome characterized by specific chromosomal abnormalities. Blood 2004, 104(13):4210-4218.
34. Call K.M., Ito C.Y., Lindberg C., et al. Mapping and characterization of 129 cosmids on human chromosome 11p. Somat Cell Mol Genet 1992, 18:463-475.
35. Haber D.A., Housman D.E. Role of the WT1 gene in Wilms' tumour. Cancer Surv 1992, 12:105-117.
36. Cilloni D., Gottardi E., Messa F., et al. Significant correlation between the degree of WT1 expression and the International Prognostic Scoring System score in patients with myelodysplastic syndromes. J Clin Oncol 2003, 21:1988-1995.
37. Cilloni D., Saglio G. WT1 as a universal marker for minimal residual disease detection and quantification in myeloid leukemias and in myelodysplastic syndrome. Acta Haematol 2004, 112:79-84.
38. Cilloni D., Gottardi E., Fava M., et al. Usefulness of quantitative assessment of the WT1 gene transcript as a marker for minimal residual disease detection. Blood 2003, 102:773-774.
39. Ellisen L.W., Carlesso N., Cheng T., et al. The Wilms tumor suppressor WT1 directs stage-specific quiescence and differentiation of human hematopoietic progenitor cells. EMBO J 2001, 20:1897-1909.
40. Shichishima T., Okamoto M., Ikeda K., et al. HLA class II haplotype and quantitation of WT1 RNA in Japanese patients with paroxysmal nocturnal hemoglobinuria. Blood 2002, 100:22-28.
41. Sloand E.M., Rezvani K., Yong A., et al. Cytotoxic CD8 T cell immune responses to Wilms tumor protein (WT-1) characterizes immunosuppression-responsive myelodysplasia (MDS). Blood 2006, 108:255.
42. Rezvani K. PR1 vaccination in myeloid malignancies. Expert Rev Vaccines 2008, 7:867-875.
43. Sloand E.M., Mainwaring L., Fuhrer M., et al. Preferential suppression of trisomy 8 compared with normal hematopoietic cell growth by autologous lymphocytes in patients with trisomy 8 myelodysplastic syndrome. Blood 2005, 106:841-851.
44. Jadersten M., Malcovati L., Dybedal I., et al. Erythropoietin and granulocyte colony-stimulating factor treatment associated with improved survival in myelodysplastic syndrome. J Clin Oncol 2008, 26:3607-3613.
45. Jonasova A., Neuwirtova R., Cermak J., et al. Cyclosporin A therapy in hypoplastic MDS patients and certain refractory anaemias without hypoplastic bone marrow. Br J Haematol 1998, 100:304-309.
46. Shimamoto T., Tohyama K., Okamoto T., et al. Cyclosporin A therapy for patients with myelodysplastic syndrome: multicenter pilot studies in Japan. Leuk Res 2003, 27:783-788.
47. Atoyebi W., Bywater L., Rawlings L., et al. Treatment of myelodysplasia with oral cyclosporin. Clin Lab Haematol 2002, 24:211-214.
48. Feng X., Kajigaya S., Solomou E.E., et al. Rabbit ATG but not horse ATG promotes expansion of functional CD4+CD25highFOXP3+ regulatory T cells in vitro. Blood 2008, 111:3675-3683.
49. Barrett A.J., Molldrem J.J., Saunthrajarian Y., et al. Prolonged transfusion independence and disease stability in patients with myelodysplastic syndrome (MDS) responding to antithymocyte glogulin (ATG). Blood 1998, 10(Suppl 1):713. [abstract].
50. Geary C.G., Harrison C.J., Philpott N.J., et al. Abnormal cytogenetic clones in patients with aplastic anaemia: response to immunosuppressive therapy. Br J Haematol 1999, 104:271-274.
51. Stadler M., Germing U., Kliche K.O., et al. A prospective, randomised, phase II study of horse antithymocyte globulin vs rabbit antithymocyte globulin as immune-modulating therapy in patients with low-risk myelodysplastic syndromes. Leukemia 2004, 18:460-465.
52. Killick S.B., Mufti G., Cavenagh J.D., et al. A pilot study of antithymocyte globulin (ATG) in the treatment of patients with low-risk myelodysplasia. Br J Haematol 2003, 120:679-684.
53. Lim Z.Y., Killick S., Germing U., et al. Low IPSS score and bone marrow hypocellularity in MDS patients predict hematological responses to antithymocyte globulin. Leukemia 2007, 21:1436-1441.
54. Broliden P.A., Dahl I.M., Hast R., et al. Antithymocyte globulin and cyclosporine A as combination therapy for low-risk non-sideroblastic myelodysplastic syndromes. Haematologica 2006, 91:667-670.
55. Steensma D.P., Dispenzieri A., Moore S.B., et al. Antithymocyte globulin has limited efficacy and substantial toxicity in unselected anemic patients with myelodysplastic syndrome. Blood 2003, 101:2156-2158.
56. Aivado M., Rong A., Germing U., et al. Long-term remission after intensive chemotherapy in advanced myelodysplastic syndromes is generally associated with restoration of polyclonal haemopoiesis. Br J Haematol 2000, 110:884-886.
57. Sloand E.M., Olnes M., Weinstein B., et al. Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions. Blood 2009, 114:53.
58. Srinivasan R., Geller N., Chakrabarti S., et al. High response rate and improved survival in patients with steroid-refractory acute graft-vs-host disease (SRGVHD) treated with daclizumab with or without infliximab. Blood 2002, 100:173. [abstract].
59. Stasi R., Amadori S. Infliximab chimaeric antitumour necrosis factor alpha monoclonal antibody treatment for patients with myelodysplastic syndromes. Br J Haematol 2002, 116:334-337.
60. Kobbe G., Schneider P., Rohr U., et al. Treatment of severe steroid refractory acute graft-versus-host disease with infliximab, a chimeric human/mouse anti-TNF-alpha antibody. Bone Marrow Transplant 2001, 28:47-49.
61. Maciejewski J.P., Risitano A.M., Sloand E.M., et al. A pilot study of the recombinant soluble human tumour necrosis factor receptor (p75)-Fc fusion protein in patients with myelodysplastic syndrome. Br J Haematol 2002, 117:119-126.
62. List A., Kurtin S., Roe D.J., et al. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med 2005, 352:549-557.
63. Strupp C., Germing U., Aivado M., et al. Thalidomide for the treatment of patients with myelodysplastic syndromes. Leukemia 2002, 16:1-6.
64. Zorat F., Pozzato G. Thalidomide in myelodysplastic syndromes. Biomed Pharmacother 2002, 56:20-30.
65. Molldrem J.J., Leifer E., Bahceci E., et al. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med 2002, 137:156-163.
66. Yazji S., Giles F.J., Tsimberidou A.M., et al. Antithymocyte globulin (ATG)-based therapy in patients with myelodysplastic syndromes. Leukemia 2003, 17:2101-2106.