Long-term clinical outcomes of hematopoietic stem cell transplantation in multiple sclerosis

Giacomo Boffa, Luca Massacesi, Matilde Inglese, Alice Mariottini, Marco Capobianco, Lucia Moiola, Maria Pia Amato, Salvatore Cottone, Francesca Gualandi, Marco de Gobbi, Raffaella Greco, Rosanna Scimè, Jessica Frau, Giovanni Bosco Zimatore, Antonio Bertolotto, Giancarlo Comi, Antonio Uccelli, Alessio Signori, Emanuele Angelucci, Chiara InnocentiFabio Ciceri, Anna Maria Repice, Maria Pia Sormani, Riccardo Saccardi, Gianluigi Mancardi

Research output: Contribution to journalArticlepeer-review

17 Scopus citations


Objective 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. Methods 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. Results 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 (p = 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], p < 0.001). Three patients died within 100 days from aHSCT (1.4%); no deaths occurred in patients transplanted after 2007. Conclusions 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.

Original languageEnglish
Pages (from-to)E1215-E1226
Issue number8
StatePublished - 23 Feb 2021
Externally publishedYes


Dive into the research topics of 'Long-term clinical outcomes of hematopoietic stem cell transplantation in multiple sclerosis'. Together they form a unique fingerprint.

Cite this