Autologous stem cell transplantation in the treatment of systemic sclerosis: Report from the EBMT/EULAR Registry

Annals of the Rheumatic Diseases

Volumn 63, Issue 8, 2004, Pages 974-981

  Farge, D ^a   Passweg, J ^l   Van Laar, J M ^b   Marjanovic, Z ^a   Besenthal, C ^c   Finke, J ^c   Peter, H H ^c   Breedveld, F C ^b   Fibbe, W E ^b   Black, C ^o   Denton, C ^o   Koetter, I ^s   Locatelli, F ^p   Martini, A ^k   Schattenberg, A V N ^v   Van Den Hoogen, F ^v   Van De Putte, L ^v   Lanza, F ^r   Arnold, R ^e   Bacon, P A ^u   Bingham, S ^t   Ciceri, F ^q   Didier, B ^j   Diez Martin, J L ^h   Emery, P ^t   Feremans, W ^f   Hertenstein, B ^m   Hiepe, F ^e   Luosujarvi R ^w   Leon Lara, A ^g   Marmont, A ⁿ   Martinez, A M ⁱ   Pascual Cascon, H ^d   Bocelli Tyndall, C ^l   Gluckman, E ^a   Gratwohl, A ^l   Tyndall, A ^l   more..

^a SAINT LOUIS HOSPITAL   (France)

^b LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^c UNIVERSITY OF FREIBURG   (Germany)

^d HOSPITAL REGIONAL UNIVERSITARIO DE MÁLAGA   (Spain)

^e CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^f ERASME HOSPITAL   (Belgium)

^g Hospital del SAS   (Spain)

^h HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑÓN   (Spain)

ⁱ HOSPITAL INFANTIL LA PAZ   (Spain)

^j INSTITUT PAOLI CALMETTES   (France)

^k G GASLINI INSTITUTE   (Italy)

^l UNIVERSITY HOSPITAL BASEL   (Switzerland)

^m HANNOVER MEDICAL SCHOOL   (Germany)

ⁿ UNIVERSITY OF GENOA   (Italy)

^o ROYAL FREE HOSPITAL   (United Kingdom)

^p FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^q SAN RAFFAELE HOSPITAL   (Italy)

^r UNIVERSITY HOSPITAL OF FERRARA   (Italy)

^s UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^t University Hospital   (United Kingdom)

^u UNIVERSITY HOSPITALS BIRMINGHAM NHS FOUNDATION TRUST   (United Kingdom)

^v RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^w KUOPIO UNIVERSITY HOSPITAL   (Finland)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; CARMUSTINE; CYCLOPHOSPHAMIDE; CYTARABINE; ETOPOSIDE; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; MELPHALAN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; THIOTEPA; THYMOCYTE ANTIBODY;

ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CAUSE OF DEATH; CLINICAL TRIAL; DISEASE COURSE; DISEASE SEVERITY; EXPERIENCE; FOLLOW UP; GRAFT REJECTION; HUMAN; KAPLAN MEIER METHOD; LYMPH NODE IRRADIATION; MORTALITY; PRIORITY JOURNAL; PROBABILITY; REGISTER; SURVIVAL TIME; SYSTEMIC SCLEROSIS; TREATMENT OUTCOME; WHOLE BODY RADIATION;

ADOLESCENT; ADULT; AGED; CHILD; CLINICAL TRIALS, PHASE I; CLINICAL TRIALS, PHASE II; EUROPE; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; MIDDLE AGED; REGISTRIES; SCLERODERMA, SYSTEMIC; SEVERITY OF ILLNESS INDEX; SKIN; SURVIVAL ANALYSIS; TREATMENT OUTCOME; VENTRICULAR FUNCTION, LEFT; VITAL CAPACITY;

EID: 3242658935     PISSN: 00034967     EISSN: None     Source Type: Journal    
DOI: 10.1136/ard.2003.011205     Document Type: Article

References (63)

1. D Furst, Clements PJ. Hypothesis for the pothogenesis of SSc. J Rheumatol 1997;24:53-7.
2. Le Roy EC, Black CM, Fleischmajer R, Jablonska S, Krieg T, Medsger TA, et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol 1998;15:202-5.
3. Wilson TJ, Van de Water J, Mohr FC, Boyd RL, Ansari A, Wick G, et al. Avian scleroderma: evidence for qualitative and quantitative T cell defects. J Autoimmun 1992;5:261-76.
4. Van Bekkum D. Short analytical review: new opportunities for the treatment of severe autoimmune diseases: bone marrow transplantation. Clin Immunol Immunopathol 1998;89:1-10.
5. Wells AU, Lorimer S, Majumdar S, Harrison NK, Corrin B, Black CM, et al. Fibrosing alveolitis in systemic sclerosis: increase in memory T-cells in lung interstitium. Eur Respir J 1995;8:266-71.
6. Yurovsky W, Wigley FM, Wise RA, White B. Skewing of the CD8+ T-cell repertoire in the lungs of patients with systemic sclerosis. Hum Immunol 1996;48:84-97.
7. Denton CP, Xu S, Welsh KI, Pearson JD, Black CM. Scleroderma fibroblast phenotype is modulated by endothelial coculture. J Rheumatol 1996;23:633-8.
8. Sakkas Li, Xu B, Arlett CM, Lu S, Jimenez SA, Plastoucas CD. Oligoclonal T cell expansion in the skin of patients with systemic sclerosis. J Immunol 2002;168:3649-59.
9. Giordano M, Valentini G, Migliaresi S, Picillo U, Vatti M. Different antibody patterns and different prognoses in patients with various extent of skin sclerosis. J Rheumatol 1986;13:911-16.
10. Negi VS, Tripathy NK, Misra R, Nityanand S. Antiendothelial cell antibodies in scleroderma correlate with severe digital ischemia and pulmonary arterial hypertension. J Rheumatol 1998;25:462-6.
11. Altman RD, Medsger TA Jr, Bloch DA, Michel BA. Predictors of survival in systemic sclerosis (scleroderma). Arthritis Rheum 1991;34:403-13.
12. Bulpitt KJ, Clements PJ, Lachenbruch PA, Paulus HE, Peter JB, Agopian MS, et al. Early differentiated connective tissue disease: III. Outcome and prognostic indicators in early scleroderma (systemic sclerosis). Ann Intern Med 1993;118:602-9.
13. Bryan C, Knight C, Black M, Silman AJ. Prediction of five-year survival following presentation with scleroderma. Development of a simple model using three disease factors at first visit. Arthritis Rheum 1999;42:2660-5.
14. Ferri C, Valentini G, Cozzi F, Sebastiani, Michelassi C, La Montagna G, et al. Systemic sclerosis. Demographic, clinical, and serologic features and survival in 1012 Italian patients. Medicine (Baltimore) 2002;81:139-53.
15. Scussel-Lonzetti L, Joyal F, Raynauld JP, Roussin A, Rich E, Goulet JR, et al. Predicting mortality in systemic sclerosis. Analysis of cohort of 309 French Canadian patients with emphasis on features at diagnosis as predictive factors for survival. Medicine (Baltimore) 2002;81:154-67.
16. Steen VD, Medsger TA Jr. Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 2000;43:2437-44.
17. Silver RM, Warrick JH, Kinsella MB, Staudt LS, Baumann MH, Strange C. Cyclophosphamide and low-dose prednisone therapy in patients with systemic sclerosis (scleroderma) with interstitial lung disease. J Rheumatol 1993;20:838-44.
18. Akesson A, Scheja A, Lundin A, Wollheim FA. Improved pulmonary function in systemic sclerosis after treatment with cyclophosphamide. Arthritis Rheum 1994;37:729-35.
19. Várai G, Earle L, Jimenez SA, Steiner RM, Varga J. A pilot study of intermittent intravenous cylcophosphamide for the treatment of systemic sclerosis associated lung disease. J Rheumatol 1998;25:1325-9.
20. Davas EM, Peppas C, Maragou M, Alvanou E, Hondros D, Dantis PC. Intravenous cyclophosphamide pulse therapy for the treatment of lung disease associated with scleroderma. Clin Rheumatol 1999;18:455-61.
21. Griffiths B, Miles S, Moss H, Robertson R, Veale D, Emery P. Systemic sclerosis and interstitial lung disease: a pilot study using pulse intravenous methyprednisolone and cyclophosphamide to assess the effect on high resolution computed tomography scan and lung function. J Rheumatol 2002;29:2371-8.
22. Schnabel A, Reuter M, Gross WL. Intravenous pulse cyclophosphamide in the treatment of interstitial lung disease due to collagen vascular diseases. Arthritis Rheum 1998;41:1215-20.
23. White B, Moore WC, Wigley FM, Xiao HQ, Wise RA. Cyclophosphamide is associated with pulmonary function and survival benefit in patients with scleroderma and alveolitis. Ann Intern Med 2000;132:947-54.
24. Marmont AM. Immune ablation followed by allogeneic or autologous bone marrow transplantation: a new treatment for severe autoimmune diseases? Stem Cells 1994;12:125-35.
25. Marmont AM, Tyndall A, Gratwhol A, Vischer T. Hematopoietic precursor cell transplants for autoimmune diseases. Lancet 1995;345:978.
26. Van de Putte LBA, Tyndall A, Van den Hoogen FJH, Smolen JS. Hematopoietic stem cell transplants for autoimmune disease: role of EULAR. J Rheumatol 1997;24:98-9.
27. Tyndall A, Gratwohl A. Hematopoietic stem and progenitor cells in the treament of severe autoimmune diseases. Ann Rheum Dis 1996;55:149-51.
28. Tyndall A, Gratwohl A. Blood and marrow stem cell transplants in autoimmune disease: a consensus report written on behalf of the European League Against Rheumatism (EULAR] and the European Group for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant 1997;19:643-5.
29. Ikehara S, Good RA, Nakamura T, Sekita K, Inoue S, Oo MM, et al. Rationale for bone marrow transplantation in the treatment of autoimmune diseases. Proc Natl Acad Sci USA 1985;82:2483-7.
30. Marmont AM. New horizons in the treatment of autoimmune diseases: immunoablation and stem cell transplantation. Annu Rev Med 2000;51:115-34.
31. Good RA, Ikehara S. Preclinical investigations that subserve efforts to employ bone marrow transplantation for rheumatoid or autoimmune diseases. J Rheumatol 1997;24:5-12.
32. Marmont AM. Immunoablation followed or not by hematopoietic stem cells as an intense therapy for severe autoimmune diseases. New perspectives, new problems. Haematologica 2001;86:337-45.
33. Farge D, Gluckman E, Tyndall A. Treatment of severe autoimmune diseases by immunoablative chemotherapy and autologous bone marrow transplantation. Eur J Intern Med 1999;10:88-96.
34. Storb R. Hematopoietic stem cell transplantation in non malignant diseases. J Rheumatol 1997;24:30-5.
35. Jantunen E, Myllykangas-Luosujärvi R. Stem cell transplantation for treatment of severe autoimmune diseases: current status-future perspectives. Bone Marrow Transplant 2000;25:351-6.
36. Binks M, Passweg JR, Furst D, McSweeney P, Sullivan K, Besenthal C, et al. Phase I/II trial of autologous stem cell transplantation in systemic sclerosis: procedure related mortality and impact on skin disease. Ann Rheum Dis 2001;60:577-84.
37. McSweeney PA, Nash RA, Sullivan KM, Storek J, Crofford LJ, Dansey R, et al. High-dose immunosuppressive therapy for severe systemic sclerosis: initial outcomes. Blood 2002;100:1602-10.
38. Farge D, Marolleau JP, Zohor S, Marjanovic Z, Cabane J, Mounier N, for the Intensification et Autogreffe dans les Maladies Auto Immunes Résistantes (ISAMAIR) study group, et al. Autologous bone marrow transplantation in systemic sclerosis: early results from a multicenter phase I-II study. Br J Haematol 2002;119:726-39.
39. Tyndall A, Fassas A, Passweg J, Ruiz de Elvira C, Attal M, Brooks P, et al. Autologous haematopoietic stem cell transplants for autoimmune disease-feasibility and transplant-related mortality. Bone Marrow Transplant 1999;24:729-34.
40. Clements P, Lachenbruch P, Seibold JR, White B, Weiner S, Martin R, et al. Inter- and intraobserver variability of total skin thickness score (modified Rodan TSS) in systemic sclerosis. J Rheumatol 1995;22:1281-5.
41. Kahaleh MB, Sultany GL, Smith EA, Huffsutteter JE, Loaholt CB, Leray C. A modified scleroderma skin scoring method. Clin Exp Rheumatol 1986;4:367-9.
42. Clements PJ, Hurtwitz EL, Wong WK, Seibold JR, Mayes M, White B, et al. High dose versus low dose D-penicillamine in early diffuse systemic sclerosisanalysis of a two year, double blind, randomised, controlled clinical trail. Arthritis Rheum 2000;43:2445-54.
43. Kaplan EL, Meier P. Nonparametric estimations from incomplete observations. J Am Stat Assoc 2001;53:457-81.
44. Tyndall A, Gratwohl A. Immune ablation and stem-cell therapy in autoimmune disease clinical experience. Arthritis Res 2000;2:276-80.
45. Tyndall A, Passweg J, Gratwohl A. Hematopoietic stem cell transplantation in the treatment of severe autoimmune diseases. Ann Rheum Dis 2001;60:577-84.
46. Waters TM, Bennett CL, Pajeau TS, Sobocinsky KA, Klein JP, Rowlings PA, et al. Economic analyses of bone marrow and blood stem cell transplantation for leukemias and lymphoma: what do we know? Bone Marrow Transplant 1998;21:641-50.
47. Laffel GL, Barnett AI, Finkelstein S, Kaye MP. The relation between experience and outcome in heart transplantation. N Engl J Med 1992;327:1220-5.
48. Denton CP, Cailes JB, Phillips GD, Wells AU, Black CM, Du Bois RM. Comparison of doppler echocardiography and right heart catheterization to assess pulmonary hypertension in systemic sclerosis. Br J Rheumatol 1997;36:239-43.
49. Naik MM, Diamond GA, Pai T, Soffer A, Siegel RJ. Correspondence of left ventricular ejection fraction determinations from two-dimensional echocardiography, radionuclide angiography and contrast cineangiography. J Am Coll Cardiol 1995;25:937-42.
50. Snowden JA, Biggs JC, Milliken ST, Fuller A, Staniforth D, Passuello F, et al. A randomised, blinded, placebo-controlled, dose escalation study of the tolerability and efficacy of filgrastim for haemopoietic stem cell mobilisation in patients with severe active rheumatoid arthritis. Bone Marrow Transplant 1998;22:1035-41.
51. Snowden JA, Biggs SC, Milliken ST, Fuller A, Brooks PM. A phase I-II escalation study of intensified cyclophophamide and autologous blood stem cell rescue in severe, active rheumatoid arthritis. Arthritis Rheum 1999;42:2277-80.
52. Openshaw H, Stuve O, Antel JP, Lundt BT, Weinet LP, Kashyap A. Multiple sclerosis flares associated with recombinant granulocyte colony-stimulating factor. Neurology 2000;54:2147-50.
53. Fassas A, Anagnostopoulos A, Kazis A, Kapinas K, Sakellari I, Kimiskidis V, et al. Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis - first results from a pilot study. Bone Morrow Transplant 1997;20:631-8.
54. Burt RK, Traynor AE, Pope R, Schroeder J, Karlin KH, Lobeck L, et al. Treatment of autoimmune disease by intense immunosuppressive conditioning and autologous hematopoietic stem cell transplantation. Blood 1998;92:3505-14.
55. Burt RK, Fassas A, Snowden J, van Laar J, Kozak T, Wulffraat N, Collection of hematopoietic stem cells from patients with autoimmune diseases, et al. Bone Marrow Transplant 2001;28:1-12.
56. Burt RK, Slavin S, Burns H, Marmont A. Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure? Blood 2002;99:768-84.
57. Traynor AE, Schroeder J, Rosa RM, Cheng D, Stefka J, Mujais S, et al. Treatment of severe systemic lupus erythematosus with high-dose chemotherapy and haemopoietic stem-cell transplantation: a phase I study. Lancet 2000;356:701-7.
58. Verburg RJ, Kruize AA, van den Hoogen FH, Fibbe WE, Petersen EJ, Preijers F, et al. High dose chemotherapy and autologous hematopoietic stem cel transplantation in patients with rhumatoid arthritis: results of an open study to assess feasibility, safety and efficacy. Arthritis Rheum 2001;44:754-60.
59. Clements P, Lachenbruch PA, Seibold JR, Zee B, Steen VD, Brennan P, et al. Skin thickness score in systemic sclerosis: an assessment of interobserver variability in 3 independent studies. J Rheumatol 1993;20:1892-6.
60. Nagy Z, Czirjak L. Predictors of survival in 171 patients with systemic sclerosis (scleroderma). Clin Rheumatol 1997;16:454-60.
61. Simeon CP, Armadans L, Fonollosa V, Vilardell M, Candell J, Tolosa C, et al. Survival prognostic factors and markers of morbidity in Spanish patients with systemic sclerosis. Ann Rheum Dis 1997;56:723-8.
62. Euler HH, Marmont AM, Bacigalupo A, Fastenrath S, Dreger P, Hoffnecht M, et al. Early recurrence or persistence of autoimmune disease after unmanipulated autologous stem cell transplantation. Blood 1996;88:3621-5.
63. Straton RJ, Wilson H, Block CM. Pilot study of antithymocyte globulin plus mycophenolate mophetil in recent onset of diffuse scleroderma. Rheumatology (Oxford) 2001;40:84-88.