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Autologous stem cell transplantation in multiple sclerosis: Results from a single centre

Pedrini, M.J., Cull, G., Augustson, B.M., Walters, S., Crosbie, C., Carroll, W.M. and Kermode, A.G. (2015) Autologous stem cell transplantation in multiple sclerosis: Results from a single centre. Multiple Sclerosis Journal, 21 (6). p. 806.

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Background: In the last 2 decades, intensive immunosuppression followed by autologous stem cell transplantation (ASCT) has been proposed as a possible strategy for treatment of severe immune-mediated disorders, including multiple sclerosis (MS).

Objective: To review the outcome of ASCT for MS in Western Australia. Methods: Eligibility criteria for ASCT were (1) progression of sustained disability with expanded disability status scale (EDSS) score increase of more than 1/10 over a 12 month period, (2) advanced MS with threatened loss of ambulation and (3) rapidly progressive disease not adequately assessed by EDSS. Stem cell mobilization was with cyclophosphamide (CY) 2g/m 2 and granu - locyte-colony stimulating factor 5ug/kg bd. conditioning chemo - therapy was with CY 50mg/kg and rabbit antithymocyte globulin 1mg/kg days -5 to -2. Patients were assessed at 3, 6, 12 and 24 months post-transplant.

Results: Fourteen patients underwent ASCT. Median age was 47 years; median time from diagnosis to transplant was 12 years. Diagnosis at transplant was secondary progressive MS (12), pri - mary progressive MS (1) and neuromyelitis optica (1). About half the cohorts were neurologically stable at 24 months while the remainder had clinically relevant neurological deterioration. Two patients had meaningful improvement in bladder function. Follow-up MRI showed no Gd-enhancing lesions, but two patients developed new cerebral lesions on T2 weighted imaging.

Conclusion: In this group of patients with advanced MS, neuro - logical function 24 months post-ASCT was essentially stable in half the cohort while the remainder experienced clinical progres - sion. It is not possible to conclude whether ASCT altered the natu - ral history of the disease.

Item Type: Journal Article
Murdoch Affiliation(s): Institute for Immunology and Infectious Diseases
Publisher: Sage Publications
Copyright: © 2015 by SAGE Publications
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