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Stem cell transplantation for systemic sclerosis: many steps forward in Poland

Dominique Farge
Aleksandra Zoń-Giebel
Eugeniusz J. Kucharz

Data publikacji online: 2016/02/11
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After twenty years of progress in stem cell transplantation for autoimmune diseases, the final results from the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial published a year ago [1] demonstrated that autologous haematopoietic stem cell transplantation (AHSCT) confers better long-term survival than 12 monthly intravenous pulses of cyclophosphamide (Cy) for the treatment of severe or rapidly progressive systemic sclerosis (SSc). In 2015, the Polish Society of Rheumatology, under the Chair of Prof. Eugene J. Kucharz, acknowledged and made efforts to disseminate the important results obtained for treatment of SSc patients. Indeed, many Polish rheumatologists and their voluntary patients as well as other EULAR (European League Against Rheumatism) colleagues met at the end of February 2015 for the EUSTAR (European Scleroderma Trials and Research Group) meeting. The EUSTAR meeting, hosted in Katowice, contributed to raising awareness about SSc, a rare heterogeneous autoimmune disease, which affects 2–22 people per one million every year and is characterized by vasculopathy, autoantibody formation, low-grade inflammation, and progressive fibrosis in the skin and internal organs (lung, heart, kidney and digestive tract). The limited (lcSSc) and diffuse cutaneous (dcSSc) forms can be distinguished by the extent of skin and organ involvement and autoantibody profile, and rapidly progressing dcSSc within the first 4 years after disease onset, observed in to 20% of cases, is a life-threatening disease with 3–5 year survival between 50 and 70%.
The EUSTAR Educational Course allowed attendees to gain knowledge in the understanding of SSc’s complex pathogenesis and the importance of the new 2013 ACR-EULAR criteria [2] for early diagnosis and adequate available therapy. Systemic sclerosis can lead to premature death by heart involvement, lung fibrosis and pulmonary hypertension, especially in patients suffering from dcSSc. These patients constitute up to 30% of the total SSc population and may benefit from AHSCT. This topic, however, was not addressed during the EUSTAR course. Therefore, a second educational course was organized in Wisla with several Polish and European EBMT members. This second meeting illustrated the activity of Polish rheumatologists in the field of SSc and was a nice opportunity to focus on innovation with stem cell transplantation applied to SSc achieved over the past twenty years.
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