Author Interviews, Duke, NEJM, Rheumatology, Stem Cells, Transplantation / 04.01.2018

MedicalResearch.com Interview with: Keith M. Sullivan, M.D. James B. Wyngaarden Professor Of Medicine Division of Cellular Therapy Duke University Medical Center Durham, North Carolina 27710, USA  MedicalResearch.com: What is the background for this study? What are the main findings?
  • Scleroderma with internal organ involvement is a devastating¬† autoimmune disorder with considerable morbidity and high mortality which have not changed in 40 years of reporting. Effective new therapies are needed.
  • Despite 2 prior randomized trials showing benefit for reduced-intensity stem cell transplant vs. conventional cyclophosphamide immune suppression, clinical practice in the US did not change due in part due to concern about patient safety and durability of response (attached).
  • The current randomized trial compares 12 monthly infusions of cyclophosphamide with high-dose chemotherapy plus whole-body irradiation designed to wipe-out (myeloablate) the defective, self-reactive immune system and replace with the patients own stem cells which had been treated to remove self-reacting lymphocytes. This was the first study to test if myeloablative autologous could re-establish a normal functioning immune system in patients with scleroderma.
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