June 26, 2014

Hematopoietic Stem Cell Transplantation for Multiple Sclerosis: Collaboration of the CIBMTR and EBMT to Facilitate International Clinical Studies


Clinical investigation of autologous hematopoietic stem cell transplantation (HSCT) as therapy for multiple sclerosis (MS) has been ongoing for over a decade. While several phase II studies have been finalized or are in progress, no definitive prospective randomized studies comparing HSCT versus alternative therapies for MS have been completed. Pasquini MC, Griffith LM, Arnold DL, Atkins HL, Bowen JD, Chen JT, Freedman MS, Kraft GH, Mancardi GL, Martin R, Muraro PA, Nash RA, Racke MK, Storek J, Saccardi R. Biol Blood Marrow Transplant 2010 Aug;16(8):1076-1083. Epub 2010 Mar 18.

In this conference report of North American and European experts who are involved in the care of MS patients, including neurologists and HSCT physicians, and representatives of the Center for International Blood and Marrow Transplant Research (CIBMTR) and European Group for Blood and Marrow Transplantation (EBMT), (1) progress to date in HSCT for MS is critically reviewed; (2) current registry based projects including long-term follow-up studies in HSCT for MS and harmonization of the MS disease-specific research forms that will be used in future by both databases are described; (3) challenges in study design for a prospective randomized clinical trial of HSCT are discussed versus alternative therapy for MS such as feasibility, and the importance of multidisciplinary clinical teams, need for a large sample size and duration of observation required for outcomes assessment; and (4) future directions in HSCT therapy for MS are considered.

To undertake a definitive multicenter clinical trial in autologous HSCT for MS, it will be important to begin well in advance to assemble the team, evaluate proposals for study design, and consider options for the infrastructure and logistical support that will be needed. International collaboration, including partnership with the CIBMTR and EBMT, may be desirable and may in fact be critical for successful completion of a definitive comparative study.

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