Mark S. Freedman, University of Ottawa, Ottawa, Ontario, Canada, described the beneficial effects of immunoablation plus autologous stem cell transplantation (ASCT) in the Canadian BMT trial of patients with aggressive MS. Among 16 patients who had failed 1 year or more of standard treatment, 6 exhibited sustained improvements in Expanded Disability Status Scale (EDSS) scores after follow-up ranging from 1.5 to 8 years, while 7 patients remained stable and 3 worsened compared to baseline.
Due to patient safety considerations (one early death due to busulfan-induced complications), busulfan dosing was later modified from 1 mg/kg/dose every 6 hours for 16 doses to 0.8 (0.6 in future) mg/kg IV for 16 doses, to avoid first pass effects and to minimize liver toxicity, Dr. Freedman noted.
In conclusion, our data show that axonal damage and demyelination are both active in patients with long-standing disease and high EDSS scores following autologous stem cell transplantation and the immunosuppression associated with it. It is therefore unlikely that AHSCT and/or potent immunosuppression halts disease progression, despite suppressing the inflammatory aspects of the disease.
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