Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: Impact on disease activity and quality of life

Riccardo Saccardi, Gian Luigi Mancardi, Alessandra Solari, Alberto Bosi, Paolo Bruzzi, Paolo Di Bartolomeo, Amedea Donelli, Massimo Filippi, Angelo Guerrasio, Francesca Gualandi, Giorgio La Nasa, Alessandra Murialdo, Francesca Pagliai, Federico Papineschi, Barbara Scappini, Alberto M. Marmont

Research output: Contribution to journalArticlepeer-review


Hematopoietic stem cell transplantation (HSCT) has been proposed for the treatment of severe multiple sclerosis (MS). In a phase 2 multicenter study we selected 19 non-primary progressive MS patients showing high disease activity on the basis of both brain magnetic resonance imaging (MRI) and sustained clinical deterioration despite conventional treatments. After stem cell mobilization with cyclophosphamide (CY) and filgrastim, patients were conditioned with BCNU (1,3-bis(2-chloroethyl)-1-nitrosourea), cytosine arabinoside, etoposide, and melphalan (BEAM) followed by antithymocyte globulin (ATG). Unmanipulated peripheral blood stem cells (PBSCs) were then infused. No maintenance treatment was administered with a median follow-up of 36 months (range, 12 to 72 months). All patients showed clinical stabilization or improvement; 3 subsequently deteriorated, 1 beyond the baseline. No MRI active lesions were detected after the HSCT except in 1 patient who showed a new lesion at 4.5 years. Infections were limited and restricted to 3 months after HSCT. Health-related quality of life was assessed through the 54-item MS quality of life (MSQOL-54) questionnaire, showing a statistically significant improvement in both composite scores and in most of the individual domains. HSCT is able to induce a prolonged clinical stabilization in severe progressive MS patients, resulting in both sustained treatment-free periods and quality of life improvement.

Original languageEnglish
Pages (from-to)2601-2607
Number of pages7
Issue number6
Publication statusPublished - Mar 15 2005

ASJC Scopus subject areas

  • Hematology


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