Myeloablative autologous stem-cell transplantation for severe scleroderma

New England Journal of Medicine

Volumn 378, Issue 1, 2018, Pages 35-47

  Sullivan, K M ^a   Goldmuntz, E A ^d   Keyes Elstein, L ^c   McSweeney, P A ^e   Pinckney, A ^c   Welch, B ^d   Mayes, M D ^f   Nash, R A ^e   Crofford, L J ^h   Eggleston, B ^c   Castina, S ^c   Griffith, L M ^d   Goldstein, J S ^d   Wallace, D ^b   Craciunescu, O ^a   Khanna, D ⁱ   Folz, R J ^j   Goldin, J ^p   Clair, E W ^a   Seibold, J R ⁱ   Phillips, K ^h   Mineishi, S ^k   Simms, R W ^l   Ballen, K ^m   Wener, M H ⁿ   Georges, G E ^o   Heimfeld, S ^o   Hosing, C ^g   Forman, S ^q   Kafaja, S ^p   Silver, R M ^r   Griffing, L ^s   Storek, J ^t   LeClercq, S ^t   Brasington, R ^u   Csuka, M E ^v   Bredeson, C ^w   Keever Taylor, C ^v   Domsic, R T ^x   Kahaleh, M B ^y   Medsger, T ^x   Furst, D E ^n,p   more..

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b RTI INTERNATIONAL   (United States)

^c Rho Federal Systems Division Inc   (United States)

^d NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^e Rocky Mountain Blood and Marrow Transplant Program   (United States)

^f UNIVERSITY OF TEXAS   (United States)

^g UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^h VANDERBILT UNIVERSITY   (United States)

ⁱ UNIVERSITY OF MICHIGAN   (United States)

^j CASE WESTERN RESERVE UNIVERSITY   (United States)

^k UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^l BOSTON UNIVERSITY   (United States)

^m UNIVERSITY OF VIRGINIA   (United States)

ⁿ UNIVERSITY OF WASHINGTON   (United States)

^o USA   (United States)

^p UNIVERSITY OF CALIFORNIA   (United States)

^q CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^r MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^s MAYO CLINIC   (United States)

^t UNIVERSITY OF CALGARY   (Canada)

^u WASHINGTON UNIVERSITY   (United States)

^v MEDICAL COLLEGE OF WISCONSIN   (United States)

^w OTTAWA HOSPITAL RESEARCH INSTITUTE   (Canada)

^x UNIVERSITY OF PITTSBURGH   (United States)

^y UNIVERSITY OF TOLEDO MEDICAL CENTER   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOPHOSPHAMIDE; DISEASE MODIFYING ANTIRHEUMATIC DRUG; GRANULOCYTE COLONY STIMULATING FACTOR; IMMUNOSUPPRESSIVE AGENT;

ADULT; AGED; ARTICLE; AUTOLOGOUS STEM CELL TRANSPLANTATION; CONTROLLED STUDY; DISEASE SEVERITY; EVENT FREE SURVIVAL; FEMALE; FORCED VITAL CAPACITY; HEALTH ASSESSMENT QUESTIONNAIRE; HEART FAILURE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INTENTION TO TREAT ANALYSIS; KAPLAN MEIER METHOD; LONG TERM CARE; LUNG DIFFUSION CAPACITY; LUNG FUNCTION TEST; MAJOR CLINICAL STUDY; MALE; MORTALITY; NONMYELOABLATIVE STEM CELL TRANSPLANTATION; OVERALL SURVIVAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCLERODERMA; SCORING SYSTEM; WILCOXON SIGNED RANKS TEST; X-RAY COMPUTED TOMOGRAPHY; ADOLESCENT; AUTOTRANSPLANTATION; CLINICAL TRIAL; COMPARATIVE STUDY; DISEASE FREE SURVIVAL; FOLLOW UP; INFECTION; MIDDLE AGED; MULTICENTER STUDY; SYSTEMIC SCLEROSIS; TRANSPLANTATION CONDITIONING; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; CYCLOPHOSPHAMIDE; DISEASE-FREE SURVIVAL; FEMALE; FOLLOW-UP STUDIES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFECTION; INTENTION TO TREAT ANALYSIS; KAPLAN-MEIER ESTIMATE; MALE; MIDDLE AGED; SCLERODERMA, SYSTEMIC; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, AUTOLOGOUS; YOUNG ADULT;

EID: 85040338916     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1703327     Document Type: Article

References (40)

1. Mayes MD, Lacey JV Jr, Beebe-Dimmer J, et al. Prevalence, incidence, survival, and disease characteristics of systemic sclerosis in a large US population. Arthritis Rheum 2003; 48: 2246-55.
2. Elhai M, Meune C, Avouac J, Kahan A, Allanore Y. Trends in mortality in patients with systemic sclerosis over 40 years: a systematic review and meta-analysis of cohort studies. Rheumatology (Oxford) 2012; 51: 1017-26.
3. Nihtyanova SI, Schreiber BE, Ong VH, et al. Prediction of pulmonary complications and long-term survival in systemic sclerosis. Arthritis Rheumatol 2014; 66: 1625-35.
4. 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: 1026-34.
5. Khanna D, Denton CP, Jahreis A, et al. Safety and efficacy of subcutaneous tocilizumab in adults with systemic scle rosis (faSScinate): a phase 2, randomised, controlled trial. Lancet 2016; 387: 2630-40.
6. Tashkin DP, Roth MD, Clements PJ, et al. Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial. Lancet Respir Med 2016; 4: 708-19.
7. Tyndall A, Black C, Finke J, et al. Treatment of systemic sclerosis with autologous haemopoietic stem cell transplantation. Lancet 1997; 349: 254.
8. 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: 577-84.
9. 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: 1388-96.
10. 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: Suppl: S48-S56.
11. Burt RK, Shah SJ, Dill K, et al. Autologous non-myeloablative haemopoietic stemcell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet 2011; 378: 498-506.
12. van Laar JM, Farge D, Sont JK, et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 2014; 311: 2490-8.
13. Sullivan KM, Shah A, Sarantopoulos S, Furst DE. Hematopoietic stem cell transplantation for scleroderma: effective immunomodulatory therapy for patients with pulmonary involvement. Arthritis Rheumatol 2016; 68: 2361-71.
14. Berger VW, Ivanova A, Knoll MD. Minimizing predictability while retaining balance through the use of less restrictive randomization procedures. Stat Med 2003; 22: 3017-28.
15. Keever-Taylor CA, Heimfeld S, Steinmiller KC, et al. Manufacture of autologous CD34+ selected grafts in the NIAIDsponsored HALT-MS and SCOT multicenter clinical trials for autoimmune diseases. Biol Blood Marrow Transplant 2017; 23: 1463-72.
16. McSweeney PA, Nash RA, Sullivan KM, et al. High-dose immunosuppressive therapy for severe systemic sclerosis: initial outcomes. Blood 2002; 100: 1602-10.
17. Craciunescu OI, Steffey BA, Kelsey CR, et al. Renal shielding and dosimetry for patients with severe systemic sclerosis receiving immunoablation with total body irradiation in the Scleroderma: Cyclophosphamide or Transplantation trial. Int J Radiat Oncol Biol Phys 2011; 79: 1248-55.
18. Hosing C, Nash R, McSweeney P, et al. Acute kidney injury in patients with systemic sclerosis participating in hematopoietic cell transplantation trials in the United States. Biol Blood Marrow Transplant 2011; 17: 674-81.
19. Hung EW, Mayes MD, Sharif R, et al. Gastric antral vascular ectasia and its clinical correlates in patients with early diffuse systemic sclerosis in the SCOT trial. J Rheumatol 2013; 40: 455-60.
20. Sullivan KM, McSweeney PA, Nash RA. Cyclophosphamide in scleroderma lung disease. N Engl J Med 2006; 355: 1173-4.
21. Clements PJ, Lachenbruch PA, Seibold JR, et al. Skin thickness score in systemic sclerosis: an assessment of interobserver variability in 3 independent studies. J Rheumatol 1993; 20: 1892-6.
22. Khanna D, Furst DE, Hays RD, et al. Minimally important difference in diffuse systemic sclerosis: results from the D-penicillamine study. Ann Rheum Dis 2006; 65: 1325-9.
23. Finkelstein DM, Schoenfeld DA. Combining mortality and longitudinal measures in clinical trials. Stat Med 1999; 18: 1341-54.
24. Stokes ME, Davis CS, Koch GG. Categorical data analysis using the SAS system. 2nd ed. Cary, NC: SAS Institute, 2009.
25. Bartunek J, Terzic A, Davison BA, et al. Cardiopoietic cell therapy for advanced ischaemic heart failure: results at 39 weeks of the prospective, randomized, double blind, sham-controlled CHART-1 clinical trial. Eur Heart J 2017; 38: 648-60.
26. Cudkowicz ME, van den Berg LH, Shefner JM, et al. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol 2013; 12: 1059-67.
27. Packer M. Development and evolution of a hierarchical clinical composite end point for the evaluation of drugs and devices for acute and chronic heart failure: a 20-year perspective. Circulation 2016; 134: 1664-78.
28. Berry JD, Miller R, Moore DH, et al. The Combined Assessment of Function and Survival (CAFS): a new endpoint for ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener 2013; 14: 162-8.
29. Wittkop L, Smith C, Fox Z, et al. Methodological issues in the use of composite endpoints in clinical trials: examples from the HIV field. Clin Trials 2010; 7: 19-35.
30. Daikeler T, Tichelli A, Passweg J. Complications of autologous hematopoietic stem cell transplantation for patients with autoimmune diseases. Pediatr Res 2012; 71: 439-44.
31. Farge D, Labopin M, Tyndall A, 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: 284-92.
32. Storek J, Zhao Z, Lin E, et al. Recovery from and consequences of severe iatrogenic lymphopenia (induced to treat autoimmune diseases). Clin Immunol 2004; 113: 285-98.
33. Onishi A, Sugiyama D, Kumagai S, Morinobu A. Cancer incidence in systemic sclerosis: meta-analysis of populationbased cohort studies. Arthritis Rheum 2013; 65: 1913-21.
34. Vaxman I, Ram R, Gafter-Gvili A, et al. Secondary malignancies following high dose therapy and autologous hematopoietic cell transplantation - systematic review and meta-analysis. Bone Marrow Transplant 2015; 50: 706-14.
35. Burt RK, Oliveira MC, Shah SJ, 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: 1116-24.
36. van Bekkum DW. Conditioning regimens for the treatment of experimental arthritis with autologous bone marrow transplantation. Bone Marrow Transplant 2000; 25: 357-64.
37. Muraro PA, Robins H, Malhotra S, et al. T cell repertoire following autologous stem cell transplantation for multiple sclerosis. J Clin Invest 2014; 124: 1168-72.
38. de Kleer I, Vastert B, Klein M, et al. Autologous stem cell transplantation for autoimmunity induces immunologic selftolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 2006; 107: 1696-702.
39. Delemarre EM, van den Broek T, Mijnheer G, et al. Autologous stem cell transplantation aids autoimmune patients by functional renewal and TCR diversification of regulatory T cells. Blood 2016; 127: 91-101.
40. Snowden JA. Rebooting autoimmunity with autologous HSCT. Blood 2016; 127: 8-10.