Haematopoietic SCT in autoimmune diseases in children: Rationale and new perspectives

Bone Marrow Transplantation

Volumn 41, Issue SUPPL. 2, 2008, Pages

  Rabusin, M ^a   Andolina, M ^a   Maximova, N ^a  

^a INSTITUTE FOR MATERNAL AND CHILD HEALTH IRCCS BURLO GAROFOLO   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; INTERLEUKIN 6 ANTIBODY; INTERLEUKIN 6 RECEPTOR; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; NONSTEROID ANTIINFLAMMATORY AGENT; PENICILLAMINE; THYMOCYTE ANTIBODY; TUMOR NECROSIS FACTOR ANTIBODY;

AUTOIMMUNE DISEASE; CHILD; CLINICAL EFFECTIVENESS; CLINICAL TRIAL; CROHN DISEASE; CYTOPENIA; DISEASE SEVERITY; GRAFT VERSUS HOST REACTION; HEMATOLOGIC DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOREGULATION; IMMUNOSUPPRESSIVE TREATMENT; JUVENILE RHEUMATOID ARTHRITIS; MYELOABLATIVE CONDITIONING; NONHUMAN; PATIENT SAFETY; PRIORITY JOURNAL; REMISSION; REVIEW; SYSTEMIC LUPUS ERYTHEMATOSUS; SYSTEMIC SCLEROSIS; T CELL DEPLETION; TREATMENT RESPONSE; WHOLE BODY RADIATION;

AUTOIMMUNE DISEASES; CHILD; CLINICAL TRIALS, PHASE III AS TOPIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; SURVIVAL ANALYSIS;

EID: 45749129126     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2008.64     Document Type: Review

References (39)

1. Jacobson DL, Gange SJ, Rose NR, Graham NM. Epidemiology and estimates population burden of selected autoimmune diseases in United States. Clin Immunol Immunopathol 1997; 84: 223-243.
2. Sinha AA, Lopez MT, Mc Devitt HO. Autoimmune diseases: The failure of self tolerance. Science 1990; 248: 1380-1385.
3. Marmont AM. New horizons in the treatment of autoimmune diseases: immunoablation and stem cell transplantation. Ann Rev Med 2000; 51: 114-134.
4. Lafferty KL, Gazda LS (eds). Costimulation and the regulation of autoimmunity. The Autoimmune Diseases. Academic: San Diego, 1998, pp 324-330.
5. van Bekkum DW, Bohre EP, Houben PF, Knaan-Shanzer S. Regression of adjuvant-induced arthritis in rats following bone marrow transplantation. Proc Soc Natl Sci USA 1989; 86: 10090-10094.
6. Knaan-Shanzer S, Houben P, Kinwel-Bohre EP, van Bekkum DW. Remission induction of adjuvant arthritis in rats by total body irradiation and autologous bone marrow transplantation. Bone Marrow Transplant 1991; 87: 833-838.
7. van Bekkum DW. BMT in experimental autoimmune diseases. Bone Marrow Transplant 1993; 11: 1183-1187.
8. Hough RE, Snowden JA, Wulffraat N. Haemopoietic stem cell transplantation in autoimmune diseases: A European perspective. Br J Haematol 2005: 128: 432-459.
9. Snowden JA, Kearney P, Kearney A, Cooley HM, Grigg A, Jacobs P et al Long-term outcome of autoimmune disease following allogeneic bone marrow transplantation. Arthritis. Rheum 1998; 41: 453-459.
10. Lopes Cubero SO, Sullivan KM, McDonald GB. Course of Crohn's disease after allogeneic bone marrow transplantation. Gastroenterology 1998; 114: 433-440.
11. Marmont AM. Immune ablation and stem cell transplantation for severe Evans syndrome and refractory thrombocytopenic purpura. Bone Marrow transplant 1999; 23: 1215-1216.
12. Marmont AM. Immunoablation followed or not by hematopoietic stem cells as an intensive therapy for severe autoimmune diseases. New perspectives, new problems. Haematologica 2000; 85: 71-80.
13. de Kleer IM, Brinkman DMC, Ferster A, Abinun M, Quartier P, van der J 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.
14. Fargo D, Passweg J, van Laar JM, Marjanovic Z, Besenthal C, Finke J 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.
15. Jayne D, Passweg J, Marmont A, Farge D, Zhao X, Arnold D et al. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus 2004; 13: 168-176.
16. Passweg J, Rabusin M, Musso M, Beguin Y, Cesaro S, Ehninger G et al Haematopoietic stem cell transplantation for refractory autoimmune cytopenia. Br J Haematol 2004; 125: 749-755.
17. Rabusin M, Andolina M, Maximova N, Lepore L, Parco S, Tuveri G et al Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease. Haematologica 2000; 85: 81-85.
18. Gore BA, Fasht A. The natural history of juvenile chronic arthritis: A population based cohort study. II. Outcome. J Rheumatol 1995; 22: 308-319.
19. Petty RE. Prognosis in children with rheumatic disease; justification for consideration of new therapies. Rheumatology (Oxford) 1999; 38: 739-742.
20. Quartier P, Taupin P, Bourdeau F, Lemelle I, Pillet P, Bost M et al Efficacy of etanercept for the treatment of JIA according to the onset type. Arthritis Rheum 2003; 48: 1093-1101.
21. Yokota S, Miyamae T, Imagawa T, Iwata N, Katakura S, Mori M et al. Therapeutic efficacy of humanized recombinant anti-interleukin-6 receptor antibody in children with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum 2005; 52: 818-825.
22. Cervera R, Khamashta MA, Font J, Sebastiani GD, Gil A, Lavilla P et al. Morbidity and mortality in SLE during a 5-year period: A multicenter prospective study of 1000 patients. Medicine 1999; 78: 167-175.
23. Goldblatt F, Isenberg DA. New therapies for systemic lupus erythematosus. Clin Exp Immunol 2005; 140: 205-212.
24. Doria A, Iaccarino L, Ghirardello A, Zampieri S, Arienti S, Satzi-Puttini P et al. Long-term prognosis and causes of death in systemic lupus erythematosus. Am J Med 2006; 119: 700-706.
25. Chen J, Wang Y, Kunkel G, Zhao H, Xue H, Xie X 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.
26. Foeldvari I, Zhavania M, Birch N, Cuttica RJ, de Oliveira SHF, Dent PB et al. Favourable outcome in 135 children with juvenile systemic sclerosis: Results of a multinational survey. Rheumatology 2000; 39: 556-569.
27. Martini G, Foeldvari I, Russo R, Cuttica R, Eberhard A, Ravelli A et al. Systemic sclerosis in childhood: Clinical and immunological features of 153 patients in an international database. Arthritis Rheum 2006; 54: 3971-3978.
28. Raetz E, Beatty PG, Adams RH. Treatment of Evans's syndrome with an allogeneic cord blood transplantation. Bone Marrow Transplant 1997; 20: 427-429.
29. Muller BU, Tichelli A, Passweg JR, Nissen C, Wodnar-Filipowicz A, Gratwohl A. Successful treatment of refractory acquired pure red cell aplasia (PRCA) by allogeneic bone marrow transplantation. Bone Marrow Transplant 1999; 23: 1205-1207.
30. De Stefano P, Zecca M, Giorgiani G, Perotti C, Giraldi E, Locatelli F. Resolution of immune haemolytic anaemia with allogeneic bone marrow transplantation after an unsuccessful autograft. Br J Haematol 1999; 106: 1063-1064.
31. Pallard C, Kanold J, Halle P, Yakouben K, Boiret N, Rapatel C et al Two-step immunoablative treatment with autologous PBSC transplantation in an 8-year-old boy with autoimmune haemolytic anaemia. Br J Haematol 2000; 110: 900-902.
32. Seeliger S, Baumann M, Mohr M, Jurgens H, Frosch M, Vormoor J. Autologous peripheral blood stem cell transplantation and anti-B-cell directed immunotherapy for refractory autoimmune haemolytic anaemia. Eur J Pediatr 2001; 160: 492-496.
33. Cosgrove M, Al-Atlas RI, Jenkins R. Epidemiology of paediatric inflammatory bowel disease. Arch Dis Child 1996; 74: 460-461.
34. Bonen DK, Clio JH. The genetics of inflammatory bowel disease. Gastroenterology 2003; 124: 521-536.
35. Peppelenbosch MP, van Deventer SJ. T cell apoptosis and inflammatory bowel disease. Gut 2004; 53: 1556-1558.
36. Oyania Y, Craig RM, Traynor AE, Quigley K, Statkute L, Halverson A et al. Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease. Gastroenterology 2005; 128: 552-563.
37. de Kleer I, Vastert B, Klein M, Teklenburg G, Arkesteijn G, Yung GP 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.
38. Marmont AM. Is there any evidence of a graft versus-autoimmunity effect in allogeneic transplantation? Blood Marrow Transplant Rev 2004; 4: 11.
39. Marmont AM, Gualandi F, Occhini D, Morandi F, Ferretti E, Pezzolo A et al. Catastrophic relapse of Evans syndrome five years after allogeneic BMT notwithstanding full donor chimerism. Terminal hemolytic-uremic syndrome. Autoimmunity 2006; 39: 505-511.