TY - JOUR
T1 - Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial
T2 - Impact on disease activity and quality of life
AU - Saccardi, Riccardo
AU - Mancardi, Gian Luigi
AU - Solari, Alessandra
AU - Bosi, Alberto
AU - Bruzzi, Paolo
AU - Di Bartolomeo, Paolo
AU - Donelli, Amedea
AU - Filippi, Massimo
AU - Guerrasio, Angelo
AU - Gualandi, Francesca
AU - La Nasa, Giorgio
AU - Murialdo, Alessandra
AU - Pagliai, Francesca
AU - Papineschi, Federico
AU - Scappini, Barbara
AU - Marmont, Alberto M.
PY - 2005/3/15
Y1 - 2005/3/15
N2 - 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.
AB - 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.
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U2 - 10.1182/blood-2004-08-3205
DO - 10.1182/blood-2004-08-3205
M3 - Article
C2 - 15546956
AN - SCOPUS:20144368500
VL - 105
SP - 2601
EP - 2607
JO - Blood
JF - Blood
SN - 0006-4971
IS - 6
ER -