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

Evolution of serum cytokine profile after hematopoietic stem cell transplantation in systemic sclerosis patients

“…7,8,12 The decrease in IL-2 and IL-8 levels, slight but significant decrease in TGF-β levels at M6, along with fall in mRSS may reflect downregulation of SSc fibroblast activation with a lower inflammatory status after aHSCT in dcSSc patients. Nonetheless, we show that repeated measures of TGF-β serum levels did not reflect the skin score regression post transplant.…”

“…The early phase II studies showed that aHSCT allowed regression of skin and lung fibrosis. 7 Then, the European Society for Blood and Marrow Transplantation (EBMT)-European League Against Rheumatism (EULAR)-randomized Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial demonstrated that aHSCT conferred significant long-term overall and event-free survival benefit in dSSc patients when compared with cyclophosphamide conventional therapy. 8 After aHSCT, the immune reconstitution process in SSc patients is characterized by the reemergence of naive B cells and T cells, and resetting of the immune system, 9 with progressive increases in the regulatory T-cell numbers 10 and in Th1/Th2 cell ratio.…”

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

“…Results from the ASSIST (39) and, ASTIS (31) randomized clinical trials have been published while the SCOT randomized trial results will be available in late 2016 after the last enrolled subject is evaluated at the 54 th month primary endpoint. Table 1 details these three controlled studies.…”

“…Health insurer support for scleroderma transplants currently face fewer restrictions because more individuals now have health insurance coverage and two published randomized trials have shown significant clinical benefit with HSCT (31, 39). Physician referral preferences can also be a barrier as witnessed by patients’ self-referral for transplant consults.…”

“… A American Scleroderma Stem Cell versus Immune Suppression Trial (39) B Autologous Stem Cell Transplantation International Scleroderma (31) C Scleroderma: Cyclophosphamide or Transplantation (ongoing) D 17 Rheumatology and 8 Transplant Centers E Insurance denials for clinical trial coverage often precluded randomization F By cardiac echo, other values by right heart catheterization G Lung and Kidney shielded to 200 cGy transmission (33) …”