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A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Mult Scler 2012 Jun;18(6):825-34

Date

03/03/2012

Pubmed ID

22383228

Pubmed Central ID

PMC3389500

DOI

10.1177/1352458512438454

Scopus ID

2-s2.0-84861802589   64 Citations

Abstract

BACKGROUND: Haematopoietic stem cell transplantation (HSCT) has been tried in the last 15 years as a therapeutic option in patients with poor-prognosis autoimmune disease who do not respond to conventional treatments. Worldwide, more than 600 patients with multiple sclerosis (MS) have been treated with HSCT, most of them having been recruited in small, single-centre, phase 1-2 uncontrolled trials. Clinical and magnetic resonance imaging outcomes from case series reports or Registry-based analyses suggest that a major response is achieved in most patients; quality and duration of response are better in patients transplanted during the relapsing-remitting phase than in those in the secondary progressive stage.

OBJECTIVES: An interdisciplinary group of neurologists and haematologists has been formed, following two international meetings supported by the European and American Blood and Marrow Transplantation Societies, for the purpose of discussing a controlled clinical trial, to be designed within the new scenarios of evolving MS treatments.

CONCLUSIONS: Objectives of the trial, patient selection, transplant technology and outcome assessment were extensively discussed. The outcome of this process is summarized in the present paper, with the goal of establishing the background and advancing the development of a prospective, randomized, controlled multicentre trial to assess the clinical efficacy of HSCT for the treatment of highly active MS.

Author List

Saccardi R, Freedman MS, Sormani MP, Atkins H, Farge D, Griffith LM, Kraft G, Mancardi GL, Nash R, Pasquini M, Martin R, Muraro PA, European Blood and Marrow Transplantation Group, Center for International Blood and Marrow Research, HSCT in MS International Study Group

Author

Marcelo C. Pasquini MD, MS Professor in the Medicine department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Clinical Trials, Phase III as Topic
Cooperative Behavior
Disability Evaluation
Europe
Hematopoietic Stem Cell Transplantation
Humans
International Cooperation
Middle Aged
Multicenter Studies as Topic
Multiple Sclerosis, Relapsing-Remitting
Prospective Studies
Randomized Controlled Trials as Topic
Research Design
Severity of Illness Index
Transplantation, Autologous
Treatment Outcome
United States
Young Adult
jenkins-FCD Prod-482 91ad8a360b6da540234915ea01ff80e38bfdb40a