Scleroderma future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

Overview

Future or Investigational Therapies

Given the difficulty in treating scleroderma, treatments with a smaller evidence base are often tried to control the disease. These include antithymocyte globulin and mycophenolate mofetil; some reports have reported improvements in the skin symptoms as well as delaying the progress of systemic disease, but neither of them have been subjected to large clinical trials.[1]

While still experimental (given its high rate of complications), hematopoietic stem cell transplantation is being studied in patients with severe systemic sclerosis; improvement in life expectancy and severity of skin changes has been noted.[2]

References

  1. Zandman-Goddard G, Tweezer-Zaks N, Shoenfeld Y (2005). "New therapeutic strategies for systemic sclerosis--a critical analysis of the literature". Clin. Dev. Immunol. 12 (3): 165–73. PMID 16295521. PMC 2275417
  2. Nash RA, McSweeney PA, Crofford LJ; et al. (2007). "High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the U.S. multicenter pilot study". Blood. 110 (4): 1388–96. doi:10.1182/blood-2007-02-072389. PMID 17452515.

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