Autologous Hematopoietic Cell Transplants for Autoimmune Diseases: Specific Diseases and Controversies

Hillard M. Lazarus: Affiliation:
Case Western Reserve University, Ohio
Robert Peter Gale: Affiliation:
Imperial College London
Armand Keating: Affiliation:
University of Toronto
Andrea Bacigalupo: Affiliation:
Ospedale San Martino, Genoa
Reinhold Munker: Affiliation:
Louisiana State University, Shreveport
Kerry Atkinson: Affiliation:
University of Queensland
Syed Ali Abutalib: Affiliation:
Midwestern Regional Medical Center, Cancer Treatment Centers of America, Chicago

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Type: Chapter
Information: Hematopoietic Cell Transplants
Concepts, Controversies and Future Directions
, pp. 535 - 551

DOI: https://doi.org/10.1017/9781316335727.058 [Opens in a new window]

Publisher: Cambridge University Press

Print publication year: 2000

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References

Marmont, AM. Stem cell transplantation for severe autoimmune diseases: progress and problems. Haematologica 1998; 83(8): 733–743.Google Scholar

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). Br J Rheumatol 1997; 36(3): 390–392.Google Scholar

Snowden, JA, Saccardi, R, Allez, M, Ardizzone, S, Arnold, R, Cervera, R et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2012; 47(6): 770–790. doi: 10.1038/bmt.2011.185CrossRef Google Scholar PubMed

Gratwohl, A, Passweg, J, Bocelli-Tyndall, C, Fassas, A, van Laar, JM, Farge, D et al. Autologous hematopoietic stem cell transplantation for autoimmune diseases. Bone Marrow Transplant 2005; 35(9): 869–879. doi: 10.1038/sj.bmt.1704892Google Scholar

Farge, D, Labopin, M, Tyndall, A, Fassas, A, Mancardi, GL, Van Laar, J et al. Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years’ experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica 2010; 95(2): 284–292. doi: 10.3324/haematol.2009.013458Google Scholar

Sun, L. Stem cell transplantation: progress in Asia. Lupus 2010; 19(12): 1468–1473. doi: 10.1177/0961203310370051Google Scholar

Passweg, JR, Baldomero, H, Peters, C, Gaspar, HB, Cesaro, S, Dreger, P et al. Hematopoietic SCT in Europe: data and trends in 2012 with special consideration of pediatric transplantation. Bone Marrow Transplant 2014; 49(6): 744–750. doi: 10.1038/bmt.2014.55Google Scholar

Gratwohl, A, Baldomero, H, Gratwohl, M, Aljurf, M, Bouzas, LF, Horowitz, M et al. Quantitative and qualitative differences in use and trends of hematopoietic stem cell transplantation: a Global Observational Study. Haematologica 2013; 98(8): 1282–1290. doi: 10.3324/haematol.2012.076349Google Scholar

Snowden, JA, Pearce, RM, Lee, J, Kirkland, K, Gilleece, M, Veys, P et al. Haematopoietic stem cell transplantation (HSCT) in severe autoimmune diseases: analysis of UK outcomes from the British Society of Blood and Marrow Transplantation (BSBMT) data registry 1997–2009. Br J Haematol 2012; 157(6): 742–746. doi: 10.1111/j.1365–2141.2012.09122.xGoogle Scholar

Pasquini, MC, Voltarelli, J, Atkins, HL, Hamerschlak, N, Zhong, X, Ahn, KW et al. Transplantation for autoimmune diseases in north and South America: a report of the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant 2012; 18(10): 1471–1478. doi: 10.1016/j.bbmt.2012.06.003Google Scholar

Sullivan, KM, Muraro, P, Tyndall, A. Hematopoietic cell transplantation for autoimmune disease: updates from Europe and the United States. Biol Blood Marrow Transplant 2010; 16(1 Suppl): S48–56. doi: 10.1016/j.bbmt.2009.10.034CrossRef Google Scholar PubMed

Sureda, A, Bader, P, Cesaro, S, Dreger, P, Duarte, RF, Dufour, C et al. Indications for allo- and auto-SCT for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2015. Bone Marrow Transplant 2015; 50(8):1037–1056. doi: 10.1038/bmt.2015.6Google Scholar

Burt, RK, Loh, Y, Cohen, B, Stefoski, D, Stefosky, D, Balabanov, R et al. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. Lancet Neurol 2009; 8(3): 244–253. doi: 10.1016/S1474-4422(09)70017-1CrossRef Google Scholar PubMed

Burt, RK, Balabanov, R, Voltarelli, J, Barreira, A, Burman, J. Autologous hematopoietic stem cell transplantation for multiple sclerosis – if confused or hesitant, remember: ‘treat with standard immune suppressive drugs and if no inflammation, no response’. Mult Scler 2012; 18(6): 772–775. doi: 10.1177/1352458512442993Google Scholar

Reston, JT, Uhl, S, Treadwell, JR, Nash, RA, Schoelles, K. Autologous hematopoietic cell transplantation for multiple sclerosis: a systematic review. Mult Scler 2011; 17(2): 204–213. doi: 10.1177/1352458510383609CrossRef Google Scholar PubMed

Burt, RK, Kozak, T. Hematopoietic stem cell transplantation for multiple sclerosis: finding equipoise. Bone Marrow Transplant 2003; 32 (Suppl 1): S45–48. doi: 10.1038/sj.bmt.1703942Google Scholar

Nash, RA, Bowen, JD, McSweeney, PA, Pavletic, SZ, Maravilla, KR, Park, MS et al. High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Blood 2003; 102(7): 2364–2372. doi: 10.1182/blood-2002-12-3908Google Scholar

Burt, RK, Cohen, B, Rose, J, Petersen, F, Oyama, Y, Stefoski, D et al. Hematopoietic stem cell transplantation for multiple sclerosis. Arch Neurol 2005; 62(6): 860–864. doi: 10.1001/archneur.62.6.860CrossRef Google Scholar PubMed

Saccardi, R, Kozak, T, Bocelli-Tyndall, C, Fassas, A, Kazis, A, Havrdova, E et al. Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database. Mult Scler 2006; 12(6): 814–823.Google Scholar

Saccardi, R, Mancardi, GL, Solari, A, Bosi, A, Bruzzi, P, Di Bartolomeo, P et al. Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life. Blood 2005; 105(6): 2601–2607. doi: 10.1182/blood-2004-08-3205Google Scholar

Hamerschlak, N, Rodrigues, M, Moraes, DA, Oliveira, MC, Stracieri, AB, Pieroni, F et al. Brazilian experience with two conditioning regimens in patients with multiple sclerosis: BEAM/horse ATG and CY/rabbit ATG. Bone Marrow Transplant 2010; 45(2): 239–248. doi: 10.1038/bmt.2009.127Google Scholar

Krasulová, E, Trneny, M, Kozák, T, Vacková, B, Pohlreich, D, Kemlink, D et al. High-dose immunoablation with autologous haematopoietic stem cell transplantation in aggressive multiple sclerosis: a single centre 10-year experience. Mult Scler 2010; 16(6): 685–693. doi: 10.1177/1352458510364538Google Scholar

Xu, J, Ji, BX, Su, L, Dong, HQ, Sun, WL, Wan, SG et al. Clinical outcome of autologous peripheral blood stem cell transplantation in opticospinal and conventional forms of secondary progressive multiple sclerosis in a Chinese population. Ann Hematol 2011; 90(3): 343–348. doi: 10.1007/s00277-010–1071-5Google Scholar

Burt, RK, Balabanov, R, Han, X, Sharrack, B, Morgan, A, Quigley, K et al. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA 2015; 313(3): 275–284. doi: 10.1001/jama.2014.17986CrossRef Google Scholar PubMed

Nash, RA, Hutton, GJ, Racke, MK, Popat, U, Devine, SM, Griffith, LM et al. High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for relapsing-remitting multiple sclerosis (HALT-MS): a 3-year interim report. JAMA Neurol 2015; 72(2): 159–169. doi: 10.1001/jamaneurol.2014.3780Google Scholar

Mancardi, GL, Sormani, MP, Gualandi, F, Saiz, A, Carreras, E, Merelli, E et al. Autologous hematopoietic stem cell transplantation in multiple sclerosis: a phase II trial. Neurology 2015; 84(10): 981–988. doi: 10.1212/WNL.0000000000001329Google Scholar

Burman, J, Iacobaeus, E, Svenningsson, A, Lycke, J, Gunnarsson, M, Nilsson, P et al. Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience. J Neurol Neurosurg Psychiatry 2014; 85(10):1116–1121. doi: 10.1136/jnnp-2013–307207Google Scholar

Carreras, E, Saiz, A, Marín, P, Martínez, C, Rovira, M, Villamor, N et al. CD34+ selected autologous peripheral blood stem cell transplantation for multiple sclerosis: report of toxicity and treatment results at one year of follow-up in 15 patients. Haematologica 2003; 88(3): 306–314.Google Scholar

van Laar, JM, Farge, D, Tyndall, A. Stem cell transplantation: a treatment option for severe systemic sclerosis? Ann Rheum Dis 2008; 67 (Suppl 3): iii35–38. doi: 10.1136/ard.2008.098384CrossRef Google Scholar PubMed

Burt, RK, Oliveira, MC, Shah, SJ, Moraes, DA, Simoes, B, Gheorghiade, M et al. Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis. Lancet 2013; 381(9872): 1116–1124. doi: 10.1016/S0140-6736(12)62114-XGoogle Scholar

Burt, RK, Shah, SJ, Dill, K, Grant, T, Gheorghiade, M, Schroeder, J et al. Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet 2011; 378(9790): 498–506. doi: 10.1016/S0140-6736(11)60982-3Google Scholar

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(6): 577–584.Google Scholar

Farge, D, Marolleau, JP, Zohar, S, Marjanovic, Z, Cabane, J, Mounier, N 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(3): 726–739.Google Scholar

van Laar, JM, Farge, D, Sont, JK, Naraghi, K, Marjanovic, Z, Larghero, J et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 2014; 311(24): 2490–2498. doi: 10.1001/jama.2014.6368Google Scholar

Oyama, 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(3): 552–563.CrossRef Google Scholar PubMed

Burt, RK, Craig, RM, Milanetti, F, Quigley, K, Gozdziak, P, Bucha, J et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: long-term follow-up. Blood 2010; 116(26): 6123–6132. doi: 10.1182/blood-2010-06-292391Google Scholar

Craig, RM, Traynor, A, Oyama, Y, Burt, RK. Hematopoietic stem cell transplantation for severe Crohn’s disease. Bone Marrow Transplant 2003; 32 (Suppl 1): S57–59. doi: 10.1038/sj.bmt.1703945CrossRef Google Scholar PubMed

Nash, RA, McDonald, GB. Crohn disease: remissions but no cure. Blood 2010; 116(26): 5790–5791. doi: 10.1182/blood-2010-09-309252Google Scholar

Hommes, DW, Duijvestein, M, Zelinkova, Z, Stokkers, PC, Ley, MH, Stoker, J et al. Long-term follow-up of autologous hematopoietic stem cell transplantation for severe refractory Crohn’s disease. J Crohns Colitis 2011; 5(6): 543–549. doi: 10.1016/j.crohns.2011.05.004Google Scholar

Burt, RK, Traynor, A, Statkute, L, Barr, WG, Rosa, R, Schroeder, J et al. Nonmyeloablative hematopoietic stem cell transplantation for systemic lupus erythematosus. JAMA 2006; 295(5): 527–535. doi: 10.1001/jama.295.5.527Google Scholar

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(9231): 701–707. doi: 10.1016/S0140-6736(00)02627–1CrossRef Google Scholar PubMed

Alchi, B, Jayne, D, Labopin, M, Demin, A, Sergeevicheva, V, Alexander, T et al. Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: data from the European Group for Blood and Marrow Transplantation registry. Lupus 2013; 22(3): 245–253. doi: 10.1177/0961203312470729Google Scholar

Marmont du Haut Champ, AM. Hematopoietic stem cell transplantation for systemic lupus erythematosus. Clin Dev Immunol 2012; 2012: 380391. doi: 10.1155/2012/380391CrossRef Google Scholar PubMed

Illei, GG, Cervera, R, Burt, RK, Doria, A, Hiepe, F, Jayne, D et al. Current state and future directions of autologous hematopoietic stem cell transplantation in systemic lupus erythematosus. Ann Rheum Dis 2011; 70(12): 2071–2074. doi: 10.1136/ard.2010.148049Google Scholar

Couri, CE, Oliveira, MC, Stracieri, AB, Moraes, DA, Pieroni, F, Barros, GM et al. C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2009; 301(15): 1573–1579. doi: 10.1001/jama.2009.470Google Scholar

Voltarelli, JC, Couri, CE, Stracieri, AB, Oliveira, MC, Moraes, DA, Pieroni, F et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA 2007; 297(14): 1568–1576. doi: 10.1001/jama.297.14.1568Google Scholar

D’Addio, F, Valderrama Vasquez, A, Ben Nasr, M, Franek, E, Zhu, D, Li, L et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in new-onset type 1 diabetes: a multicenter analysis. Diabetes 2014. doi: 10.2337/db14-0295Google Scholar

Burt, RK, Loh, Y, Pearce, W, Beohar, N, Barr, WG, Craig, R et al. Clinical applications of blood-derived and marrow-derived stem cells for nonmalignant diseases. JAMA 2008; 299(8): 925–936. doi: 10.1001/jama.299.8.925Google Scholar

Ikehara, S. Bone marrow transplantation for autoimmune diseases. Acta Haematol 1998; 99(3): 116–132. doi: 40826Google Scholar

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 U S A 1985; 82(8): 2483–2487.Google Scholar

van Bekkum, DW. Stem cell transplantation for autoimmune disorders. Preclinical experiments. Best Pract Res Clin Haematol 2004; 17(2): 201–222. doi: 10.1016/j.beha.2004.04.003Google Scholar

van Bekkum, DW. Stem cell transplantation in experimental models of autoimmune disease. J Clin Immunol 2000; 20(1): 10–16.Google Scholar

Van Bekkum, DW. Experimental basis for the treatment of autoimmune diseases with autologous hematopoietic stem cell transplantation. Bone Marrow Transplant 2003; 32 (Suppl 1): S37–39. doi: 10.1038/sj.bmt.1703941Google Scholar

van Bekkum, DW. Experimental basis of hematopoietic stem cell transplantation for treatment of autoimmune diseases. J Leukoc Biol 2002; 72(4): 609–620.Google Scholar

van Bekkum, DW. Autologous stem cell transplantation for treatment of autoimmune diseases. Stem Cells 1999; 17(3): 172–178. doi: 10.1002/stem.170172Google Scholar

Nelson, JL, Torrez, R, Louie, FM, Choe, OS, Storb, R, Sullivan, KM. Pre-existing autoimmune disease in patients with long-term survival after allogeneic bone marrow transplantation. J Rheumatol Suppl 1997; 48: 23–29.Google Scholar

Marmont, AM. Stem cell transplantation for autoimmune disorders. Coincidental autoimmune disease in patients transplanted for conventional indications. Best Pract Res Clin Haematol 2004; 17(2): 223–232. doi: 10.1016/j.beha.2004.04.004CrossRef Google Scholar PubMed

Burt, RK, Traynor, AE, Pope, R, Schroeder, J, Cohen, B, Karlin, KH et al. Treatment of autoimmune disease by intense immunosuppressive conditioning and autologous hematopoietic stem cell transplantation. Blood 1998; 92(10): 3505–3514.Google Scholar

Burt, RK, Cohen, BA, Russell, E, Spero, K, Joshi, A, Oyama, Y et al. Hematopoietic stem cell transplantation for progressive multiple sclerosis: failure of a total body irradiation-based conditioning regimen to prevent disease progression in patients with high disability scores. Blood 2003; 102(7): 2373–2378. doi: 10.1182/blood-2003-03-0877CrossRef Google Scholar PubMed

Tyndall, A, Saccardi, R. Haematopoietic stem cell transplantation in the treatment of severe autoimmune disease: results from phase I/II studies, prospective randomized trials and future directions. Clin Exp Immunol 2005; 141(1): 1–9. doi: 10.1111/j.1365–2249.2005.02806.xGoogle Scholar

Alexander, T, Bondanza, A, Muraro, PA, Greco, R, Saccardi, R, Daikeler, T et al. SCT for severe autoimmune diseases: consensus guidelines of the European Society for Blood and Marrow Transplantation for immune monitoring and biobanking. Bone Marrow Transplant 2015; 50(2):173–80. doi: 10.1038/bmt.2014.251Google Scholar

Saccardi, R, Tyndall, A, Coghlan, G, Denton, C, Edan, G, Emdin, M et al. Consensus statement concerning cardiotoxicity occurring during haematopoietic stem cell transplantation in the treatment of autoimmune diseases, with special reference to systemic sclerosis and multiple sclerosis. Bone Marrow Transplant 2004; 34(10): 877–881. doi: 10.1038/sj.bmt.1704656Google Scholar

Illei, GG. Hematopoietic stem cell transplantation in autoimmune diseases: is the glass half full or half empty? Arthritis Rheum 2006; 54(12): 3730–3734. doi: 10.1002/art.22257Google Scholar

Domsic, RT, Medsger, TA. Connective tissue diseases: Predicting death in SSc: planning and cooperation are needed. Nat Rev Rheumatol 2011; 7(11): 628–630. doi: 10.1038/nrrheum.2011.152CrossRef Google Scholar PubMed

Fransen, J, Popa-Diaconu, D, Hesselstrand, R, Carreira, P, Valentini, G, Beretta, L et al. Clinical prediction of 5-year survival in systemic sclerosis: validation of a simple prognostic model in EUSTAR centres. Ann Rheum Dis 2011; 70(10): 1788–1792. doi: 10.1136/ard.2010.144360CrossRef Google Scholar PubMed

Burt, RK, Shah, SJ, Gheorghiade, M, Ruderman, E, Schroeder, J. Hematopoietic stem cell transplantation for systemic sclerosis: if you are confused, remember: “it is a matter of the heart”. J Rheumatol 2012; 39(2): 206–209. doi: 10.3899/jrheum.111302Google Scholar

Burt, RK, Oliveira, MC, Shah, SJ. Cardiac assessment before stem cell transplantation for systemic sclerosis. JAMA 2014; 312(17): 1803. doi: 10.1001/jama.2014.12566Google Scholar

Houssiau, FA, Vasconcelos, C, D’Cruz, D, Sebastiani, GD, Garrido Ed Ede, E, Danieli, MG et al. Immunosuppressive therapy in lupus nephritis: the Euro-Lupus Nephritis Trial, a randomized trial of low-dose versus high-dose intravenous cyclophosphamide. Arthritis Rheum 2002; 46(8): 2121–2131. doi: 10.1002/art.10461Google Scholar

Contreras, G, Pardo, V, Leclercq, B, Lenz, O, Tozman, E, O’Nan, P et al. Sequential therapies for proliferative lupus nephritis. N Engl J Med 2004; 350(10): 971–980. doi: 10.1056/NEJMoa031855Google Scholar

Illei, GG, Austin, HA, Crane, M, Collins, L, Gourley, MF, Yarboro, CH et al. Combination therapy with pulse cyclophosphamide plus pulse methylprednisolone improves long-term renal outcome without adding toxicity in patients with lupus nephritis. Ann Intern Med 2001; 135(4): 248–257.Google Scholar

Doria, A, Iaccarino, L, Ghirardello, A, Zampieri, S, Arienti, S, Sarzi-Puttini, P et al. Long-term prognosis and causes of death in systemic lupus erythematosus. Am J Med 2006; 119(8): 700–706. doi: 10.1016/j.amjmed.2005.11.034Google Scholar

Jayne, D, Tyndall, A. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus 2004; 13(5): 359–365.Google Scholar

Traynor, AE, Barr, WG, Rosa, RM, Rodriguez, J, Oyama, Y, Baker, S et al. Hematopoietic stem cell transplantation for severe and refractory lupus. Analysis after five years and fifteen patients. Arthritis Rheum 2002; 46(11): 2917–2923. doi: 10.1002/art.10594CrossRef Google Scholar PubMed

Moore, J, Brooks, P, Milliken, S, Biggs, J, Ma, D, Handel, M et al. A pilot randomized trial comparing CD34-selected versus unmanipulated hemopoietic stem cell transplantation for severe, refractory rheumatoid arthritis. Arthritis Rheum 2002; 46(9): 2301–2309. doi: 10.1002/art.10495Google Scholar

Snowden, JA, Passweg, J, Moore, JJ, Milliken, S, Cannell, P, Van Laar, J et al. Autologous hemopoietic stem cell transplantation in severe rheumatoid arthritis: a report from the EBMT and ABMTR. J Rheumatol 2004; 31(3): 482–488.Google Scholar

Snowden, JA, Kapoor, S, Wilson, AG. Stem cell transplantation in rheumatoid arthritis. Autoimmunity 2008; 41(8): 625–631. doi: 10.1080/08916930802198550Google Scholar

Verburg, RJ, Sont, JK, van Laar, JM. Reduction of joint damage in severe rheumatoid arthritis by high-dose chemotherapy and autologous stem cell transplantation. Arthritis Rheum 2005; 52(2): 421–424. doi: 10.1002/art.20859Google Scholar

Teng, YK, Verburg, RJ, Sont, JK, van den Hout, WB, Breedveld, FC, van Laar, JM. Long-term followup of health status in patients with severe rheumatoid arthritis after high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Arthritis Rheum 2005; 52(8): 2272–2276. doi: 10.1002/art.21219Google Scholar

Moore, J, Ma, D, Will, R, Cannell, P, Handel, M, Milliken, S. A phase II study of Rituximab in rheumatoid arthritis patients with recurrent disease following haematopoietic stem cell transplantation. Bone Marrow Transplant 2004; 34(3): 241–247. doi: 10.1038/sj.bmt.1704570CrossRef Google Scholar PubMed

Roord, ST, de Jager, W, Boon, L, Wulffraat, N, Martens, A, Prakken, B et al. Autologous bone marrow transplantation in autoimmune arthritis restores immune homeostasis through CD4+CD25+Foxp3+ regulatory T cells. Blood 2008; 111(10): 5233–5241. doi: 10.1182/blood-2007-12-128488Google Scholar

Wulffraat, NM, de Kleer, IM, Prakken, B. Refractory juvenile idiopathic arthritis: using autologous stem cell transplantation as a treatment strategy. Expert Rev Mol Med 2006; 8(26): 1–11. doi: 10.1017/S1462399406000135Google Scholar

Wu, Q, Pesenacker, AM, Stansfield, A, King, D, Barge, D, Foster, HE et al. Immunological characteristics and T-cell receptor clonal diversity in children with systemic juvenile idiopathic arthritis undergoing T-cell-depleted autologous stem cell transplantation. Immunology 2014; 142(2): 227–236. doi: 10.1111/imm.12245Google Scholar

Mancardi, GL, Sormani, MP, Di Gioia, M, Vuolo, L, Gualandi, F, Amato, MP et al. Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: the Italian multi-centre experience. Mult Scler 2012; 18(6): 835–842. doi: 10.1177/1352458511429320Google Scholar

Bowen, JD, Kraft, GH, Wundes, A, Guan, Q, Maravilla, KR, Gooley, TA et al. Autologous hematopoietic cell transplantation following high-dose immunosuppressive therapy for advanced multiple sclerosis: long-term results. Bone Marrow Transplant 2012; 47(7): 946–951. doi: 10.1038/bmt.2011.208Google Scholar

Saccardi, R, Freedman, MS, Sormani, MP, Atkins, H, Farge, D, Griffith, LM et al. A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Mult Scler 2012; 18(6): 825–834. doi: 10.1177/1352458512438454Google Scholar

Phillips, JT, Giovannoni, G, Lublin, FD, O’Connor, PW, Polman, CH, Willoughby, E et al. Sustained improvement in Expanded Disability Status Scale as a new efficacy measure of neurological change in multiple sclerosis: treatment effects with natalizumab in patients with relapsing multiple sclerosis. Mult Scler 2011; 17(8): 970–979. doi: 10.1177/1352458511399611Google Scholar

Greco, R, Bondanza, A, Oliveira, MC, Badoglio, M, Burman, J, Piehl, F et al. Autologous hematopoietic stem cell transplantation in neuromyelitis optica: a registry study of the EBMT Autoimmune Diseases Working Party. Mult Scler 2015; 21(2): 189–197. doi: 10.1177/1352458514541978Google Scholar

Matiello, M, Pittock, SJ, Porrata, L, Weinshenker, BG. Failure of autologous hematopoietic stem cell transplantation to prevent relapse of neuromyelitis optica. Arch Neurol 2011; 68(7): 953–955. doi: 10.1001/archneurol.2011.38Google Scholar

Peng, F, Qiu, W, Li, J, Hu, X, Huang, R, Lin, D et al. A preliminary result of treatment of neuromyelitis optica with autologous peripheral hematopoietic stem cell transplantation. Neurologist 2010; 16(6): 375–378. doi: 10.1097/NRL.0b013e3181b126e3Google Scholar

Bewtra, M, Kaiser, LM, TenHave, T, Lewis, JD. Crohn’s disease and ulcerative colitis are associated with elevated standardized mortality ratios: a meta-analysis. Inflamm Bowel Dis 2013; 19(3): 599–613. doi: 10.1097/MIB.0b013e31827f27aeGoogle Scholar

Gu, Y, Gong, C, Peng, X, Wei, L, Su, C, Qin, M et al. Autologous hematopoietic stem cell transplantation and conventional insulin therapy in the treatment of children with newly diagnosed type 1 diabetes: long term follow-up. Chin Med J (Engl) 2014; 127(14): 2618–2622.Google Scholar PubMed

Gu, W, Hu, J, Wang, W, Li, L, Tang, W, Sun, S et al. Diabetic ketoacidosis at diagnosis influences complete remission after treatment with hematopoietic stem cell transplantation in adolescents with type 1 diabetes. Diabetes Care 2012; 35(7): 1413–1419. doi: 10.2337/dc11-2161Google Scholar

Snarski, E, Milczarczyk, A, Torosian, T, Paluszewska, M, Urbanowska, E, Król, M et al. Independence of exogenous insulin following immunoablation and stem cell reconstitution in newly diagnosed diabetes type I. Bone Marrow Transplant 2011; 46(4): 562–566. doi: 10.1038/bmt.2010.147Google Scholar

Notkins, AL, Lernmark, A. Autoimmune type 1 diabetes: resolved and unresolved issues. J Clin Invest 2001; 108(9): 1247–1252. doi: 10.1172/JCI14257Google Scholar

Burt, RK, Fassas, A, Snowden, J, van Laar, JM, Kozak, T, Wulffraat, NM et al. Collection of hematopoietic stem cells from patients with autoimmune diseases. Bone Marrow Transplant 2001; 28(1): 1–12. doi: 10.1038/sj.bmt.1703081Google Scholar

De Santis, GC, de Pina Almeida Prado, B, de Lima Prata, K, Brunetta, DM, Orellana, MD, Palma, PV et al. Mobilization and harvesting of PBPC in newly diagnosed type 1 diabetes mellitus. Bone Marrow Transplant 2012; 47(7): 993–994. doi: 10.1038/bmt.2011.188Google Scholar

Dubinsky, AN, Burt, RK, Martin, R, Muraro, PA. T-cell clones persisting in the circulation after autologous hematopoietic SCT are undetectable in the peripheral CD34+ selected graft. Bone Marrow Transplant 2010; 45(2): 325–331. doi: 10.1038/bmt.2009.139Google Scholar

Nash, RA, Dansey, R, Storek, J, Georges, GE, Bowen, JD, Holmberg, LA et al. Epstein–Barr virus-associated posttransplantation lymphoproliferative disorder after high-dose immunosuppressive therapy and autologous CD34-selected hematopoietic stem cell transplantation for severe autoimmune diseases. Biol Blood Marrow Transplant 2003; 9(9): 583–591.Google Scholar

Tsukamoto, H, Ayano, M, Miyamoto, T, Niiro, H, Arinobu, Y, Akahoshi, M et al. Comparison of CD34-selected and unmanipulated autologous hematopoietic stem cell transplantation for systemic sclerosis: four-year follow-up results. EULAR Conference 2014; 73 (Suppl 2): 96–97.Google Scholar

Oliveira, M, Labopin, M, Henes, J, Moore, J, Del Papa, N, Stanciu, S et al. Does ex vivo CD34+ cell selection change the outcome of systemic sclerosis patients treated with autologous hematopoietic stem cell transplantation (AHSCT), an ADWP EBMT Study? ASH Conference 2014; 124(21).Google Scholar

Nash, RA, McSweeney, PA, Crofford, LJ, Abidi, M, Chen, CS, Godwin, JD 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(4): 1388–1396. doi: 10.1182/blood-2007-02-072389Google Scholar

Burt, RK, Marmont, A, Oyama, Y, Slavin, S, Arnold, R, Hiepe, F 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(12): 3750–3760. doi: 10.1002/art.22256Google Scholar

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(5): 1602–1610.Google Scholar

Openshaw, H, Lund, BT, Kashyap, A, Atkinson, R, Sniecinski, I, Weiner, LP et al. Peripheral blood stem cell transplantation in multiple sclerosis with busulfan and cyclophosphamide conditioning: report of toxicity and immunological monitoring. Biol Blood Marrow Transplant 2000; 6(5A): 563–575.Google Scholar

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(5): R124. doi: 10.1186/ar2534Google Scholar

Henes, JC, Koetter, I, Horger, M, Schmalzing, M, Mueller, K, Eick, C et al. Autologous stem cell transplantation with thiotepa-based conditioning in patients with systemic sclerosis and cardiac manifestations. Rheumatology (Oxford) 2014; 53(5): 919–922. doi: 10.1093/rheumatology/ket464Google Scholar

Chen, B, Zhou, M, Ouyang, J, Zhou, R, Xu, J, Zhang, Q et al. Long-term efficacy of autologous haematopoietic stem cell transplantation in multiple sclerosis at a single institution in China. Neurol Sci 2012; 33(4): 881–886. doi: 10.1007/s10072-011–0859-yGoogle Scholar

Curro’, D, Vuolo, L, Gualandi, F, Bacigalupo, A, Roccatagliata, L, Capello, E et al. Low intensity lympho-ablative regimen followed by autologous hematopoietic stem cell transplantation in severe forms of multiple sclerosis: A MRI-based clinical study. Mult Scler 2015; 21(11):1423–1430. doi: 10.1177/1352458514564484Google Scholar

Greco, R, Bondanza, A, Vago, L, Moiola, L, Rossi, P, Furlan, R et al. Allogeneic hematopoietic stem cell transplantation for neuromyelitis optica. Ann Neurol 2014; 75(3): 447–453. doi: 10.1002/ana.24079Google Scholar

Aouad, P, Li, J, Arthur, C, Burt, R, Fernando, S, Parratt, J. Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant. J Clin Neurosci 2015; 22(7): 1215–1217. doi: 10.1016/j.jocn.2015.02.007Google Scholar

Clerici, M, Cassinotti, A, Onida, F, Trabattoni, D, Annaloro, C, Della Volpe, A et al. Immunomodulatory effects of unselected haematopoietic stem cells autotransplantation in refractory Crohn’s disease. Dig Liver Dis 2011; 43(12): 946–952. doi: 10.1016/j.dld.2011.07.021Google Scholar

Kriván, G, Szabó, D, Kállay, K, Benyó, G, Kassa, C, Sinkó, J et al. [Successful autologous haematopoietic stem cell transplantation in severe, therapy-resistant childhood Crohn’s disease. Report on the first case in Hungary]. Orv Hetil 2014; 155(20): 789–792. doi: 10.1556/OH.2014.29892Google Scholar

Kountouras, J, Sakellari, I, Tsarouchas, G, Tsiaousi, E, Michael, S, Zavos, C et al. Autologous haematopoietic stem cell transplantation in a patient with refractory Crohn’s disease. J Crohns Colitis 2011; 5(3): 275–276. doi: 10.1016/j.crohns.2011.03.004Google Scholar

Hasselblatt, P, Drognitz, K, Potthoff, K, Bertz, H, Kruis, W, Schmidt, C et al. Remission of refractory Crohn’s disease by high-dose cyclophosphamide and autologous peripheral blood stem cell transplantation. Aliment Pharmacol Ther 2012; 36(8): 725–735. doi: 10.1111/apt.12032Google Scholar

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(12): 2660–2665. doi: 10.1002/1529-0131(199912)42:12<2660::AID-ANR23>3.0.CO;2-NGoogle Scholar

Hussein, H, Lee, P, Chau, C, Johnson, SR. The effect of male sex on survival in systemic sclerosis. J Rheumatol 2014; 41(11): 2193–2200. doi: 10.3899/jrheum.140006Google Scholar

Sampaio-Barros, PD, Bortoluzzo, AB, Marangoni, RG, Rocha, LF, Del Rio, AP, Samara, AM et al. Survival, causes of death, and prognostic factors in systemic sclerosis: analysis of 947 Brazilian patients. J Rheumatol 2012; 39(10): 1971–1978. doi: 10.3899/jrheum.111582Google Scholar

Rahman, P, Gladman, DD, Urowitz, MB, Hallett, D, Tam, LS. Early damage as measured by the SLICC/ACR damage index is a predictor of mortality in systemic lupus erythematosus. Lupus 2001; 10(2): 93–96.Google Scholar

Kaufman, DW, Reshef, S, Golub, HL, Peucker, M, Corwin, MJ, Goodin, DS et al. Survival in commercially insured multiple sclerosis patients and comparator subjects in the U.S. Mult Scler Relat Disord 2014; 3(3): 364–371. doi: 10.1016/j.msard.2013.12.003CrossRef Google Scholar PubMed

Mancardi, G, Saccardi, R. Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol 2008; 7(7): 626–636. doi: 10.1016/S1474-4422(08)70138–8Google Scholar

Kohno, K, Nagafuji, K, Tsukamoto, H, Horiuchi, T, Takase, K, Aoki, K et al. Infectious complications in patients receiving autologous CD34-selected hematopoietic stem cell transplantation for severe autoimmune diseases. Transpl Infect Dis 2009; 11(4): 318–323. doi: 10.1111/j.1399–3062.2009.00401.xGoogle Scholar

Bloomgren, G, Richman, S, Hotermans, C, Subramanyam, M, Goelz, S, Natarajan, A et al. Risk of natalizumab-associated progressive multifocal leukoencephalopathy. N Engl J Med 2012; 366(20): 1870–1880. doi: 10.1056/NEJMoa1107829Google Scholar

Pfitzer, C, Orawa, H, Balcerek, M, Langer, T, Dirksen, U, Keslova, P et al. Dynamics of fertility impairment and recovery after allogeneic haematopoietic stem cell transplantation in childhood and adolescence: results from a longitudinal study. J Cancer Res Clin Oncol 2015; 141(1): 135–142. doi: 10.1007/s00432-014–1781-5Google Scholar

Nabhan, SK, Bitencourt, MA, Duval, M, Abecasis, M, Dufour, C, Boudjedir, K et al. Fertility recovery and pregnancy after allogeneic hematopoietic stem cell transplantation in Fanconi anemia patients. Haematologica 2010; 95(10): 1783–1787. doi: 10.3324/haematol.2010.023929Google Scholar

Snarski, E, Snowden, JA, Oliveira, MC, Simoes, B, Badoglio, M, Carlson, K et al. Onset and outcome of pregnancy after autologous haematopoietic SCT (AHSCT) for autoimmune diseases: a retrospective study of the EBMT autoimmune diseases working party (ADWP). Bone Marrow Transplant 2015; 50(2): 216–220. doi: 10.1038/bmt.2014.248Google Scholar

Leal, AM, Oliveira, MC, Couri, CE, Moraes, DA, Stracieri, AB, Pieroni, F et al. Testicular function in patients with type 1 diabetes treated with high-dose CY and autologous hematopoietic SCT. Bone Marrow Transplant 2012; 47(3): 467–468. doi: 10.1038/bmt.2011.113Google Scholar

Daikeler, T, Tichelli, A, Passweg, J. Complications of autologous hematopoietic stem cell transplantation for patients with autoimmune diseases. Pediatr Res 2012; 71(4 Pt 2): 439–444. doi: 10.1038/pr.2011.57Google Scholar

Bohgaki, T, Atsumi, T, Koike, T. Autoimmune disease after autologous hematopoietic stem cell transplantation. Autoimmun Rev 2008; 7(3): 198–203. doi: 10.1016/j.autrev.2007.11.005Google Scholar

Holbro, A, Abinun, M, Daikeler, T. Management of autoimmune diseases after haematopoietic stem cell transplantation. Br J Haematol 2012; 157(3): 281–290. doi: 10.1111/j.1365–2141.2012.09070.xGoogle Scholar

Daikeler, T, Labopin, M, Di Gioia, M, Abinun, M, Alexander, T, Miniati, I 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(6): 1693–1698. doi: 10.1182/blood-2011-02-336156Google Scholar

Loh, Y, Oyama, Y, Statkute, L, Quigley, K, Yaung, K, Gonda, E et al. Development of a secondary autoimmune disorder after hematopoietic stem cell transplantation for autoimmune diseases: role of conditioning regimen used. Blood 2007; 109(6): 2643–2548. doi: 10.1182/blood-2006-07-035766Google Scholar

Muraro, PA, Douek, DC. Renewing the T cell repertoire to arrest autoimmune aggression. Trends Immunol 2006; 27(2): 61–67. doi: 10.1016/j.it.2005.12.003Google Scholar

Abrahamsson, S, Muraro, PA. Immune re-education following autologous hematopoietic stem cell transplantation. Autoimmunity 2008; 41(8): 577–584. doi: 10.1080/08916930802197081Google Scholar

Hügle, T, Daikeler, T. Stem cell transplantation for autoimmune diseases. Haematologica 2010; 95(2): 185–188. doi: 10.3324/haematol.2009.017038Google Scholar

Muraro, PA, Robins, H, Malhotra, S, Howell, M, Phippard, D, Desmarais, C et al. T cell repertoire following autologous stem cell transplantation for multiple sclerosis. J Clin Invest 2014; 124(3): 1168–1172. doi: 10.1172/JCI71691Google Scholar

Muraro, PA, Douek, DC, Packer, A, Chung, K, Guenaga, FJ, Cassiani-Ingoni, R 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(5): 805–816. doi: 10.1084/jem.20041679Google Scholar

Baraut, J, Grigore, EI, Jean-Louis, F, Khelifa, SH, Durand, C, Verrecchia, F et al. Peripheral blood regulatory T cells in patients with diffuse systemic sclerosis (SSc) before and after autologous hematopoietic SCT: a pilot study. Bone Marrow Transplant 2014; 49(3): 349–354. doi: 10.1038/bmt.2013.202Google Scholar

Farge, D, Henegar, C, Carmagnat, M, Daneshpouy, M, Marjanovic, Z, Rabian, C et al. Analysis of immune reconstitution after autologous bone marrow transplantation in systemic sclerosis. Arthritis Rheum 2005; 52(5): 1555–1563. doi: 10.1002/art.21036Google Scholar

Alexander, T, Thiel, A, Rosen, O, Massenkeil, G, Sattler, A, Kohler, S 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(1): 214–223. doi: 10.1182/blood-2008-07-168286Google Scholar

Li, HW, Sykes, M. Emerging concepts in haematopoietic cell transplantation. Nat Rev Immunol 2012; 12(6): 403–416. doi: 10.1038/nri3226CrossRef Google Scholar PubMed

Sykes, M, Nikolic, B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005; 435(7042): 620–627. doi: 10.1038/nature03728Google Scholar

Zhang, L, Bertucci, AM, Ramsey-Goldman, R, Burt, RK, Datta, SK. Regulatory T cell (Treg) subsets 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(10): 6346–6358. doi: 10.4049/jimmunol.0901773Google Scholar

Abrahamsson, SV, Angelini, DF, Dubinsky, AN, Morel, E, Oh, U, Jones, JL et al. Non-myeloablative autologous haematopoietic stem cell transplantation expands regulatory cells and depletes IL-17 producing mucosal-associated invariant T cells in multiple sclerosis. Brain 2013; 136(Pt 9): 2888–2903. doi: 10.1093/brain/awt182Google Scholar

Darlington, PJ, Touil, T, Doucet, JS, Gaucher, D, Zeidan, J, Gauchat, D et al. Diminished Th17 (not Th1) responses underlie multiple sclerosis disease abrogation after hematopoietic stem cell transplantation. Ann Neurol 2013; 73(3): 341–354. doi: 10.1002/ana.23784Google Scholar

Tsukamoto, H, Nagafuji, K, Horiuchi, T, Mitoma, H, Niiro, H, Arinobu, Y 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(5): 944–952. doi: 10.1093/rheumatology/keq414Google Scholar

Li, L, Shen, S, Ouyang, J, Hu, Y, Hu, L, Cui, W et al. Autologous hematopoietic stem cell transplantation modulates immunocompetent cells and improves β-cell function in Chinese patients with new onset of type 1 diabetes. J Clin Endocrinol Metab 2012; 97(5): 1729–1736. doi: 10.1210/jc.2011–2188Google Scholar

Bohgaki, T, Atsumi, T, Bohgaki, M, Furusaki, A, Kondo, M, Sato-Matsumura, KC 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(6): 1240–1248. doi: 10.3899/jrheum.081025Google Scholar

de Oliveira, GL, Malmegrim, KC, Ferreira, AF, Tognon, R, Kashima, S, Couri, CE et al. Up-regulation of fas and fasL pro-apoptotic genes expression in type 1 diabetes patients after autologous haematopoietic stem cell transplantation. Clin Exp Immunol 2012; 168(3): 291–302. doi: 10.1111/j.1365–2249.2012.04583.xGoogle Scholar

Arruda, LC, Lorenzi, JC, Sousa, AP, Zanette, DL, Palma, PV, Panepucci, RA et al. Autologous hematopoietic SCT normalizes miR-16, -155 and -142–3p expression in multiple sclerosis patients. Bone Marrow Transplant 2014;50:380–389Google Scholar

Connick, P, Kolappan, M, Crawley, C, Webber, DJ, Patani, R, Michell, AW et al. Autologous mesenchymal stem cells for the treatment of secondary progressive multiple sclerosis: an open-label phase 2a proof-of-concept study. Lancet Neurol 2012; 11(2): 150–156. doi: 10.1016/S1474-4422(11)70305-2Google Scholar

Gu, F, Wang, D, Zhang, H, Feng, X, Gilkeson, GS, Shi, S et al. Allogeneic mesenchymal stem cell transplantation for lupus nephritis patients refractory to conventional therapy. Clin Rheumatol 2014; 33(11): 1611–1619. doi: 10.1007/s10067-014–2754-4Google Scholar

Carlsson, PO, Schwarcz, E, Korsgren, O, Le Blanc, K. Preserved β-cell function in type 1 diabetes by mesenchymal stromal cells. Diabetes 2015; 64(2): 587–592. doi: 10.2337/db14-0656Google Scholar

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Chapter 57 - Autologous Hematopoietic Cell Transplants for Autoimmune Diseases: Specific Diseases and Controversies

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