Technology Insight: Hematopoietic stem cell transplantation for systemic rheumatic disease

Nature Clinical Practice Rheumatology

Volumn 4, Issue 4, 2008, Pages 184-191

  Nikolov, Nikolay P ^a   Pavletic, Steven Z ^b  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; ANTIBIOTIC AGENT; ANTIFUNGAL AGENT; ANTIVIRUS AGENT; BUSULFAN; CARMUSTINE; CYCLOPHOSPHAMIDE; CYTARABINE; DISEASE MODIFYING ANTIRHEUMATIC DRUG; ETOPOSIDE; FLUDARABINE; GLUCOCORTICOID; GRANULOCYTE COLONY STIMULATING FACTOR; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; IMMUNOSUPPRESSIVE AGENT; MELPHALAN; METHOTREXATE; METHYLPREDNISOLONE; RITUXIMAB; STEROID; THYMOCYTE ANTIBODY;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ANAPHYLAXIS; APLASTIC ANEMIA; AUTOIMMUNE DISEASE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BONE MARROW TOXICITY; CARDIOTOXICITY; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL TRIAL; CRYOPRESERVATION; DISEASE FREE SURVIVAL; DRUG MEGADOSE; GRAFT VERSUS HOST REACTION; HEMATOLOGIC MALIGNANCY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HEMORRHAGIC CYSTITIS; HUMAN; IMMUNE DEFICIENCY; IMMUNOSUPPRESSIVE TREATMENT; INBORN ERROR OF METABOLISM; INFECTION; INFERTILITY; JUVENILE RHEUMATOID ARTHRITIS; LIVER TOXICITY; LUNG TOXICITY; LYMPH NODE IRRADIATION; MONOTHERAPY; MULTIPLE SCLEROSIS; MYELOABLATIVE CONDITIONING; NEOPLASM; OVERALL SURVIVAL; PATIENT SELECTION; PRIORITY JOURNAL; REMISSION; REVIEW; RHEUMATIC DISEASE; RHEUMATOID ARTHRITIS; RISK BENEFIT ANALYSIS; STEM CELL MOBILIZATION; SYSTEMIC LUPUS ERYTHEMATOSUS; SYSTEMIC SCLEROSIS; WHOLE BODY RADIATION; MORTALITY;

AUTOIMMUNE DISEASES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; RHEUMATIC DISEASES;

EID: 45549086848     PISSN: 17458382     EISSN: 17458390     Source Type: Journal    
DOI: 10.1038/ncprheum0756     Document Type: Review

References (49)

1. Copelan EA (2006) Hematopoietic stem-cell transplantation. N Engl J Med 354: 1813-1826
2. Appelbaum FR (2007) Hematopoietic-cell transplantation at 50. N Engl J Med 367: 1472-1475
3. Voltarelli JC et al. (2007) Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 297: 1568-1576.
4. Sykes M and Nikolic B (2005) Treatment of severe autoimmune disease by stem-cell transplantation. Nature 435: 620-627
5. Bekkum DW (2000) Immune ablation and stem-cell therapy in autoimmune disease. Experimental basis for autologous stem-cell transplantation. Arthritis Res 2: 281-284
6. van Bekkum DW (2000) Stem cell transplantation in experimental models of autoimmune disease. J Clin Immunol 20: 10-16
7. Lipsky PE (2001) Systemic lupus erythematosus: An autoimmune disease of B cell hyperactivity. Nat Immunol 2: 764-766
8. Shlomchik MJ et al. (2001) From T to B and back again: Positive feedback in systemic autoimmune disease. Nat Rev Immunol 1: 147-153
9. Muraro PA and Douek DC (2006) Renewing the T cell repertoire to arrest autoimmune aggression. Trends Immunol 27: 61-67
10. Muraro PA et al. (2005) Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J Exp Med 201: 805-816
11. Chen X et al. (2007) Absence of regulatory T cell control of TH1 and TH17 cells is responsible for the autoimmune-mediated pathology in chronic graft versus host disease. Blood 110: 3804-3813
12. Laurence A and O'Shea JJ (2007) T(H)-17 differentiation: Of mice and men. Nat Immunol 8: 903-905
13. Zorina TD et al. (2003) Recovery of the endogenous beta cell function in the NOD model of autoimmune diabetes. Stem Cells 21 377-388
14. Griffith LM at al. (2005) Feasibility of allogeneic hematopoietic stem cell transplantation for autoimmune disease: Position statement from a National Institute of Allergy and Infectious Diseases and National Cancer Institute-Sponsored International Workshop, Bethesda, MD, March 12 and 13,2005. Biol Blood Marrow Transplant 11: 862-870
15. Lu Q et al. (2006) Non-myeloablative allogeneic stem cell transplant in a patient with refractory systemic lupus erythematosus. Bone Marrow Transplant 37: 979-981
16. Muraro PA et al. (2006) Granulocytic invasion of the central nervous system after hematopoietic stem cell transplantation for systemic lupus erythematosus. Haematologica 91: ECR21
17. Brinkman DM et al. (2007) Resetting the adaptive immune system after autologous stem cell transplantation: Lessons from responses to vaccines. J Clin Immunol 27: 647-658
18. Storb RF et al. (2003) Hematopoietic cell transplantation for benign hematological disorders and solid tumors. Hematology Am Soc Hematol Educ Program 2003: 372-397
19. de Kleer I et al. (2006) Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 107: 1696-1702
20. Avanzini MA et al. (2005) B lymphocyte reconstitution after hematopoietic stem cell transplantation: Functional immaturity and slow recovery of memory CD27+ B cells. Exp Hematol 33: 480-486
21. Loh Y et al. (2007) Development of a secondary autoimmune disorder after hematopoietic stem cell transplantation for autoimmune diseases: role of conditicning regimen used. Blood 109: 2643-2548
22. Daikeler T and Tyndall A (2007) Autoimmunity following haematopoietic stem-cell transplantation. Best Pract Res Clin Haematol 20: 349-360
23. Gratwohl A et al. (2005) Autologous hematopoietic stem cell transplantation for autoimmune diseases. Bone Marrow Transplant 35: 869-879
24. Urowitz MB et al. (1997) Mortality studies in systemic lupus erythematosus. Results from a single center. III. Improved survival over 24 years. J Rheumatol 24: 1061-1065
25. Burt RK et al. (2006) Nonmyeloablative hematopoletic stem cell transplantation for systemic lupus erythematosus. JAMA 295: 527-535
26. Jayne D et al. (2004) Autologous stem cell transplantation for systemic lupus erythematosus. Lupus 13: 168-176
27. Snowden JA et al. (2004) Autologous hemopoietic stem cell transplantation in severe rheumatoid arthritis: A report from the EBMT and ABMTR. J Rheumatol 31: 482-488
28. Burt RK et al. (2004) Induction of remission of severe and refractory rheumatoid arthritis by allogeneic mixed chimerism. Arthritis Rheum 50: 2466-2470
29. Brinkman DM et al. (2007) Autologous stem cell transplantation in children with severe progressive systemic or polyarticular juvenile idiopathic arthritis: Long-term follow-up of a prospective clinical trial. Arthritis Rheum 56: 2410-2421
30. Steen VD and Medsger TA Jr (2000) Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 43: 2437-2444
31. Mayes M D et al. (2003) Prevalence, incidence, survival, and disease characteristics of systemic sclerosis in a large US population. Arthritis Rheum 48: 2246-2255
32. Hoyles RK et al. (2006) A multicenter, prospective, randomized, double-blind, placebo-controlled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum 54: 3962-3970
33. Tashkin DIP et al. (2006) Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 354: 2655-2666
34. Nash RA et al. (2007) High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: Long-term follow-up of the US multicenter pilot study. Blood 110: 1388-1396
35. Farge D et al. (2004) Autologous stem cell transplantation in the treatment of systemic sclerosis: Report from the EBMT/EULAR Registry. Ann Rheum Dis 63: 974-981
36. Oyama Y et al. (2007) Autologous non-myeloablative hematopoietic stem cell transplantation in patients with systemic sclerosis. Bone Marrow Transplant 40: 549-555
37. The ASTIS trial [http://www.astistdal.com/] (accessed 14 January 2008)
38. The SCOT trial [http://www.clinicaltrials.gov/ct2/show/ NCT00114530?term=NCT00114530&rank=1] (accessed 14 January 2008)
39. The ASSIST trial [http://www.clinicaltrials.gov/ct2/show/ NCT00278525?term=00278525&rank=1] (accessed 14 January 2008)
40. van Laar JM et al. (2005) Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial: Hope on the horizon for patients with severe systemic sclerosis. Ann Rheum Dis 64: 1515
41. Burt RK et al. (2005) Hematopoietic stem cell transplantation for multiple sclerosis. Arch Neurol 62: 860-864
42. Nash RA et al. (2003) High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Blood 102: 2364-2372
43. Saccardi R et al. (2006) Autologous stem cell transplantation for progressive multiple sclerosis: Update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database. Mult Scler 12: 814-823
44. Daikeler T et al. (2007) Haematopoietic stem cell transplantation for vasculitis including Behcet's disease and polychondritis: A retrospective analysis of patients recorded in the European Bone Marrow Transplantation and European League Against Rheumatism databases and a review of the literature. Ann Rheum Dis 66: 202-207
45. Statkute L et al. (2007) Autologous non-myeloablative hematopoietic stem cell transplantation for refractory systemic vasculitis. Ann Rheum Dis [doi:10.1136/ard.2007.070227]
46. Burt RK et al. (2006) Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases: The evolution from myeloablative to lymphoablative transplant regimens. Arthritis Rheum 54: 3750-3760
47. Tyndall A et al. (2007) Immunomodulatory properties of mesenchymal stem cells: A review based on an interdisciplinary meeting held at the Kennedy Institute of Rheumatology Division, London, UK, 31 October 2005. Arthritis Res Ther 9: 301
48. Tisato V et al. (2007) Mesenchymal stem cells of cord blood origin are effective at preventing but not treating graft-versus-host disease. Leukemia 21: 1992-1999
49. Dazzi F et al. (2007) Cell therapy for autoimmune diseases. Arthritis Res Ther 9: 206