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Systemic scleroderma
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==Research== Given the difficulty in treating scleroderma, treatments with a smaller [[evidence based medicine|evidence base]] are often tried to control the disease. These include [[antithymocyte globulin]] and [[mycophenolate mofetil]]; some reports have shown improvements in the skin symptoms, as well as delaying the progress of systemic disease, but neither has been subjected to large clinical trials.<ref name=Zandberg/> Autologous [[hematopoietic stem cell transplantation]] (HSCT) is based on the assumption that autoimmune diseases such as systemic sclerosis occur when the white blood cells of the immune system attack the body. In this treatment, stem cells from the patient's blood are extracted and stored to preserve them. The patient's white blood cells are destroyed with cyclophosphamide and rabbit antibodies against the white blood cells. Then, the stored blood is returned to the patient's bloodstream to reconstitute a healthy blood and immune system that will not attack the body. The results of a phase-III trial, the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial, with 156 patients, were published in 2014. HSCT itself has a high treatment mortality, so in the first year, the survival of patients in the treatment group was lower than the placebo group, but at the end of 10 years, the survival in the treatment group was significantly higher. The authors concluded that HSCT could be effective, if limited to patients who were healthy enough to survive HSCT itself. Therefore, HSCT should be given early in the progression of the disease, before it does damage. Patients with heart disease, and patients who smoked cigarettes, were less likely to survive.<ref name="JAMA2014-editorial">{{cite journal| title =Autologous Hematopoietic Stem Cell Therapy in Severe Systemic Sclerosis: Ready for Clinical Practice?| journal =JAMA | date =June 25, 2014 |vauthors=Dinesh Khanna D, Georges GE, Couriel DR | volume =311| issue =24| pages =2485β2487 | doi =10.1001/jama.2014.6369| pmid =25058081| pmc =4926767}}</ref><ref name="JAMA2014">{{cite journal |title=Autologous Hematopoietic Stem Cell Transplantation vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis: A Randomized Clinical Trial |journal=JAMA |date=June 25, 2014 |vauthors=van Laar JM, Farge Sont JK, etal |volume=311 |issue=24 |pages=2490β2498 |doi=10.1001/jama.2014.6368 |pmid=25058083 |hdl=2066/136804 |s2cid=205060178 |url=https://eprints.whiterose.ac.uk/134696/1/P909.pdf |hdl-access=free}}</ref> Another trial, the Stem Cell Transplant vs. Cyclophosphamide (SCOT) trial, is ongoing.<ref>Stem Cell Transplant vs. Cyclophosphamide (SCOT), NCT00114530</ref> [[Asengeprast]] is an experimental systemic scleroderma drug candidate. It is a small molecule inhibitor of the G-protein coupled receptor GPR68 with antifibrotic activity.
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