Author Interviews, Duke, NEJM, Rheumatology, Stem Cells, Transplantation / 04.01.2018 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 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.
Author Interviews, Hematology, JAMA, Stem Cells, Transplantation / 03.07.2014

John F. Tisdale, Interview with: Dr. John Tisdale MD Molecular and Clinical Hematology Branch National Institute of Diabetes and Digestive and Kidney Diseases, National Heart, Lung, and Blood Institute, Bethesda, Maryland MedicalResearch: What are the main findings of the study? Dr. Tisdale: Using a nonmyeloablative allogeneic HLA-match peripheral blood stem cell transplantation strategy aimed at tolerance induction, we were able to revert the phenotype in 26 of 30 adult patients with severe sickle cell disease ranging in age from 16 to 65 years. In contrast to standard transplantation strategies which rely on high doses of chemo and/or radiotherapy after which the entire bone marrow and blood system is replaced by that of the donor, our patients had a mixture of their own and that of their donor. This procedure was well tolerated, with no non-relapse mortality, and led to complete replacement of red blood cells by that of the donor in successfully engrafted patients. This replacement resulted in decreases in pain, pain medication usage, hospitalizations, and improvements in organ function. (more…)