Abstract
BackgroundSystemic sclerosis (SSc) is an autoimmune disease involving the skin and several internal organs. Most therapies available for this disease are symptomatic. Given the difficulty in treating SSc, we conducted this study to investigate the effect of combined plasmapheresis (PE) and allogeneic mesenchymal stem cells transplantation (MSCT) therapy on SSc.MethodsFourteen patients underwent three repeated PE treatments with subsequent pulse cyclophosphamide on days 1, 3 and 5. Patients received a single MSCT (1 × 106 cells/kg of body weight) on day 8. During follow up, evaluations performed included complete physical examination, serologic testing, and organ function.ResultsThe mean modified Rodnan skin score (MRSS) improved from 20.1 ± 3.1 to 13.8 ± 10.2 (P < 0.001) at 12 months of follow up. Three patients had interstitial lung disease, all had improvement of lung function and improved computed tomography (CT) images after 12 months of combined therapy. This combined treatment also significantly decreased the anti-Scl70 autoantibody titer and serum transforming growth factor-β and vascular endothelial growth factor levels during follow up.ConclusionThe results indicate that PE combined with MSCT is a feasible treatment associated with possible clinical benefit for SSc patients.Trial registrationClinicalTrials.gov, NCT00962923. Registered on 19 August 2009.
Highlights
Systemic sclerosis (SSc) is an autoimmune disease involving the skin and several internal organs
Many inflammatory cytokines and growth factors are associated with the onset and progression of fibrosis, such as transforming growth factor (TGF)-β, endothelin1, interleukin (IL)-17, IL-23 and tumor necrosis factor (TNF)-α [3,4,5,6]
Demographic findings Fourteen patients with SSc underwent allogeneic mesenchymal stem cells transplantation (MSCT); all of those were classified as having the diffuse cutaneous subsets of the disease
Summary
Systemic sclerosis (SSc) is an autoimmune disease involving the skin and several internal organs. Given the difficulty in treating SSc, we conducted this study to investigate the effect of combined plasmapheresis (PE) and allogeneic mesenchymal stem cells transplantation (MSCT) therapy on SSc. Systemic sclerosis (SSc) is a rare chronic autoimmune disease characterized by increased synthesis and deposition of extra-cellular matrix in skin and various internal organs. Depending on the extent of skin fibrosis, SSc can be classified as two main subtypes, the limited cutaneous form (lcSSc) and the diffuse cutaneous form (dcSSc). The extensive degree of skin involvement coincides with future severe internal organ manifestations, poor prognosis and mortality, at least in the early phase of dcSSc [2]. Skin disease in SSc patients is treated with immunosuppressive agents such as methotrexate (MTX) or mycophenolate mofetil (MMF) [7, 8], but are not acceptable to all Chinese patients in terms of
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