Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life.
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.
Saccardi
Riccardo
R
Bone Marrow Transplantation Unit, Careggi Hospital, University of Florence, Italy. r.saccardi@dac.unifi.it
Mancardi
Gian Luigi
GL
Solari
Alessandra
A
Bosi
Alberto
A
Bruzzi
Paolo
P
Di Bartolomeo
Paolo
P
Donelli
Amedea
A
Filippi
Massimo
M
Guerrasio
Angelo
A
Gualandi
Francesca
F
La Nasa
Giorgio
G
Murialdo
Alessandra
A
Pagliai
Francesca
F
Papineschi
Federico
F
Scappini
Barbara
B
Marmont
Alberto M
AM
D000328:Adult; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage; D002330:Carmustine / Q000008:administration & dosage; D003561:Cytarabine / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D019650:Hematopoietic Stem Cell Mobilization; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000150:complications / Q000000981:diagnostic imaging / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D011034:Podophyllotoxin / Q000008:administration & dosage; D011788:Quality of Life; D011859:Radiography; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D003561:Cytarabine; D011034:Podophyllotoxin; D008558:Melphalan; D002330:Carmustine