Sustained improvement for stem cell transplantation in systemic sclerosis

A recent report describes improved survival, reduced skin thickening and stabilisation of organ function in systemic sclerosis patients treated with autologous hematopoietic stem cell transplantation (HSCT). To evaluate HSCT as a therapeutic strategy in systemic sclerosis patients with a poor prognosis the investigators enrolled 26 patients with rapidly progressive, diffuse cutaneous systemic sclerosis. Participants had a disease duration of two years or less, a modified Rodnan skin score (mRSS) above 20, an erythrocyte sedimentation rate above 25 and a haemoglobin level below 11 g/dl. Alternatively, patients had disease of more than two years with involvement of one or more major organs. Peripheral blood stem cells were mobilised using cyclophosphamide and G-CSF and were reinfused after positive CD34+ selection. Conditioning was performed with cyclophosphamide before re-injection of the stem cells. After a median follow-up of 5.3 years, 81% (n=21/26) of the patients demonstrated a clinical beneficial response. Survival at five years was estimated at 96.2% and at 84.8% at seven years. Event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% at five years and 57.1% at seven years. According to a recently published meta-analysis, the mortality rate was "strikingly” lower compared to the five-year mortality rate estimated at 40% in such severe systemic sclerosis patients, the authors noted. "Five years after transplantation, the percentage of patients with a World Health Organization performance status of zero had increased to 56% compared to 4% at baseline. Therefore, hematopoietic stem cell transplant treatment showed a sustained positive effect on the combination of functional status, skin, lung, heart and kidney involvement," they concluded. Reference...