9 janvier, 2008 21:01

Clinical

NEW YORK (Reuters Health) - Autologous hematopoietic stem cell transplantation improves survival and reduces skin thickening and other symptoms of systemic sclerosis, a team of European researchers reports in the January issue of the Annals of the Rheumatic Diseases.

Dr. Madelon C. Vonk, of Radboud University Nijmegen Medical Centre in the Netherlands, and colleagues evaluated the response to autologous hematopoietic stem cell transplantation in 26 patients with rapidly progressive, diffuse cutaneous systemic sclerosis. Patients had a disease duration of 2 years or less, a modified Rodnan skin score (mRSS) above 20, an erythrocyte sedimentation rate above 25 and a hemoglobin level below 11 g/dl. Alternatively, patients had a disease duration of more than 2 years with involvement of one or more major organs.

Peripheral blood stem cells were mobilized using cyclophosphamide and G-CSF and collected by apheresis. The cells were selected for CD34+, and T cells were depleted by 4-5 log. Conditioning was performed with cyclophosphamide 5 to 2 days before reinjection of the stem cells.

After a median follow-up of 5.3 years, 21 of the 26 patients (81%) showed a clinical response. The estimated survival at 5 years was 96.2% and at 7 years was 84.8%. Event-free survival at 5 years was 64.3% and at 7 years it was 57.1%.

The mortality rate "is strikingly lower compared to the 5-year mortality rate estimated at 40% in such severe systemic sclerosis patients, according to a recently published meta-analysis," the investigators report.

Skin scores declined substantially with stem cell transplantation, with a 25% decrease in mRSS being achieved in 73% of patients at 1 year and in 94% after 5 years. "To our knowledge, no other therapeutic intervention in diffuse systemic sclerosis has ever shown such a rapid and sustained improvement in skin condition," Dr. Vonk and colleagues note.

"The strongest effect of treatment by hematopoietic stem cell transplantation was observed on functional status, as assessed by the WHO performance status, possibly due to improvement of the skin condition," the team notes. "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."

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