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FRI0406 Seven-year follow-up of autologous hematopoietic stem cell transplantation for severe systemic sclerosis
  1. H. Tsukamoto1,
  2. T. Horiuchi1,
  3. T. Miyamoto1,
  4. H. Niiro1,
  5. Y. Arinobu2,
  6. M. Akahoshi1,
  7. M. Ayano1,
  8. A. Tanaka1,
  9. N. Ueda1,
  10. K. Akashi1
  1. 1Department of Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences
  2. 2Center for Cellular and Molecular Medicine, Kyushu University Hospital, Fukuoka, Japan


Background Autologous stem cell transplantation for international scleroderma (ASTIS) trial demonstrated that autologous hematopoietic stem cell transplantation (auto-HSCT) is more effective than conventional intravenous cyclophosphamide in the treatment of severe systemic sclerosis (SSc). However, there is paucity of the study showing the long-term efficacy of auto-HSCT for severe SSc.

Objectives The aim of this study is to investigate seven-year follow-up results of auto-HSCT for severe SSc.

Methods Nineteen patients (4 male and 15 female) with severe SSc were enrolled. Peripheral blood stem cells (PBSCs) were mobilized with cyclophosphamide (CY, 4 g/m2) and G-CSF. After collecting PBSCs more than 2x 106CD34+cells/kg by apheresis, CD34+ cells were immunologically selected in 11 patients. All of the patients were treated with high-dose CY (200 mg/kg) and received CD34-selected (n=11; a group with CD34-selection) or unmanipulated (n=8; a group without CD34-selection) auto-HSCT. Immune reconstitution was evaluated serially by analyzing lymphocyte subpopulations for 36 months after HSCT.

Results There was no treatment-related mortality. As toxicity, there were a variety of post-transplant infections such as adenoviral hemorrhagic cystitis, herpes zoster and cytomegaloviral antigenemia. Sclerosis of the skin was markedly improved in all of the patients within 6 months and the improvement was sustained for 84 months after auto-HSCT. Vital capacity was significantly increased at 48 and 60 months after HSCT and KL-6, a marker for interstitial pneumonia (IP), was significantly decreased in patients with IP during 12-84 months after HSCT. A titer of anti-Scl-70 was significantly decreased during 1-84 months after HSCT. Five and seven years after auto-HSCT, the overall survival was 89 and 78%, and the progression-free survival was 65 and 57%, respectively. Interestingly, the reduction of the skin scores was significantly greater and viral infections were more frequent in the patients with CD34-selection than those without. Effect of auto-HSCT on interstitial pneumonia, and the recovery of lymphocyte subpopulations after auto-HSCT were not significantly different between two groups.

Conclusions Auto-HSCT was effective in the treatment of SSc and the effect was sustained for 7 years.


  1. Tsukamoto H, Nagafuji K, Horiuchi T, et al. A Phase I-II Trial of Autologous Peripheral Blood Stem Cell Transplantation in the Treatment of Refractory Autoimmune Disease. Ann Rheum Dis. 65: 508-14, 2006.

  2. Tsukamoto H, Nagafuji K, Horiuchi T, et al. Analysis of immune reconstitution after autologous CD34+ stem/progenitor cell transplantation for systemic sclerosis: predominant reconstitution of Th1 CD4+ T cells. Rheumatology 2011; 50: 944-52.

Disclosure of Interest None Declared

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