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Review
. 2016 Oct;68(10):2361-71.
doi: 10.1002/art.39748.

Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement

Keith M Sullivan  1 , Ankoor Shah  2 , Stefanie Sarantopoulos  2 , Daniel E Furst  3
Affiliations
Review

Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement

Keith M Sullivan et al. Arthritis Rheumatol. 2016 Oct.
No abstract available

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Figures

Figure 1
Figure 1
Survival of patients with limited cutaneous systemic sclerosis (lcSSc) or diffuse cutaneous SSc (dcSSc) with internal organ complications enrolled at the Royal Free Hospital (London, UK). Adapted, with permission, from ref. 7.
Figure 2
Figure 2
Results after total body irradiation, cyclophosphamide and antithymocyte globulin preparative conditioning, and CD34+ selected autologous hematopoietic stem cell transplantation in 34 patients with severe scleroderma 39. A, Modified Rodnan skin thickness score. B, Forced vital capacity. C, Diffusing capacity for carbon monoxide (DLCO). D, Quality of life measures (modified Health Assessment Questionnaire). Solid black lines show the mean; broken lines represent the generalized estimating equation; gray lines represent individual patient values.
Figure 3
Figure 3
Results from the Autologous Stem Cell Transplantation International Scleroderma trial comparing CD34+ selected autologous hematopoietic stem cell transplantation (HSCT) after conditioning with cyclophosphamide (CYC) and antithymocyte globulin to 12 months of CYC treatment (control) 32. A, Survival free of organ failure. B, Overall survival. HR = hazard ratio.
Figure 4
Figure 4
Immune homeostasis following autologous hematopoietic stem cell transplantation (HSCT). A and B, T cell receptor (TCR) diversity in Treg cells restored after autologous HSCT in a patient with autoimmune disease (A) and in 4 healthy controls (HC) (B) 46. The number of different TCR sequences per sample and diversity (Di), where 0 = none and 1 = maximum, before and after transplantation are shown. Adapted, with permission, from ref. 46. C and D, Model of how autologous HSCT results in immune tolerance and immune homeostasis. C, Outcompetition of nonautoreactive lymphocytes results in the death of autoreactive clones. Interleukin‐2 (IL‐2) and serum BAFF (sBAFF) levels increase after lymphomyeloablation 1. The proportions of regulatory T and B cells, both known to suppress or kill effector T and B cells, are increased 2. D, Supranormal numbers of B cells and activated regulatory cells eliminate autoreactive clones during immune recovery. The surge in cell numbers abates as immune homeostasis is achieved.
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References

    1. Gabrielli A, Avvedimento E, Krieg T. Mechanisms of disease: scleroderma. N Engl J Med 2009;360:1989–2003. - PubMed
    1. Whitfield ML, Finlay DR, Murray JI, Troyanskaya OG, Chi JT, Pergamenschikov A, et al. Systemic and cell type‐specific gene expression patterns in scleroderma skin. Proc Natl Acad Sci U S A 2003;100:12319–24. - PMC - PubMed
    1. Assassi S, Swindell WR, Wu M, Tan FD, Khanna D, Furst DE, et al. Dissecting the heterogeneity of skin gene expression patterns in systemic sclerosis. Arthritis Rheumatol 2015;67:3016–26. - PMC - PubMed
    1. Van Bon L, Affandi AJ, Broen J, Christmann RB, Marijnissen RJ, Stawski L, et al. Proteome‐wide analysis and CXCL4 as a biomarker in systemic sclerosis. N Engl J Med 2014;370:433–43. - PMC - PubMed
    1. Domsic RT, Rodriguez‐Reyna T, Lucas M, Fertig N, Medsger TA Jr. Skin thickness progression rate: a predictor of mortality and early internal organ involvement in diffuse scleroderma. Ann Rheum Dis 2011;70:104–9. - PMC - PubMed

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