Glucocorticoid-Refractory Acute Graft-versus-Host Disease

Biology of Blood and Marrow Transplantation

Volumn 16, Issue 11, 2010, Pages 1504-1518

  Pidala, Joseph ^a,b   Anasetti, Claudio ^a,b  

^a H LEE MOFFITT CANCER CENTER AND RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF SOUTH FLORIDA   (United States)

Author keywords

Acute graft versus host disease; Allogeneic hematopoietic cell transplantation; Glucocorticoid refractory

Indexed keywords

ALEMTUZUMAB; ANTIBODY; BASILIXIMAB; BMA 031; BTI 322; CD147 ANTIBODY; CD2 ANTIBODY; CD5 ANTIBODY; CYCLOSPORIN A; DACLIZUMAB; DENILEUKIN DIFTITOX; ETANERCEPT; GLUCOCORTICOID; INFLIXIMAB; INTERCELLULAR ADHESION MOLECULE 1 ANTIBODY; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; OKT 3; PENTOSTATIN; PSORALEN; RAPAMYCIN; RITUXIMAB; TACROLIMUS; THALIDOMIDE; THYMOCYTE ANTIBODY; UNCLASSIFIED DRUG; VISILIZUMAB;

ACUTE GRAFT VERSUS HOST DISEASE; BONE MARROW SUPPRESSION; CLINICAL PRACTICE; CLINICAL TRIAL; DRUG DOSE ESCALATION; DRUG FEVER; DRUG HYPERSENSITIVITY; DRUG MEGADOSE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HYPOTENSION; NONHUMAN; PHOTOCHEMOTHERAPY; PROPHYLAXIS; REVIEW; SALVAGE THERAPY; SEIZURE; SINGLE DRUG DOSE; TREATMENT RESPONSE;

DRUG RESISTANCE; GLUCOCORTICOIDS; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; SALVAGE THERAPY;

EID: 77957753981     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2010.01.007     Document Type: Review

References (80)

1. Ferrara J.L., Levine J.E., Reddy P., Holler E. Graft-versus-host disease. Lancet 2009, 373:1550-1561.
2. Ferrara J.L., Levy R., Chao N.J. Pathophysiologic mechanisms of acute graft-vs.-host disease. Biol Blood Marrow Transplant 1999, 5:347-356.
3. Ferrara J.L., Reddy P. Pathophysiology of graft-versus-host disease. Semin Hematol 2006, 43:3-10.
4. Copelan E.A. Hematopoietic stem-cell transplantation. N Engl J Med 2006, 354:1813-1826.
5. Ferrara J.L., Yanik G. Acute graft versus host disease: pathophysiology, risk factors, and prevention strategies. Clin Adv Hematol Oncol 2005, 428:415-419.
6. Hahn T., McCarthy P.L., Zhang M.J., et al. Risk factors for acute graft-versus-host disease after human leukocyte antigen-identical sibling transplants for adults with leukemia. J Clin Oncol 2008, 26:5728-5734.
7. Anasetti C., Beatty P.G., Storb R., et al. Effect of HLA incompatibility on graft-versus-host disease, relapse, and survival after marrow transplantation for patients with leukemia or lymphoma. Hum Immunol 1990, 29:79-91.
8. Weisdorf D., Hakke R., Blazar B., et al. Risk factors for acute graft-versus-host disease in histocompatible donor bone marrow transplantation. Transplantation 1991, 51:1197-1203.
9. Stem Cell Trialists' Collaborative Group. Allogeneic peripheral blood stem-cell compared with bone marrow transplantation in the management of hematologic malignancies: an individual patient data meta-analysis of nine randomized trials. J Clin Oncol 2005, 23:5074-5087.
10. MacMillan M.L., Weisdorf D.J., Wagner J.E., et al. Response of 443 patients to steroids as primary therapy for acute graft-versus-host disease: comparison of grading systems. Biol Blood Marrow Transplant 2002, 8:387-394.
11. Martin P.J., Schoch G., Fisher L., et al. A retrospective analysis of therapy for acute graft-versus-host disease: initial treatment. Blood 1990, 76:1464-1472.
12. Weisdorf D., Haake R., Blazar B., et al. Treatment of moderate/severe acute graft-versus-host disease after allogeneic bone marrow transplantation: an analysis of clinical risk features and outcome. Blood 1990, 75:1024-1030.
13. Macmillan M.L., Couriel D., Weisdorf D.J., et al. A phase 2/3 multicenter randomized clinical trial of ABX-CBL versus ATG as secondary therapy for steroid-resistant acute graft-versus-host disease. Blood 2007, 109:2657-2662.
14. Barnes P.J., Adcock I.M. Glucocorticoid resistance in inflammatory diseases. Lancet 2009, 373:1905-1917.
15. Chikanza I.C., Kozaci D.L. Corticosteroid resistance in rheumatoid arthritis: molecular and cellular perspectives. Rheumatology (Oxford) 2004, 43:1337-1345.
16. Farrell R.J., Kelleher D. Glucocorticoid resistance in inflammatory bowel disease. J Endocrinol 2003, 178:339-346.
17. Hew M., Bhavsar P., Torrego A., et al. Relative corticosteroid insensitivity of peripheral blood mononuclear cells in severe asthma. Am J Respir Crit Care Med 2006, 174:134-141.
18. Barnes P.J. How corticosteroids control inflammation: Quintiles Prize Lecture 2005. Br J Pharmacol 2006, 148:245-254.
19. Rhen T., Cidlowski J.A. Antiinflammatory action of glucocorticoids-new mechanisms for old drugs. N Engl J Med 2005, 353:1711-1723.
20. Irusen E., Matthews J.G., Takahashi A., Barnes P.J., Chung K.F., Adcock I.M. p38 Mitogen-activated protein kinase-induced glucocorticoid receptor phosphorylation reduces its activity: role in steroid-insensitive asthma. J Allergy Clin Immunol 2002, 109:649-657.
21. Abraham S.M., Lawrence T., Kleiman A., et al. Antiinflammatory effects of dexamethasone are partly dependent on induction of dual specificity phosphatase 1. J Exp Med 2006, 203:1883-1889.
22. Wallace A.D., Cidlowski J.A. Proteasome-mediated glucocorticoid receptor degradation restricts transcriptional signaling by glucocorticoids. J Biol Chem 2001, 276:42714-42721.
23. Webster J.C., Oakley R.H., Jewell C.M., Cidlowski J.A. Proinflammatory cytokines regulate human glucocorticoid receptor gene expression and lead to the accumulation of the dominant negative beta isoform: a mechanism for the generation of glucocorticoid resistance. Proc Natl Acad Sci USA 2001, 98:6865-6870.
24. Adcock I.M., Lane S.J., Brown C.R., Lee T.H., Barnes P.J. Abnormal glucocorticoid receptor-activator protein 1 interaction in steroid-resistant asthma. J Exp Med 1995, 182:1951-1958.
25. Matthews J.G., Ito K., Barnes P.J., Adcock I.M. Defective glucocorticoid receptor nuclear translocation and altered histone acetylation patterns in glucocorticoid-resistant patients. J Allergy Clin Immunol 2004, 113:1100-1108.
26. Tsujimura S., Saito K., Nawata M., Nakayamada S., Tanaka Y. Overcoming drug resistance induced by P-glycoprotein on lymphocytes in patients with refractory rheumatoid arthritis. Ann Rheum Dis 2008, 67:380-388.
27. Ishiguro Y., Ohkawara T., Sakuraba H., et al. Macrophage migration inhibitory factor has a proinflammatory activity via the p38 pathway in glucocorticoid-resistant ulcerative colitis. Clin Immunol 2006, 120:335-341.
28. Hakonarson H., Bjornsdottir U.S., Halapi E., et al. Profiling of genes expressed in peripheral blood mononuclear cells predicts glucocorticoid sensitivity in asthma patients. Proc Natl Acad Sci USA 2005, 102:14789-14794.
29. Arai S., Margolis J., Zahurak M., Anders V., Vogelsang G.B. Poor outcome in steroid-refractory graft-versus-host disease with antithymocyte globulin treatment. Biol Blood Marrow Transplant 2002, 8:155-160.
30. Dugan M.J., DeFor T.E., Steinbuch M., Filipovich A.H., Weisdorf D.J. ATG plus corticosteroid therapy for acute graft-versus-host disease: predictors of response and survival. Ann Hematol 1997, 75:41-46.
31. Khoury H., Kashyap A., Adkins D.R., et al. Treatment of steroid-resistant acute graft-versus-host disease with anti-thymocyte globulin. Bone Marrow Transplant 2001, 27:1059-1064.
32. MacMillan M.L., Weisdorf D.J., Davies S.M., et al. Early antithymocyte globulin therapy improves survival in patients with steroid-resistant acute graft-versus-host disease. Biol Blood Marrow Transplant 2002, 8:40-46.
33. Martin P.J., Schoch G., Fisher L., et al. A retrospective analysis of therapy for acute graft-versus-host disease: secondary treatment. Blood 1991, 77:1821-1828.
34. Remberger M., Aschan J., Barkholt L., Tollemar J., Ringden O. Treatment of severe acute graft-versus-host disease with anti-thymocyte globulin. Clin Transplant 2001, 15:147-153.
35. Curtis R.E., Travis L.B., Rowlings P.A., et al. Risk of lymphoproliferative disorders after bone marrow transplantation: a multi-institutional study. Blood 1999, 94:2208-2216.
36. Deeg H.J., Blazar B.R., Bolwell B.J., et al. Treatment of steroid-refractory acute graft-versus-host disease with anti-CD147 monoclonal antibody ABX-CBL. Blood 2001, 98:2052-2058.
37. Hebart H., Ehninger G., Schmidt H., et al. Treatment of steroid-resistant graft-versus-host disease after allogeneic bone marrow transplantation with anti-CD3/TCR monoclonal antibodies. Bone Marrow Transplant 1995, 15:891-894.
38. Carpenter P.A., Appelbaum F.R., Corey L., et al. A humanized non-FcR-binding anti-CD3 antibody, visilizumab, for treatment of steroid-refractory acute graft-versus-host disease. Blood 2002, 99:2712-2719.
39. Carpenter P.A., Lowder J., Johnston L., et al. A phase II multicenter study of visilizumab, humanized anti-CD3 antibody, to treat steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant 2005, 11:465-471.
40. Byers V.S., Henslee P.J., Kernan N.A., et al. Use of an anti-pan T-lymphocyte ricin a chain immunotoxin in steroid-resistant acute graft-versus-host disease. Blood 1990, 75:1426-1432.
41. Wandroo F., Auguston B., Cook M., Craddock C., Mahendra P. Successful use of Campath-1H in the treatment of steroid refractory liver GvHD. Bone Marrow Transplant 2004, 34:285-287.
42. Carella A.M., Beltrami G., Scalzulli P.R., Carella A.M., Corsetti M.T. Alemtuzumab can successfully treat steroid-refractory acute graft-versus-host disease (aGVHD). Bone Marrow Transplant 2004, 33:131-132.
43. Gómez-Almaguer D., Ruiz-Arguelles G.J., del Carmen Tarin-Arzaga L., et al. Alemtuzumab for the treatment of steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant 2008, 14:10-15.
44. Martinez C., Solano C., Ferra C., Sampol A., Valcarcel D., Perez-Simon J.A. Alemtuzumab as treatment of steroid-refractory acute graft-versus-host disease: results of a phase II study. Biol Blood Marrow Transplant 2009, 15:639-642.
45. Anasetti C., Hansen J.A., Waldmann T.A., et al. Treatment of acute graft-versus-host disease with humanized anti-Tac: an antibody that binds to the interleukin-2 receptor. Blood 1994, 84:1320-1327.
46. Bordigoni P., Dimicoli S., Clement L., et al. Daclizumab, an efficient treatment for steroid-refractory acute graft-versus-host disease. Br J Haematol 2006, 135:382-385.
47. Przepiorka D., Kernan N.A., Ippoliti C., et al. Daclizumab, a humanized anti-interleukin-2 receptor alpha chain antibody, for treatment of acute graft-versus-host disease. Blood 2000, 95:83-89.
48. Willenbacher W., Basara N., Blau I.W., Fauser A.A., Kiehl M.G. Treatment of steroid refractory acute and chronic graft-versus-host disease with daclizumab. Br J Haematol 2001, 112:820-823.
49. Perales M.A., Ishill N., Lomazow W.A., et al. Long-term follow-up of patients treated with daclizumab for steroid-refractory acute graft-vs-host disease. Bone Marrow Transplant 2007, 40:481-486.
50. Pinana J.L., Valcarcel D., Martino R., et al. Encouraging results with inolimomab (anti-IL-2 receptor) as treatment for refractory acute graft-versus-host disease. Biol Blood Marrow Transplant 2006, 12:1135-1141.
51. Massenkeil G., Rackwitz S., Genvresse I., Rosen O., Dorken B., Arnold R. Basiliximab is well tolerated and effective in the treatment of steroid-refractory acute graft-versus-host disease after allogeneic stem cell transplantation. Bone Marrow Transplant 2002, 30:899-903.
52. Schmidt-Hieber M., Fietz T., Knauf W., et al. Efficacy of the interleukin-2 receptor antagonist basiliximab in steroid-refractory acute graft-versus-host disease. Br J Haematol 2005, 130:568-574.
53. Vlad G., Ho E.K., Vasilescu E.R., et al. Anti-CD25 treatment and FOXP3-positive regulatory T cells in heart transplantation. Transpl Immunol 2007, 18:13-21.
54. Wang Z., Xiao L., Shi B.Y., et al. Short-term anti-CD25 monoclonal antibody treatment and neogenetic CD4(+)CD25(high) regulatory T cells in kidney transplantation. Transpl Immunol 2008, 19:69-73.
55. Oh U., Blevins G., Griffith C., et al. Regulatory T cells are reduced during anti-CD25 antibody treatment of multiple sclerosis. Arch Neurol 2009, 66:471-479.
56. Rech A.J., Vonderheide R.H. Clinical use of anti-CD25 antibody daclizumab to enhance immune responses to tumor antigen vaccination by targeting regulatory T cells. Ann N Y Acad Sci 2009, 1174:99-106.
57. Ho V.T., Zahrieh D., Hochberg E., et al. Safety and efficacy of denileukin diftitox in patients with steroid-refractory acute graft-versus-host disease after allogeneic hematopoietic stem cell transplantation. Blood 2004, 104:1224-1226.
58. Yamane T., Yamamura R., Aoyama Y., et al. Infliximab for the treatment of severe steroid refractory acute graft-versus-host disease in three patients after allogeneic hematopoietic transplantation. Leuk Lymphoma 2003, 44:2095-2097.
59. Couriel D., Saliba R., Hicks K., et al. Tumor necrosis factor-alpha blockade for the treatment of acute GVHD. Blood 2004, 104:649-654.
60. Patriarca F., Sperotto A., Damiani D., et al. Infliximab treatment for steroid-refractory acute graft-versus-host disease. Haematologica 2004, 89:1352-1359.
61. Pidala J., Kim J., Field T., et al. Infliximab for managing steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant 2009, 15:1116-1121.
62. Busca A., Locatelli F., Marmont F., Ceretto C., Falda M. Recombinant human soluble tumor necrosis factor receptor fusion protein as treatment for steroid refractory graft-versus-host disease following allogeneic hematopoietic stem cell transplantation. Am J Hematol 2007, 82:45-52.
63. Alousi A.M., Weisdorf D.J., Logan B.R., et al. Etanercept, mycophenolate, denileukin, or pentostatin plus corticosteroids for acute graft-versus-host disease: a randomized phase 2 trial from the Blood and Marrow Transplant Clinical Trials Network. Blood 2009, 114:511-517.
64. Martin P.J., Storer B.E., Rowley S.D., et al. Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood 2009, 113:5074-5082.
65. Baudard M., Vincent A., Moreau P., Kergueris M.F., Harousseau J.L., Milpied N. Mycophenolate mofetil for the treatment of acute and chronic GVHD is effective and well tolerated but induces a high risk of infectious complications: a series of 21 BM or PBSC transplant patients. Bone Marrow Transplant 2002, 30:287-295.
66. Krejci M., Doubek M., Buchler T., Brychtova Y., Vorlicek J., Mayer J. Mycophenolate mofetil for the treatment of acute and chronic steroid-refractory graft-versus-host disease. Ann Hematol 2005, 84:681-685.
67. Pidala J., Kim J., Perkins J., et al. Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease. Bone Marrow Transplant 2009.
68. Kim J.G., Sohn S.K., Kim D.H., et al. Different efficacy of mycophenolate mofetil as salvage treatment for acute and chronic GVHD after allogeneic stem cell transplant. Eur J Haematol 2004, 73:56-61.
69. Takami A., Mochizuki K., Okumura H., et al. Mycophenolate mofetil is effective and well tolerated in the treatment of refractory acute and chronic graft-versus-host disease. Int J Hematol 2006, 83:80-85.
70. Furlong T., Martin P., Flowers M.E., et al. Therapy with mycophenolate mofetil for refractory acute and chronic GVHD. Bone Marrow Transplant 2009, 44:739-748.
71. Benito A.I., Furlong T., Martin P.J., et al. Sirolimus (rapamycin) for the treatment of steroid-refractory acute graft-versus-host disease. Transplantation 2001, 72:1924-1929.
72. Ghez D., Rubio M.T., Maillard N., et al. Rapamycin for refractory acute graft-versus-host disease. Transplantation 2009, 88:1081-1087.
73. Hoda D., Pidala J., Salgado-Vila N., et al. Sirolimus for treatment of steroid-refractory acute graft-versus-host disease. Bone Marrow Transplant 2009, 72:1924-1929.
74. Bolanos-Meade J., Jacobsohn D.A., Margolis J., et al. Pentostatin in steroid-refractory acute graft-versus-host disease. J Clin Oncol 2005, 23:2661-2668.
75. Kulkarni S., Powles R., Sirohi B., et al. Thalidomide after allogeneic haematopoietic stem cell transplantation: activity in chronic but not in acute graft-versus-host disease. Bone Marrow Transplant 2003, 32:165-170.
76. Greinix H.T., Volc-Platzer B., Kalhs P., et al. Extracorporeal photochemotherapy in the treatment of severe steroid-refractory acute graft-versus-host disease: a pilot study. Blood 2000, 96:2426-2431.
77. Messina C., Locatelli F., Lanino E., et al. Extracorporeal photochemotherapy for paediatric patients with graft-versus-host disease after haematopoietic stem cell transplantation. Br J Haematol 2003, 122:118-127.
78. Perfetti P., Carlier P., Strada P., et al. Extracorporeal photopheresis for the treatment of steroid refractory acute GVHD. Bone Marrow Transplant 2008, 42:609-617.
79. Kennedy G.A., Butler J., Western R., Morton J., Durrant S., Hill G.R. Combination antithymocyte globulin and soluble TNFalpha inhibitor (etanercept) +/- mycophenolate mofetil for treatment of steroid refractory acute graft-versus-host disease. Bone Marrow Transplant 2006, 37:1143-1147.
80. Rao K., Rao A., Karlsson H., Jagani M., Veys P., Amrolia P.J. Improved survival and preserved antiviral responses after combination therapy with daclizumab and infliximab in steroid-refractory graft-versus-host disease. J Pediatr Hematol Oncol 2009, 31:456-461.