Hematopoietic stem cell transplantation for curing children with severe autoimmune diseases: Is this a valid option?

Pediatric Transplantation

Volumn 16, Issue 5, 2012, Pages 413-425

  Pession, Andrea ^a   Zama, Daniele ^a   Masetti, Riccardo ^a   Gasperini, Pietro ^a   Prete, Arcangelo ^a  

^a UNIVERSITY OF BOLOGNA   (Italy)

Author keywords

autoimmune diseases; children; hematopoietic stem cell transplantation

Indexed keywords

ADALIMUMAB; ALEMTUZUMAB; CARMUSTINE; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; CYTARABINE; DISEASE MODIFYING ANTIRHEUMATIC DRUG; ETOPOSIDE; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; INFLIXIMAB; INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; INTERLEUKIN 6 ANTIBODY; MELPHALAN; METHOTREXATE; METHYLPREDNISOLONE; STEROID; THYMOCYTE ANTIBODY; TUMOR NECROSIS FACTOR INHIBITOR;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; AUTOIMMUNE DISEASE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CROHN DISEASE; DISEASE SEVERITY; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE CYTOPENIA; IMMUNOSUPPRESSIVE TREATMENT; INSULIN DEPENDENT DIABETES MELLITUS; JUVENILE RHEUMATOID ARTHRITIS; NONHUMAN; PEDIATRIC SURGERY; REVIEW; SYSTEMIC LUPUS ERYTHEMATOSUS; SYSTEMIC SCLEROSIS; WHOLE BODY RADIATION;

AUTOIMMUNE DISEASES; CHILD; CROHN DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; SCLERODERMA, SYSTEMIC; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 84863721492     PISSN: 13973142     EISSN: 13993046     Source Type: Journal    
DOI: 10.1111/j.1399-3046.2012.01691.x     Document Type: Review

References (95)

1. Abrahamsson S, Muraro PA,. Immune re-education following autologous hematopoietic stem cell transplantation. Autoimmunity 2008: 41: 577-584.
2. Jacobson DL, Gange SJ, Rose NR, Graham NM,. Epidemiology and estimated population burden of selected autoimmune disease in the United States. Clin Immunol Immunopathol 1997: 84: 223-243.
3. Davidson A, Diamond B,. Autoimmune disease. N Engl J Med 2001: 345: 340-350.
4. Snowden JA, Saccardi R, Allez M, et al., Haematopoietic SCT in severe autoimmune diseases: Updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2011: Oct 17. doi: 10.1038/bmt.2011.185 [Epub ahead of print].
5. Podolskaya A, Stadermann M, Pilkington C, Marks SD, Tllus K,. B cell depletion therapy for 19 patients with refractory systemic lupus erythematosus. Arch Dis Child 2008: 93: 401-406.
6. Marmont AM, Van Lint MT, Gualandi F, Bacigalupo A,. Autologous marrow stem cell transplantation for severe systemic lupus erythematosus of long duration. Lupus 1997: 6: 545-548.
7. Marmont AM,. Hematopoietic stem cell transplantation for severe autoimmune disease: Progress and perspectives. Cell Ther Transplant 2011: 2: 1-8.
8. Rabusin M, Andolina M, Maximova N, on behalf of the EBMT P aediatric and A utoimmune D iseases W orking P arty. Hematopoietic SCT in autoimmune disease in children: Rationale and new perspectives. Bone Marrow Transplant 2008: 41: S96-S99.
9. Loh Y, Oyama Y, Statkute L, et al., Development of a secondary autoimmune disorder after hematopoietic stem cell transplantation for autoimmune diseases: Role of conditioning regimen used. Blood 2007: 109: 2643e8.
10. Burt RK, Craig R, Cohen B, Suffit R, Barr W,. Hematopoietic stem cell transplantation for autoimmune diseases: What have we learned? J Autoimmun 2008: 30: 116-120.
11. Knaan-Shanzer S, Houbew P, Kinwel-Bohre EP, et al., Remission induction of adjuvant arthritis in rats by total body irradiation and autologous bone marrow transplantation. Bone Marrow Transplant 1991: 8: 333-338.
12. Van Gelder M, Van Bekkum DW,. Effective treatment of relapsing experimental auto immune encephalomyelitis with pseudoautologous bone marrow transplantation. Bone Marrow Transplant 1996: 18: 1029-1034.
13. Van Bekkum DW,. Conditioning regimens for the treatment of experimental arthritis with autologous bone marrow transplantation. Bone Marrow Transplant 2000: 25: 357-364.
14. Muraro PA, Douek DC, Packer A, et al., Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J Exp Med 2005: 201: 805-816.
15. Farge D, Henegar C, Carmagnat M, et al., Analysis of immune reconstitution after autologous bone marrow transplantation in systemic sclerosis. Arthritis Rheum 2005: 52: 1555-1563.
16. Alexander T, Thiel A, Rosen O, et al., Depletion of autoreactive immunologic memory followed by autologous hematopoietic stem cell transplantation in patients with refractory SLE induces long-term remission through de novo generation of a juvenile and tolerant immune system. Blood 2008: 113: 214-223.
17. Bennett CL,. The immune dysregulation, polyendocrinopathy, enteropathy, X linked syndrome (IPEX) is caused by mutations FOXP3. Nat Genet 2001: 27: 20-21.
18. Zhang L, Bertucci AM, Ramsey-Goldman R, Burt RK, Datta SK, Regulatory T cell (treg) subset return in patients with refractory lupus following stem cell transplantation, and TGF-beta-producing CD8+ Treg cells are associated with immunological remission of lupus. J Immunol 2009: 183: 6346-6358.
19. 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 CD4+CD25+ immune regulatory network. Blood 2006: 107: 1696-1702.
20. Roord STA, De Jager W, Boon L, et al., Autologous bone marrow transplantation in autoimmune arthritis restores immune homeostasis through CD4+CD25+Foxp3+ regulatory T cells. Blood 2008: 111: 5233-5241.
21. Van Wijk F, Roord ST, Vastert B, De Kleer I, Wulffraat N, Prakken BJ,. Regulatory T cells in autologous stem cell transplantation for autoimmune disease. Autoimmunity 2008: 41: 585-591.
22. Bingham S, Veale D, Fearon U, et al., High-dose cyclophosphamide with stem cell rescue for severe rheumatoid arthritis: Short-term efficacy correlates with reduction of macroscopic and histologic synovitis. Arthritis Rheum 2002: 46: 837-839.
23. Muraro P,. Restoring self-tolerance: Lessons from the clinic. Bone Marrow Transplant 2010: 45: S2.
24. Verrecchia F, Laboureau J, Verola O, et al., Skin involvement in scleroderma-where histological and clinical scores meet. Rheumatology 2007: 46: 833-841.71.
25. Farge D, Nash R, Laar JM,. Autologous stem cell transplantation for systemic sclerosis. Autoimmunity 2008: 41: 616-624.
26. Fleming JN, Nash RA, Mcleod DO, et al., Capillary regeneration in scleroderma: Stem cell therapy reverses phenotype? PLoS ONE 2008: 3: e1452.
27. Miniati I, Guiducci S, Conforti ML, et al., Autologous stem cell transplantation improves microcirculation in systemic sclerosis. Ann Rheum Dis 2009: 68: 94-98.
28. Launay D, Marjanovic Z, De Bazelaire C, et al., Autologous hematopoietic stem cell transplant in systemic sclerosis: Quantitative high resolution computed tomography of the chest scoring. J Rheumatol 2009: 36: 1460-1463.
29. Baldwin JL, Storb R, Thomas ED, Mannik M,. Bone marrow transplantation in patients with gold-induced marrow aplasia. Arthritis Rheum 1977: 20: 1043-1048.
30. Eedy DJ, Burrows D, Bridges JM, Jones FG,. Clearance of severe psoriasis after allogenic bone marrow transplantation. BMJ 1990: 300: 908.
31. Meloni G, Capria S, Vignetti M, Mandelli F, Modena V,. Blast crisis of chronic myelogenous leukemia in long-lasting systemic lupus erythematosus: Regression of both diseases after autologous bone marrow transplantation. Blood 1997: 89: 4659.
32. Gratwhol A,. Allogeneic hematopoietic stem cell transplantation for severe autoimmune disease. Autoimmunity 2008: 41: 673-678.
33. Lampeter EF, Homberg M, Quabeck K, et al., Transfer of insulin-dependent diabetes between HLA-identical siblings by bone marrow transplantation. Lancet 1993: 341: 1243-1244.
34. Berisso GA, Van Lint MT, Bacigalupo A, Marmont AM,. Adoptive autoimmune hyperthyroidism following allogeneic stem cell transplantation from an HLA-identical sibling with Graves' disease. Bone Marrow Transplant 1999: 23: 1091-1092.
35. Marazuela M, Steegman JL,. Transfer of autoimmune hypothyroidism following bone marrow transplantation from a donor with Graves' disease. Bone Marrow Transplant 2000: 26: 1217-1220.
36. Sykes M, Nikolic B,. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005: 435: 620-627.
37. Marmont AM, Gualandi F, Van Lint MT, Bacigalupo A,. Refractory Evans' syndrome treated with allogeneic SCT followed by DLI. Demonstration of a graft-versus-autoimmunity effect. Bone Marrow Transplant 2003: 31: 399-402.
38. Hough RE, Snowden JA, Wulffraat NM,. Haemopoietic stem cell transplantation in autoimmune diseases: A European perspective. Br J Haematol 2005: 128: 432-459.
39. Griffith LM, Pavletic SZ, Tyndall A, et al., 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. Biol Blood Marrow Transplant 2005: 11: 862-870.
40. Marmont AM, Dominietto A, Gualandi F, Piaggio G, Van Lint MT, Bacigalupo A,. Pure white cell aplasia (PWCA) relapsing after allogeneic BMT and successfully treated with nine DLIs. Biol Blood Marrow Transplant 2006: 12: 987-989.
41. Seung E, et al., Allogeneic hematopoietic chimerism in mice treated with sublethal myeloablation and anti-CD154 antibody: Absence of graft-versus-host disease, induction of skin allograft tolerance, and prevention of recurrent autoimmunity in islet-allografted NOD/Lt mice. Blood 2000: 95: 2175-2182.
42. Marmont AM,. Stem cells transplantation for autoimmune disorders. Coincidental autoimmune autoimmune disease in patients transplanted for conventional indications. Best Pract Clin Haematol 2004: 17: 223-232.
43. Marmont AM,. Historical perspectives, rationale and future directions for hematopoietic stem cell transplantation for severe autoimmune disease. Hematologica 2002: 87: 24-27.
44. Daikeler T, Hugle T, Farge D, et al., Allogeneic hematopoietic SCT for patients with autoimmune diseases. BMT 2009: 44: 27-33.
45. Manners PJ, Bower C,. Worldwide prevalence of juvenile arthritis: Why does it vary so much? J Rheumatol 2002: 29: 1520-1530.
46. Cassidy JT, Petty RE, Laxer RM, Lindsey CB,. Textbook of Pediatric Rheumatology, 5th edn. Philadelphia: Elsevier Saunders, 2006.
47. Yokota S, Imagawa T, Mori M, et al., Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: A randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet 2008: 371: 998-1006.
48. De Kleer IM, Brinkman DMC, 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.
49. Brinkman DMC, De Kleer IM, Ten Cate R, et al., Autologous stem cell transplantation in children with severe progressive systemic or polyarticular juvenile idiopathic arthritis. Arthritis Rheum 2007: 56: 2410-2421.
50. Ferreira RA, Vastert SJ, Abinun M, et al., Hemophagocytosis during fludarabine-based SCT for systemic juvenile idiopathic arthritis. BMT 2006: 38: 249-251.
51. Albinum A, Flood TJ, Cant AJ, et al., Autologous T cell depleted haematopoietic stem cell transplantation in children with severe juvenile idiopathic arthritis in the UK (200-2007). Mol Immunol 2007: 47: 46-51.
52. Cortés-Hernández J, Torres-Salido MT, Medranoa S, Vilardell Tarrés M, Ordi-Ros J,. Long-term outcomes-mycophenolate mofetil treatment for lupus nephritis with addition of tacrolimus for resistant cases. Nephrol Dial Transplant 2010: 25: 3939-3948.
53. Jayne D, Passweg J, Marmont A, et al., Autologous stem cell transplantation for systemic lupus erythematosus. Lupus 2004: 13: 168-176.
54. Chen J, Wang Y, Kunkel G, et al., Use of CD34+ autologous stem cell transplantation in the treatment of children with refractory systemic lupus erythematosus. Clin Rheumatol 2005: 24: 464-468.
55. Burt RK, Oyama Y, Verda L, et al., Nonmyeloablative hematopoietic stem cell transplantation for systemic lupus review for systemic lupus erythematosus. JAMA 2006: 295: 5277-5535.
56. Bamias G, Nyce MR, Delarue SA, Comminelli F,. New concepts in the pathophysiology of inflammatory bowel disease. Ann Intern Med 2005: 143: 895-904.
57. Nikolaus S, Raedler A, Kühbacker T, Sfikas N, Fölsch UR, Schreiber S,. Mechanisms in failure of infliximab for Crohn's disease. Lancet 2000: 356: 1475-1479.
58. Kugathasan S, Werlin SL, Martinez A, Rivera MT, Heikenen JB, Binion DG,. Prolonged duration of response to infliximab in early but not late pediatric Crohn's disease. Am J Gastroenterol 2000: 95: 3189-3194.
59. Kliegman RM, Stanton BMD, Geme JS, Schor N, Behrman RE, eds. Nelson Textbook of Pediatrics, 19th edn. WB Saunders: Philadelphia, PA, 2007.
60. Burt RK, Craig RM, Milanetti F, et al., Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: Long-term follow-up. Blood 2010: 116: 6123-6132.
61. Oyama Y, Craig RM, Traynor AE, et al., Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease. Gastroenterology 2005: 128: 552-563.
62. Patterson CC, Dahlquist GG, Gyürüs E, Green A, Soltész G, the EURODIAB S tudy G roup. Incidence trends for childhood type 1 diabetes in Europe during 1989-2003 and predicted new cases 2005-20: A multicentre prospective registration study. Lancet 2009: 373: 2027-2033.
63. Couri CEB, Oliveira MCB, Stracieri ABPL, et al., C-peptide levels and insulin independence following autologous nonmyeloablative stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2009: 301: 1573-1579.
64. Voltarelli JC, Couri CE, Stracieri AB, et al., Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2007: 297: 1568-1576.
65. Snarski E, Milczarczyk A, Torosian T, et al., Independence of exogenous insulin following immunoablation and stem cell reconstitution in newly diagnosed diabetes type I. Bone Marrow Transplant 2011: 46: 562-566.
66. Rabusin M, Andolina M, Maximova N, Giurici N, on behalf of the A utoimmune D iseases and P aediatric D isease W orking P arty. Haematopoietic stem cell transplantation with refractory autoimmune cytopenia: A retrospective analysis from the EBMT registry. Bone Marrow Transplant 2011: 46 (S1): S377.
67. Passweg J, Rabusin M, Musso M, et al., Haematopoietic stem cell transplantation for refractory autoimmune cytopenia. Br J Haematol 2004: 125: 749-755.
68. Farge D, Passweg J, Van Laar JM, et al., Autologous stem cell transplantation in the treatment of systemic sclerosis: Report from the EBMT/EULAR Registry. Ann Rheum Dis 2004: 63: 974-981.
69. Vonk MC, Marjanovic Z, Van Den Hoogen FH, et al., Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis. Ann Rheum Dis 2008: 67: 98-104.
70. Wulffraat N, Van Royen A, Bierings M, Vossen J, Kuis W,. Autologous haemopoietic stem-cell transplantation in four patients with refractory juvenile chronic arthritis. Lancet 1999: 353: 550-553.
71. Singh JA, Wells GA, Christensen R, et al., Adverse effects of biologics: A network meta-analysis and Cochrane overview. Cochrane Database Syst Rev 2011: 2: CD008794.
72. Burt RK, Marmont A, Oyama Y, et al., Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases. The evolution from myeloablative to lymphoablative transplant regimens. Arthtritis Rheum 2006: 54: 3750-3760.
73. Farge D, Labopin M, Tyndall A, et al., Autologus stem cell transplantation for autoimmune disease: An observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Party on Autoimmune Disease. Hematologica 2010: 95: 284-292.
74. Gratwohl A, Passweg J, Bocelli-Tyndall C, et al., Autologous haematopoietic stem cell transplantation for autoimmune diseases. Bone Marrow Transplant 2005: 15: 869-879.
75. Loberiza FR Jr, Zhang MJ, Lee SJ, et al., Association of transplant center and physician factor on mortality after hematopoietic stem cell transplantation in the United States. Blood 2005: 105: 2979-2987.
76. Krauss AC, Kamani NR,. Haemapoietic stem cell transplantation for pediatric autoimmune disease: Where we stand and where we need to go. Bone Marrow Transplant 2009: 44: 137-143.
77. Prince FHM, Otten MH, Van Suijlekom-Smit LWA,. Diagnosis and management of juvenile idiopathic arthritis. BMJ 2011: 342: 95-102.
78. Daikeler T, Labopin M, Di Gioia M, et al., Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: A retrospective study of the EBMT Autoimmune Disease Working Party. Blood 2011: 118: 1693-1698.
79. Duell T, Van Lint MT, Ljungman P, et al., Health and functional status of long-term survivors of bone marrow transplantation. EBMT Working Party on Late Effects and EULEP Study Group on Late Effects. European Group for Blood and Marrow Transplantation. Ann Intern Med. 1997: 126: 184-192.
80. Faraci M, Békássy AN, De Fazio V, Tichelli A, Dini G, EBMT P aediatric and L ate E ffects W orking P arties. Non-endocrine late complications in children after allogeneic haematopoietic SCT. Bone Marrow Transplant 2008: 41: S49-S57.
81. Cohen A, Békássy AN, Gaiero A, et al., Endocrinological late complications after hematopoietic SCT in children. Bone Marrow Transplant 2008: 41: S43-S48.
82. Marmont AM,. Allogeneic haematopoietic stem cell transplantation for severe autoimmune diseases: Great expectations but controversial evidence. Bone Marrow Transplant 2006: 38: 1-4.
83. Mckendry RJ, Huebsch L, Leclair B,. Progression of rheumatoid arthritis following bone marrow transplantation. A case report with a 13-year follow-up. Arthritis Rheum 1996: 39: 1246-1253.
84. Tapprich C, Feuk R, Schneider P, Bernhardt A, Haas R, Kobbe G,. Early recurrence of rheumatoid arthritis after nonmyeloablative allogeneic blood stem cell transplantation in a patient with multiple myeloma. Bone Marrow Transplant 2003: 32: 629-631.
85. Hawkey CJ,. ASTIC Autologous Stem Cell Transplantation for Crohn's Disease. Available at:.
86. Szabolcs P,. Autologous Stem Cell Transplantation for Crohn's Disease. Available at:.
87. Burt R,. Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Refractory Crohn. Available at:.
88. Lavalle FJ,. Safety and Efficacy Study of Autologous Stem Cell Transplantation for Early Onset Type I Diabetes Mellitus, Available at:.
89. Zhu D,. Efficacy and Safety Study of Autologous Hematopoietic Stem Cell Transplantation to Treat New Onset Type 1 Diabetes. Available at:.
90. Burt R,. A Trial of High Dose Immunosuppression and Autologous Hematopoietic Stem Cell Support Versus Intensive Insulin Therapy in Adults With Early Onset Type I Diabetes Mellitus. Available at:.
91. Ning G,. Autologous Hematopoietic Stem Cell Transplantation for Early Onset Type 1 Diabetes. Available at:.
92. Burt R,. Allogeneic Stem Cell Transplantation in Systemic Lupus Erythematosus. Available at:.
93. Sullivan K,. Nonmyeloablative Allo Stem Cell Transplant for Severe Autoimmune Diseases. Available at:.
94. Hiepe F,. Autologous Stem Cell Transplantation for Refractory Systemic Lupus Erythematosus (ASSIST). Available at:.
95. Burt R,. Allogeneic hematopoietic stem cell transplantation (NST) for patients with systemic sclerosis. Available at:.