Hematopoietic Stem Cell Transplantation for Multiple Sclerosis
Posted on 12/13/2011
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. 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), we (1) critically review progress to date in HSCT for MS; (2) describe 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; (3) discuss challenges in study design for a prospective randomized clinical trial of HSCT 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) address future directions in HSCT therapy for MS. 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, maybe desirable and may infact be critical for successful completion of a definitive comparative study.
Marcelo C. Pasquini, Linda M. Griffith, Douglas L. Arnold, Harold L. Atkins, James D. Bowen, Jacqueline T. Chen, Mark S. Freedman, George H. Kraft, Gian Luigi Mancardi, Roland Martin, Paolo A. Muraro, Richard A. Nash, Michael K. Racke, Jan Storek, Riccardo Saccardi.
Biol Blood Marrow Transplant 16: 1076-1083 (2010) 2010 American Society for Blood and Marrow Transplantation
KEY WORDS: Allogeneic hematopoietic cell transplantation, Autoimmune disease, Autologous hematopoietic cell transplantation, Clinical trial, Multiple sclerosis
