Changing winds in refractory autoimmune disease in children: Clearing the road for tolerance with cellular therapies

Current Opinion in Rheumatology

Volumn 24, Issue 3, 2012, Pages 267-273

  Swart, Joost F ^a   Lindemans, Caroline A ^a   Van Royen, Annet ^a   Boelens, Jaap J ^a   Prakken, Berent J ^a   Wulffraat, Nico ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

autoimmune disease; cellular therapy; hematopoietic stem cell transplantation; juvenile idiopathic arthritis; mesenchymal stem cell

Indexed keywords

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTHRITIS; AUTOIMMUNE DISEASE; CHILDHOOD DISEASE; DERMATOMYOSITIS; DISEASE SEVERITY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE SYSTEM; IMMUNOSUPPRESSIVE TREATMENT; MESENCHYMAL STEM CELL TRANSPLANTATION; MESENCHYMAL STROMA CELL; MORTALITY; POLYMYOSITIS; PRIORITY JOURNAL; PROTEINURIA; REGULATORY T LYMPHOCYTE; REVIEW; RHEUMATIC DISEASE; RHEUMATOID ARTHRITIS; SYSTEMIC LUPUS ERYTHEMATOSUS; TREATMENT FAILURE;

ARTHRITIS, JUVENILE RHEUMATOID; AUTOIMMUNE DISEASES; CHILD; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNE TOLERANCE; LUPUS ERYTHEMATOSUS, SYSTEMIC; MESENCHYMAL STROMAL CELLS;

EID: 84859265811     PISSN: 10408711     EISSN: 15316963     Source Type: Journal    
DOI: 10.1097/BOR.0b013e32835264f4     Document Type: Review

References (34)

1. Cohen SB, Emery P, Greenwald MW, et al. Rituximab for rheumatoid arthritis refractory to antitumor necrosis factor therapy: results of a multicenter, randomized, double-blind, placebo-controlled, phase III trial evaluating primary efficacy and safety at twenty-four weeks. Arthritis Rheum 2006; 54:2793-2806 (Pubitemid 44497758)
2. Redlich K, Schett G, Steiner G, et al. Rheumatoid arthritis therapy after tumor necrosis factor and interleukin-1 blockade. Arthritis Rheum 2003; 48:3308-3319 (Pubitemid 37531897)
3. Ruperto N, Lovell DJ, Quartier P, et al. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet 2008; 372:383-391
4. de Kleer I, Brinkman DM, Ferster A, et al. Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity. Ann Rheum Dis 2004; 63:1318-1326 (Pubitemid 39273515)
5. Snowden JA, Saccardi R, Allez M, et al. Haematopoietic HSCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2011. [Epub ahead of print]
6. Flato B, Aasland A, Vinje O, et al. Outcome and predictive factors in juvenile rheumatoid arthritis and juvenile spondyloarthropathy. J Rheumatol 1998; 25:366-375 (Pubitemid 28088271)
7. de Kleer I, Vastert B, Klein M, et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4RCD25R immune regulatory network. Blood 2006; 107:1696-1702 (Pubitemid 43242411)
8. van Laar JM, Tyndall A. Adult stem cells in the treatment of autoimmune diseases. Rheumatol (Oxford) 2006; 45:1187-1193 (Pubitemid 44542057)
9. Deligny C, Clave E, Sibon D, et al. New onset of myasthenia gravis after treatment of systemic sclerosis by autologous hematopoietic stem cell transplantation: sustained autoimmunity or inadequate reset of tolerance? Hum Immunol 2010; 71:363-365
10. Wulffraat NM, van Rooijen EM, Tewarie R, et al. Current perspectives of autologous stem cell transplantation for severe juvenile idiopathic arthritis. Autoimmunity 2008; 41:632-638
11. Rabusin M, Andolina M, Maximova N, et al. Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease. Haematologica 2000; 85:81-85
12. Abinun M, Flood TJ, Cant AJ, et al. Autologous T cell depleted haematopoietic stem cell transplantation in children with severe juvenile idiopathic arthritis in the UK (2000-2007). Mol Immunol 2009; 47:46-51
13. Le Blanc K, Rasmusson I, Sundberg B, et al. Treatment of severe acute graftversus-host disease with third party haploidentical mesenchymal stem cells. Lancet 2004; 363:1439-1441 (Pubitemid 38586721)
14. Le Blanc K, Frassoni F, Ball L, et al. Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet 2008; 371:1579-1586 (Pubitemid 351626841)
15. Christopeit M, Schendel M, Foll J, et al. Marked improvement of severe progressive systemic sclerosis after transplantation of mesenchymal stem cells from an allogeneic haploidentical-related donor mediated by ligation of CD137L. Leukemia 2008; 22:1062-1064 (Pubitemid 351689891)
16. Keyszer G, Christopeit M, Fick S, et al. Treatment of severe progressive systemic sclerosis with transplantation of mesenchymal stromal cells from allogeneic related donors: report of five cases. ArthritisRheum 2011; 63:2540-2542
17. Ishigatsubo Y, Ihata A, Kobayashi H, et al. Therapeutic angiogenesis in patients with systemic sclerosis by autologous transplantation of bonemarrow-derived cells. Modern Rheumatol 2010; 20:263-272
18. Carrion F, Nova E, Ruiz C, et al. Autologous mesenchymal stem cell treatment increased T regulatory cells with no effect on disease activity in two systemic lupus erythematosus patients. Lupus 2010; 19:317-322
19. Liang J, Zhang H, Hua B, et al. Allogenic mesenchymal stem cells transplantation in refractory systemic lupus erythematosus: a pilot clinical study. Ann Rheum Dis 2010; 69:1423-1429
20. Sun L, Wang D, Liang J, et al. Umbilical cord mesenchymal stem cell transplantation in severe and refractory systemic lupus erythematosus. Arthritis Rheum 2010; 62:2467-2475
21. Wang DD, Zhang HY, Cao MS, et al. Efficacy of allogeneic mesenchymal stem cell transplantation in patients with drug-resistant polymyositis and dermatomyositis. AnnRheum Dis 2011; 70:1285-1288
22. Liang J, Li X, Zhang H, et al. Allogeneic mesenchymal stem cells transplantation in patients with refractory RA. Clin Rheumatol 2012; 31:157-161
23. Jones EA, English A, Henshaw K, et al. Enumeration and phenotypic characterization of synovial fluid multipotential mesenchymal progenitor cells in inflammatory and degenerative arthritis. Arthritis Rheum 2004; 50:817-827 (Pubitemid 38326182)
24. Endres M, Neumann K, Haupl T, et al. Synovial fluid recruits human mesenchymal progenitors from subchondral spongious bone marrow. J Orthop Res 2007; 25:1299-1307 (Pubitemid 47523359)
25. Jones E, Churchman SM, English A, et al. Mesenchymal stem cells in rheumatoid synovium: enumeration and functional assessment in relation to synovial inflammation level. Ann Rheum Dis 2010; 69:450-457
26. Kastrinaki MC, Sidiropoulos P, Roche S, et al. Functional, molecular and proteomic characterisation of bone marrow mesenchymal stem cells in rheumatoid arthritis. Ann Rheum Dis 2008; 67:741-749 (Pubitemid 351829372)
27. Sun LY, Zhang HY, Feng XB, et al. Abnormality of bone marrow-derived mesenchymal stem cells in patients with systemic lupus erythematosus. Lupus 2007; 16:121-128 (Pubitemid 46347934)
28. Nie Y, Lau C, Lie A, et al. Defective phenotype of mesenchymal stem cells in patients with systemic lupus erythematosus. Lupus 2010; 19:850-859
29. Larghero J, Farge D, Braccini A, et al. Phenotypical and functional characteristics of in vitro expanded bone marrow mesenchymal stem cells from patients with systemic sclerosis. Ann Rheum Dis 2008; 67:443-449 (Pubitemid 351498421)
30. Bocelli-Tyndall C, Bracci L, Spagnoli G, et al. Bone marrow mesenchymal stromal cells (BM-MSCs) from healthy donors and auto-immune disease patients reduce the proliferation of autologous-and allogeneic-stimulated lymphocytes in vitro. Rheumatology 2007; 46:403-408 (Pubitemid 46344649)
31. Herrmann MM, Gaertner S, Stadelmann C, et al. Tolerance induction by bone marrow transplantation in a multiple sclerosis model. Blood 2005; 106: 1875-1883 (Pubitemid 41208608)
32. Van Wijmeersch B, Sprangers B, Rutgeerts O, et al. Allogeneic bone marrow transplantation in models of experimental autoimmune encephalomyelitis: evidence for a graft-versus-autoimmunity effect. Biol Blood Marrow Transplant 2007; 13:627-637 (Pubitemid 46768004)
33. Daikeler T, Hugle T, Farge D, et al. Allogeneic hematopoietic HSCT for patients with autoimmune diseases. Bone Marrow Transplant 2009; 44: 27-33
34. Sorror M, Storer B, Sandmaier BM, et al. Hematopoietic cell transplantationcomorbidity index and Karnofsky performance status are independent predictors of morbidity and mortality after allogeneic nonmyeloablative hematopoietic cell transplantation. Cancer 2008; 112:1992-2001. (Pubitemid 351574075)