Project details


    Autologous Hematopoietic Stem Cell Transplantation For Secondary Progressive Multiple Sclerosis: a comparative study with matched control patients from the Italian Multiple Sclerosis Register


  • Data start Data end
    2019-06-10 2021-12-31

    Principal Investigator
    Matilde Inglese
    Co-Principal Investigator
    Giacomo Boffa
    Research Unit of the Principal Investigator
    Giovanni Luigi Mancardi, Department of Neurology, Genova
    Daniela Currò, Department of Neurology, Savona
    Elisabetta Capello, Department of Neurology, Genova
    Elvira Sbragia, Department of Neurology, Genova

    External Collaboration
    Luca Massacesi, Department of Neurology, Firenze
    Maria Pia Amato, Department of Neurology, Firenze
    Riccardo Saccardi, Department of Hematology, Bone marrow transplantation unit, Firenze
    Antonio Bertolotto, Department of Neurology, MS center, Torino
    Marco De Gobbi, Department of Hematology, Bone marrow transplantation unit, Torino
    Salvatore Cottone, Department of Neurology, Palermo
    Giovanni Bosco Zimatore, Department of Neurology, MS center, Barletta
    Maria Pia Sormani, Department of Health Sciences, Genova
    Alessio Signori, Department of Health Sciences, Genova
    Francesca Gualandi, Department of Hematology, Bone marrow transplantation unit, Genova


    Introduction and aims
    Treatment of secondary progressive multiple sclerosis (SPMS) remains unsatisfactory. Uncontrolled evidence suggests that autologous hematopoietic stem cell transplantation (AHSCT) can be effective in people with active SPMS, as a result of the profound immune ablative effect of CNS-penetrant chemotherapy. In this study we compared the effect of AHSCT with that of other anti-inflammatory disease modifying therapies (DMT) on long-term disability worsening in active SPMS.
    We collected data from the Italian-Bone-Marrow-Transplantation-Study-Group and the Italian-Multiple-Sclerosis-Register. Patients were considered eligible if treatment had been started after the diagnosis of SPMS. Disability worsening was assessed by the cumulative proportion of patients with a 6-months confirmed-disability-progression (CDP) according to the Expanded-Disability-Status-Scale (EDSS) score. Key secondary endpoints were the EDSS time-trend after treatment start and the prevalence of disability improvement over time. A linear mixed model with a time*treatment group interaction was used to assess the longitudinal EDSS time-trends. Time to CDP was assessed by mean of proportional hazard Cox regression models. Prevalence of improvement was estimated according to a modified Kaplan-Meier estimator and compared between groups by bootstrapping the area under the curve.

    79 AHSCT-treated patients and 1975 patients treated with other DMT were matched to reduce treatment selection bias using propensity-score and overlap weighting approaches. Time to first CDP was significantly longer in transplanted patients (HR= 0.50; 95% CI: 0.31, 0.81; p=0.005), with 61.7% of transplanted patients free from CDP at 5 years. Accordingly, EDSS time-trend over 10 years was higher in patients treated with other DMT than in AHSCT- treated patients (+0.157 EDSS points per year compared to -0.013 EDSS points per year; interaction p<0.001). Patients who underwent AHSCT were more likely to experience a sustained disability improvement: 34.7% of patients maintained an improvement (a lower EDSS than baseline) 3 years after transplant versus 4.6% of patients treated by other DMT (p<0.001).

    The use of AHSCT in people with active SPMS is associated with a slowing of disability progression and a higher likelihood of disability improvement compared to standard immunotherapy

    Pubblicazioni e Comunicazioni a Congressi/ Publications and Congress Presentations

    • Boffa G. et al Hematopoietic Stem Cell Transplantation in people with active Secondary Progressive Multiple Sclerosis, under review Neurology
    • Boffa G. ECTRIMS 2022: Oral communication, Presenter: Young Investigator Award MSIF

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Fondazione Italiana Sclerosi Multipla – FISM – Ente del Terzo Settore/ETS e, in forma abbreviata, FISM ETS.
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