Autologous non-myeloablative hematopoietic stem cell transplantation in patients with systemic sclerosis

Bone Marrow Transplant. 2007 Sep;40(6):549-55. doi: 10.1038/sj.bmt.1705782. Epub 2007 Jul 23.


Autologous hematopoietic stem cell transplantation (HSCT) utilizing a myeloablative regimen containing total body irradiation has been performed in patients with systemic sclerosis (SSc), but with substantial toxicity. We, therefore, conducted a phase I non-myeloablative autologous HSCT study in 10 patients with SSc and poor prognostic features. PBSC were mobilized with CY and G-CSF. The PBSC graft was cryopreserved without manipulation and re-infused after the patient was treated with a non-myeloablative conditioning regimen of 200 mg/kg CY and 7.5 mg/kg rabbit antithymocyte globulin. There was a statistically significant improvement of modified Rodnan skin score whereas cardiac (ejection fraction, pulmonary arterial pressure), pulmonary function (DLCO) and renal function (creatinine) remained stable without significant change. One patient with advanced disease died 2 years after the transplant from progressive disease. After median follow-up of 25.5 months, the overall and progression-free survival rates are 90 and 70% respectively. Autologous HSCT utilizing a non-myeloablative conditioning regimen appears to result in improved skin flexibility similar to a myeloablative TBI containing regimen, but without the toxicity and risks associated with TBI.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Adult
  • Blood Sedimentation
  • Child
  • Erythrocyte Transfusion
  • Female
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Kidney Function Tests
  • Male
  • Middle Aged
  • Platelet Transfusion
  • Prognosis
  • Pulmonary Wedge Pressure
  • Respiratory Function Tests
  • Scleroderma, Systemic / therapy*
  • Skin
  • Stroke Volume
  • Survival Rate
  • Transplantation Conditioning / adverse effects*
  • Transplantation Conditioning / methods*
  • Transplantation, Autologous
  • Treatment Outcome