Hematopoietic stem cell transplantation for systemic sclerosis: History and current status

Current Opinion in Rheumatology

Volumn 23, Issue 6, 2011, Pages 519-529

  Burt, Richard K ^a   Milanettib, Francesca ^b  

^a NORTHWESTERN UNIVERSITY   (United States)

^b SAPIENZA UNIVERSITY OF ROME   (Italy)

Author keywords

Hematopoietic stem cell; Scleroderma; Systemic sclerosis; Transplantation

Indexed keywords

AZATHIOPRINE; CHLOROQUINE; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN; GRANULOCYTE COLONY STIMULATING FACTOR; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENICILLAMINE; PREDNISONE; RITUXIMAB; THYMOCYTE ANTIBODY;

ANGIOGENESIS; AUTOIMMUNE DISEASE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CYTOKINE RELEASE SYNDROME; DISEASE SEVERITY; DRUG MEGADOSE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INFECTION; INTERSTITIAL LUNG DISEASE; LUNG EDEMA; LUNG INJURY; LYMPHOCYTOPENIA; MORTALITY; NEPHROTOXICITY; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SKIN FIBROSIS; SYSTEMIC SCLEROSIS;

CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; IMMUNOSUPPRESSIVE AGENTS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SCLERODERMA, SYSTEMIC; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, AUTOLOGOUS;

EID: 82955187639     PISSN: 10408711     EISSN: 15316963     Source Type: Journal    
DOI: 10.1097/BOR.0b013e32834aa45f     Document Type: Review

References (83)

1. LeRoy EC, Black C, Fleischmajer R, et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol 1988; 15:202-205. (Pubitemid 18073738)
2. Ioannidis JP, Vlachoyiannopoulos PG, Haidich AB, et al. Mortality in systemic sclerosis: an international meta-analysis of individual patient data. Am J Med 2005; 118:2-10. (Pubitemid 40082450)
3. Ferri C, Valentini G, Cozzi F, et al. Systemic sclerosis: demographic, clinical, and serologic features and survival in 1 012 Italian patients. Medicine(Baltimore) 2002; 81:139-153. (Pubitemid 34218569)
4. Scussel-Lonzetti L, Joyal F, Raynauld JP, et al. Predicting mortality in systemic sclerosis: analysis of a cohort of 309 French Canadian patients with emphasis on features at diagnosis as predictive factors for survival. Medicine (Baltimore) 2002; 81:154-167. (Pubitemid 34218570)
5. Bryan C, Knight C, Black CM, 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; 42:2660-2665. (Pubitemid 30327188)
6. Czirják L, Nagy Z, Szegedi Gy. Survival analysis of 118 patients with systemic sclerosis. J Intern Med 1993; 234:335-337. (Pubitemid 23263424)
7. Nagy Z, Czirják L. Predictors of survival in 171 patients with systemic sclerosis (scleroderma). Clin Rheumatol 1997; 16:454-460. (Pubitemid 27425422)
8. Nagy Z, Czirják L. Increased C reactive protein and aminoterminal propeptide of type III procollagen levels are unfavourable predictors of survival in systemic sclerosis (scleroderma). Clin Exp Rheumatol 2005; 23:165-172. (Pubitemid 40662995)
9. Jacobsen S, Ullman S, Shen GQ, et al. Influence of clinical features, serum antinuclear antibodies, and lung function on survival of patients with systemic sclerosis. J Rheumatol 2001; 28:2454-2459. (Pubitemid 33031976)
10. Hesselstrand R, Scheja A, Akesson A. Mortality and causes of death in a Swedish series of systemic sclerosis patients. Ann Rheum Dis 1998; 57:682-686. (Pubitemid 28558973)
11. Jacobsen S, Halberg P, Ullman S. Mortality and causes of death of 344 Danish patients with systemic sclerosis (scleroderma). Br J Rheumatol 1998; 37:750-755.
12. Al-Dhaher FF, Pope JE, Ouimet JM. Determinants of morbidity and mortality of systemic sclerosis in Canada. Semin Arthritis Rheum 2010; 39:269-277.
13. Czirják L, Kumánovics G, Varjú C, et al. Survival and causes of death in 366 Hungarian patients with systemic sclerosis. Ann Rheum Dis 2008; 67:59-63.
14. Wynn J, Fineberg N, Matzer L, et al. Prediction of survival in progressive systemic sclerosis by multivariate analysis of clinical features. Am Heart J 1985; 110:123-127. (Pubitemid 16119691)
15. Zarafonetis CJ, Dabich L, Negri D, et al. Retrospective studies in scleroderma: effect of potassium para-aminobenzoate on survival. J Clin Epidemiol 1988; 41:193-205. (Pubitemid 18069579)
16. Medsger TA Jr, Masi AT. Survival with scleroderma II. A life-table analysis of clinical and demographic factors in 358 male U.S. veteran patients. J Chron Dis 1973; 26:647-660.
17. Simeon CP, Armadans L, Fonollosa V, et al. Mortality and prognostic factors in Spanish patients with systemic sclerosis. Ann Rheum Dis 2003; 42:71-75. (Pubitemid 36131570)
18. Steen VD, Medsger TA Jr. Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 2000; 43:2437-2444.
19. Bulpitt KJ, Clements PJ, Lachenbruch PA, et al. Early undifferentiated connective tissue disease: III. Outcome and prognostic indicators in early scleroderma (systemic sclerosis). Ann Intern Med 1993; 118:602-609.
20. Farmer RG, Gifford RW, Hines EA. Prognostic significance of Raynaud's phenomenon and other clinical characteristics of systemic scleroderma. Circulation 1960; 21:1088-1095.
21. Geirsson ÁJ, Wollheim FA, Akesson A. Disease severity of 100 patients with systemic sclerosis over a period of 14 years: using a modified Medsger scale. Ann Rheum Dis 2001; 60:1117-1122. (Pubitemid 33099994)
22. Clements PJ, Hurwitz EL, Wong WK, et al. Skin thickness score as a predictor and correlate of outcome in systemic sclerosis. Arthritis Rheum 2000; 43:2445-2454.
23. Majewski S, Wojas-Pelc A, Malejczyk M, et al. Serum levels of soluble TNF alpha receptor type I and the severity of systemic sclerosis. Acta Derm Venereol 1999; 79:207-210. (Pubitemid 29251507)
24. Giordano M, Valentini G, Migliaresi S, et al. Different antibody patterns and different prognosis in patients with scleroderma with various extent of skin sclerosis. J Rheumatol 1986; 13:911-916. (Pubitemid 17203925)
25. Tamaki T, Mori S, Takehara K. Epidemiological study of patients with systemic sclerosis in Tokyo. Arch Dermatol Res 1991; 283:366-371.
26. Shand L, Lunt M, Nihtynova S, et al. Relationship between change in skin score and disease outcome in diffuse cutaneous systemic sclerosis. Application of a latent linear trajectory model. Arthritis Rheum 2007; 56:2422-2431. (Pubitemid 47173590)
27. Steen VD, Medsger TA Jr. Improvement in skin thickening in systemic sclerosis associated with improved survival. Arthritis Rheum 2001; 44:2828-2835. (Pubitemid 34016202)
28. Hachulla E, de Groote P, Gressin V, et al. The three-year incidence of pulmonary arterial hypertension associated with systemic sclerosis in a multicenter nationwide longitudinal study in France. Arthritis Rheum 2009; 60:1831-1839.
29. Beretta L, Santaniello A, Cappiello F, et al. Development of a five-year mortality model in systemic sclerosis patients by different analytical approaches. Clin Exp Rheumatol 2010; 28 (2 Suppl 58):S18-S27.
30. Tyndall AJ, Bannert B, Vonk M, et al. Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) database. Ann Rheum Dis 2010; 69:1809-1815.
31. Kuo CF, See LC, Yu KH et al. Epidemiology and mortality of systemic sclerosis: a nationwide population study in Taiwan. Scand J Rheumatol 2011. [Epub ahead of print].
32. Milanetti F, Bucha J, Testori A, Burt RK. Autologous hematopoietic stem cell transplantation for systemic sclerosis. Curr Stem Cell Res Ther. 2011; 6:16-28.
33. Valentini G, Della Rossa A, Bombardieri S, et al. European multicentre study to define disease activity criteria for systemic sclerosis II. Identification of disease activity variables and development of preliminary activity indices. Ann Rheum Dis 2001; 60:592-598. (Pubitemid 32493986)
34. Pope JE, Bellamy N, Seibold JR, et al. A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001; 44:1351-1358. (Pubitemid 32537533)
35. Van Den Hoogen FH, Boerbooms AM, Swaak AJ, et al. Comparison of methotrexate with placebo in the treatment of systemic sclerosis: a 24-week randomized double-blind trial, followed by a 24-week observational trial. Br J Rheumatol 1996; 35:364-372.
36. Clements PJ, Furst DE, Wong WK, et al. High-dose versus low-dose Dpenicillamine in early diffuse systemic sclerosis: analysis of a two-year, double-blind, randomized, controlled clinical trial. Arthritis Rheum 1999; 42:1194-1203. (Pubitemid 29268956)
37. Daoussis D, Liossis SN, Tsamandas AC, et al. Experience with rituximab in scleroderma: results from a 1-year, proof-of-principle study. Rheumatology (Oxford) 2010; 49:271-280.
38. Steen VD, Medsger TA Jr. Case-control study of corticosteroids and other drugs that either precipitate or protect from the development of scleroderma renal crisis. Arthritis Rheum 1998; 41:1613-1619. (Pubitemid 28459919)
39. Clements PJ, Sterz M, Danovitch G, et al. Cyclosporin (CsA) in systemic sclerosis (SSc): results of a 48-week open safety study in 10 SSc patients. Arthritis Rheum 1993; 36:75-83. (Pubitemid 23048682)
40. Tashkin DP, Elashoff R, Clements PJ, et al. Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 2006; 354:2655-2666.
41. Tashkin DP, Elashoff R, Clements PJ, et al. Effects of 1-year treatment with cyclophosphamide on outcomes at 2 years in scleroderma lung disease. Am J Respir Crit Care Med 2007; 176:1026-1034. (Pubitemid 350127847)
42. Nadashkevich O, Davis P, Fritzler M, Kovalenko W. A randomized unblinded trial of cyclophosphamide versus azathioprine in the treatment of systemic sclerosis. Clin Rheumatol 2006; 25:205-212. (Pubitemid 43372319)
43. Nannini C, West CP, Erwin PJ, Matteson EL. Effects of cyclophosphamide on pulmonary function in patients with scleroderma and interstitial lung disease: a systematic review and meta-analysis of randomized controlled trials and observational prospective cohort studies. Arthritis Res Ther 2008; 10:R124.
44. Hunzelmann N, Moinzadeh P, Genth E, et al. High frequency of corticosteroid and immunosuppressive therapy in patients with systemic sclerosis despite limited evidence for efficacy. Arthritis Res Ther 2009; 11:R30.
45. Pope JE, Ouimet JM, Krizova A. Scleroderma treatment differs between experts and general rheumatologists. Arthritis Rheum 2006; 55:138-145. (Pubitemid 43235139)
46. Frère P, Pereira M, Fillet G, Beguin Y. Infections after CD34-selected or unmanipulated autologous hematopoietic stem cell transplantation. Eur J Haematol 2006; 76:102-108.
47. Kohno K, Nagafuji K, Tsukamoto H, et al. Infectious complications in patients receiving autologous CD34-selected hematopoietic stem cell transplantation for severe autoimmune diseases. Transpl Infect Dis 2009; 11:318-323.
48. Schwartz RH. T cell anergy. Annu Rev Immunol 2003; 21:305-334. (Pubitemid 37174534)
49. Burt RK, Loh Y, Pearce W, et al. J. Clinical applications of blood-derived and marrow-derived stem cells for nonmalignant diseases. JAMA 2008; 299:925-936. (Pubitemid 351397068)
50. 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. (Pubitemid 40395124)
51. Zhang L, Bertucci AM, Ramsey-Goldman R, et al. Regulatory T cell (TReg) subsets return in patients with refractory lupus following adult stem cell transplantation: TGF-b producing CD81 Treg (CD8TGF-b Treg) are associated with suppressing lupus into immunologic remission. J immunol 2009; 183:6346-6358.
52. 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 2009; 113:214-223.
53. Tsukamoto H, Nagafuji K, Horiuchi T, et al. Analysis of immune reconstitution after autologous CD34 stem/progenitor cell transplantation for systemic sclerosis: predominant reconstitution of Th1 CD4 T cells. Rheumatology (Oxford) 2011; 50:944-952.
54. Bohgaki T, Atsumi T, Bohgaki M, et al. Immunological reconstitution after autologous hematopoietic stem cell transplantation in patients with systemic sclerosis: relationship between clinical benefits and intensity of immunosuppression. J Rheumatol 2009; 36:1240-1248.
55. Burt RK, Abinun M, Farge-Bancel D, et al. Risks of immune system treatments. Science 2010; 328:825-826.
56. 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. Arthritis Rheum 2006; 54:3750-3760. (Pubitemid 44955875)
57. Montoto S, Canals C, Rohatiner AZ, et al. Long-term follow-up of high-dose treatment with autologous haematopoietic progenitor cell support in 693 patients with follicular lymphoma: an EBMT registry study. Leukemia 2007; 21:2324-2331. (Pubitemid 350011705)
58. Emerit I, Housset J, De Grouchy J, Carnus JP. Chromosomal breakage in diffuse scleroderma. A study of 27 patients. Europ J Clin Biol Res 1971; 16:683-694.
59. Pan SF, Rodnan GP, Seutsch M, Wald N. Chromosomal abnormalities in progressive systemic sclerosis (scleroderma) with consideration of radiation effects. J Lab Clin Med 1975; 86:300-308.
60. Rittner G, Schwanitz G, Baur MP, et al. Family studies in scleroderma (systemic sclerosis) demonstrating an HLA-linked increased chromosomal breakage rate in cultured lymphocytes. Hum Genet 1988; 81:64-70. (Pubitemid 19014127)
61. Migliore L, Bevilacqua C, Scarpato R. Cytogenetic study and FISH analysis in lymphocytes of systemic lupus erythematosus and systemic sclerosis patients. Mutagenesis 1999; 14:227-231. (Pubitemid 29123789)
62. Artlett CM, Black CM, Briggs DC, et al. Telomere reduction in scleroderma patients: a possible cause for chromosomal instability. Br J Rheumatol 1996; 35:732-737.
63. Wolff DJ, Needleman BW, Wasserman SS, Schwarts S. Spontaneous and clastogen induced chromosomal breakage in scleroderma. J Rheumatol 1991; 18:837-840.
64. Binks M, Passweg JR, Furst D, 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; 60:577-584. (Pubitemid 32493984)
65. Farge D, Marolleau JP, Zohar S, et al. Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I-II study. Br J Haematol 2002; 119:726-739. (Pubitemid 35366216)
66. McSweeney PA, Nash RA, Sullivan KM, et al. High-dose immunosuppressive therapy for severe systemic sclerosis: initial outcomes. Blood 2002; 100:1602-1610. (Pubitemid 34925135)
67. Nash RA, McSweeney PA, Crofford LJ, et al. High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study. Blood 2007; 110:1388-1396. (Pubitemid 47281441)
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. (Pubitemid 38961924)
69. Tsukamoto H, Nagafuji K, Horiuchi T, et al. A phase I-II trial of autologous peripheral blood stem cell transplantation in the treatment of refractory autoimmune disease. Ann Rheum Dis 2006; 65:508-514.
70. Oyama Y, Barr WG, Statkute L, et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with systemic sclerosis. Bone Marrow Transplant 2007; 40:549-555. (Pubitemid 47382125)
71. 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.
72. Snowden JA, Saccardi RM, Allez S, et al. Haematopoietic stem cell transplantation (HSCT) in severe autoimmune diseases (ADs): updated guidelines of the European Group for Blood and Marrow Transplantation (EBMT). Submitted.
73. Forfia PR, Fisher MR, Mathai SC, et al. Tricuspid annular displacement predicts survival in pulmonary hypertension. Am J Respir Crit Care Med 2006; 174:1034-1041. (Pubitemid 44684453)
74. Englert H, Kirkham S, Moore J, et al. Autologous stem cell transplantation in diffuse scleroderma: impact on hand structure and function. Intern Med J 2008; 38:692-696.
75. Taniguchi Y, Frickhofen N, Raghavachar A, et al. Antilymphocyte immunoglobulins stimulate peripheral blood lymphocytes to proliferate and release lymphokines. Eur J Haematol 1990; 44:244-251. (Pubitemid 20203067)
76. Wing M. Monoclonal antibody first dose cytokine release syndromes-mechanisms and prediction. J Immunotoxicol 2008; 5:11-15. (Pubitemid 351431265)
77. Yabuuchi H, Matsuo Y, Tsukamoto H, et al. Correlation between pretreatment or follow-up CT findings and therapeutic effect of autologous peripheral blood stem cell transplantation for interstitial pneumonia associated with systemic sclerosis. Eur J Radiol 2011 [Epub ahead of print].
78. Verrecchia F, Laboureau J, Verola O, et al. Skin involvement in scleroderma- where histological and clinical scores meet. Rheumatology (Oxford) 2007; 46:833-841. (Pubitemid 47061718)
79. Miniati I, Guiducci S, Conforti ML, et al. Autologous stem cell transplantation improves microcirculation in systemic sclerosis. Ann Rheum Dis 2009; 68:94-98.
80. Fleming JN, Nash RA, McLeod DO, et al. Capillary regeneration in scleroderma: stem cell therapy reverses phenotype? PLoS ONE 2008; 3:e1452.
81. http://www.astistrial.com.
82. http://www.clinicaltrials.gov(NCT00114530).
83. http://www.clinicaltrials.gov(NCT00278525).