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Boffa, Giacomo; Massacesi, Luca; Inglese, Matilde; Mariottini, Alice; Capobianco, Marco; Moiola, Lucia; Amato, Maria Pia; Cottone, Salvatore; Gualandi, Francesca; De Gobbi, Marco; Greco, Raffaella; Scimè, Rosanna; Frau, Jessica; Zimatore, Giovanni Bosco; Bertolotto, Antonio; Comi, Giancarlo; Uccelli, Antonio; Signori, Alessio; Angelucci, Emanuele; Innocenti, Chiara; Ciceri, Fabio; Repice, Anna Maria; Sormani, Maria Pia; Saccardi, Riccardo; Mancardi, Gianluigi
Neurology, 02/2021, Volume: 96, Issue: 8Journal Article
To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after transplantation in a large cohort of patients with MS. To be included, a minimum dataset (consisting of age, MS phenotype, Expanded Disability Status Scale EDSS score at baseline, information on transplantation technology, and at least 1 follow-up visit after transplantation) was required. Two hundred ten patients were included (relapsing-remitting RR MS 122 58%). Median baseline EDSS score was 6 (1-9); mean follow-up was 6.2 (±5.0) years. Among patients with RRMS, disability worsening-free survival (95% confidence interval CI) was 85.5% (76.9%-94.1%) at 5 years and 71.3% (57.8%-84.8%) at 10 years. In patients with progressive MS, disability worsening-free survival was 71.0% (59.4%-82.6%) and 57.2% (41.8%-72.7%) at 5 and 10 years, respectively. In patients with RRMS, EDSS significantly reduced after aHSCT ( = 0.001; mean EDSS change per year -0.09 95% CI -0.15% to -0.04%). In patients with RRMS, the use of the BCNU+Etoposide+Ara-C+Melphalan (BEAM) + anti-thymocyte globulin (ATG) conditioning protocol was independently associated with a reduced risk of no evidence of disease activity 3 failure (hazard ratio 0.27 95% CI 0.14-0.50, < 0.001). Three patients died within 100 days from aHSCT (1.4%); no deaths occurred in patients transplanted after 2007. aHSCT prevents disability worsening in the majority of patients and induces durable improvement in disability in patients with RRMS. The BEAM + ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity. This study provides Class IV evidence that for people with MS, aHSCT induces durable disease remission in most patients.
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