Stem Cell Transplantation Offers Hope For Severe Scleroderma Interview with:

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Picture of a female patient’s left arm, showing skin lesions caused by Scleroderma
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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. What is the background for this study? What are the main findings?

  • The study endpoint at 54 months after randomization found a significant clinical benefit with transplant as evidenced by a global rank composite scored based on a hierarchy of scleroderma features including death, organ failure, quality of life and skin hardening. At 72 months, overall survival was 86% after transplant vs 51% after cyclophosphamide, a highly significant benefit.
  • Compared to less intensive transplant regimens, treatment-related mortality and autoimmune disease recurrences were both lower. What should readers take away from your report?

  • Patients with scleroderma and internal organ involvement should be referred by their primary physicians for consultation at stem cell transplant centers to weigh the pros and cons of transplant.
  • In severe scleroderma, survival and other clinical benefits of transplant were superior to conventional care and these benefits appeared long-lasting.
  • This approach represents a new standard for effective care of this devastating disease. Thank you for your contribution to the community.


Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma

Keith M. Sullivan, M.D., Ellen A. Goldmuntz, M.D., Ph.D., Lynette Keyes-Elstein, Dr.P.H., Peter A. McSweeney, M.B., Ch.B., Ashley Pinckney, M.S., Beverly Welch, R.N., M.S.N., Maureen D. Mayes, M.D., M.P.H., Richard A. Nash, M.D., Leslie J. Crofford, M.D., Barry Eggleston, M.S., Sharon Castina, R.N., M.S.N., Linda M. Griffith, M.D., M.H.S., Ph.D., Julia S. Goldstein, M.D., Dennis Wallace, Ph.D., Oana Craciunescu, Ph.D., Dinesh Khanna, M.D., Rodney J. Folz, M.D., Ph.D., Jonathan Goldin, M.D., E. William St. Clair, M.D., James R. Seibold, M.D., Kristine Phillips, M.D., Ph.D., Shin Mineishi, M.D., Robert W. Simms, M.D., Karen Ballen, M.D., Mark H. Wener, M.D., George E. Georges, M.D., Shelly Heimfeld, Ph.D., Chitra Hosing, M.D., Stephen Forman, M.D., Suzanne Kafaja, M.D., Richard M. Silver, M.D., Leroy Griffing, M.D., Jan Storek, M.D., Ph.D., Sharon LeClercq, M.D., Richard Brasington, M.D., Mary E. Csuka, M.D., Christopher Bredeson, M.D., Carolyn Keever-Taylor, Ph.D., Robyn T. Domsic, M.D., M.P.H., M. Bashar Kahaleh, M.D., Thomas Medsger, M.D., and Daniel E. Furst, M.D., for the SCOT Study Investigators*

N Engl J Med 2018; 378:35-47January 4, 2018DOI: 10.1056/NEJMoa1703327

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

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Last Updated on January 4, 2018 by Marie Benz MD FAAD