[P02.145] Neurological Recovery Following Treatment of Aggressive MS with Immunoablation and Autologous Stem Cell Transplantation

Mark S. Freedman, Harold L. Atkins, Marjorie Bowman, Canadian BMT Study Group, Ottawa, ON, Canada

OBJECTIVE: Review the clinical (EDSS) and MRI (T2 lesion volume) data for changes of improvement in patients who had between 1 and 8 years of follow-up. BACKGROUND: We hypothesized that complete immunoablation abrogates further immune-mediated CNS damage and that reconstitution with hematopoietic stem cells (HSC) would result in disease tolerance. In the absence of ongoing damage we considered that the HSC might also help to stimulate CNS repair by differentiation into other types of cells, including CNS cells that could migrate to the CNS. DESIGN/METHODS: The Canadian BMT trial is a non-randomized phase II trial of immunoablation and purified autologous hematopoietic stem cell transplantation in aggressive MS patients who have failed 1 year of standard treatment. Immunoablation involved busulphan, cyclophosphamide and rabbit ATG. Patients were assessed clinically every 3 months and MRI scanning performed every 6 months. RESULTS: 6/16 patients with 1.5 year of follow-up (range 1.5-8 years) showed sustained EDSS improvements. (3/16 worsened and 7/16 were unchanged compared with baseline.) Improvements varied with the duration of MS prior to the treatment; those showing the earliest changes also had the shortest disease course. Changes in all aspects of KFS scores were noted; particularly in pyramidal, cerebellar, brainstem and visual systems. Clinical stability was mirrored by the lack of any perceptible MRI activity. No gadolinium enhancing lesions were seen whatsoever. T2 lesion volumes remained stable in 7/16 ( 10% change), but the majority (9/16) showed overall reductions compared to baseline (range 12-34%). CONCLUSIONS/RELEVANCE: Immunoablation and ASCT stabilizes or improves these very aggressive MS patients for up to 8 years. Some demonstrated remarkable improvements in their functional scores. The degree of improvement varied widely across patients, however the timing of these changes correlated with the duration of MS prior to treatment; patients with longer disease took longer to show any perceived benefit. Individualized data will be presented. Supported by: Multiple Sclerosis Foundation of Canada
Category - MS and Related Diseases - Clinical Science

Tuesday, April 28, 2009 11:30 AM

Poster Session II: Multiple Sclerosis: Outcomes and Scales I (11:30 AM-2:30 PM)