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STUDY: HSCT in RRMS and SPMS, Australia

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  • STUDY: HSCT in RRMS and SPMS, Australia

    Findings after two years. -D

    Autologous haematopoietic stem cell transplantation (AHSCT) in relapse remitting (RR-MS) and secondary progressive multiple sclerosis (SP-MS): a phase II trial from St Vincentís Hospital, Sydney Australia

    Abstract: P1134
    Type: Poster
    Abstract Category: Therapy - disease modifying - 26 Immunomodulation/Immunosuppression

    Objective: To evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation with high-intensity immunosuppresion in patients with multiple sclerosis (MS).

    Methods: A Phase II clinical trial of AHSCT using a BEAM + ATG chemotherapeutic regimen for patients with relapsing remitting and secondary progressive MS began at St Vincent's Hospital in December 2010. Patients eligible for AHSCT had an Expanded Disability Status Scale [EDSS] of 3.0-6.5, have failed two prior disease-modifying therapies and display evidence of ongoing disease activity with clinical relapses and/or new MRI lesions prior to AHSCT.

    Results: Thirty-five participants underwent AHSCT. Median follow-up is 23.6 months
    (range Progression-free survival, clinical relapse-free survival, and MRI activity- free survival were 88.6%, 94.3% and 91.4% respectively. Adverse effects due to AHSCT were consistent with expected toxicities and there were no significant late neurologic adverse effects noted. Changes were noted in neurologic disability with 13/35 (37%) of patients demonstrating a sustained (> 6 months) improvement in EDSS.

    Conclusion: AHSCT was effective for inducing long-term sustained remissions in the majority of active RRMS patients at 5 years. Younger age and lower baseline EDSS scores were associated with better outcomes. The results support the need for further refinement of which patients are most suitable to undergo AHSCT and for longer follow up of cohorts.
    Dave Bexfield