title: Clinical outcome of autologous peripheral blood stem cell transplantation in opticospinal and conventional forms of secondary progressive multiple sclerosis in a Chinese population.
abstract: To evaluate clinical outcomes of autologous peripheral blood stem cell transplantation (APBCST) between opticospinal multiple sclerosis (OSMS) and conventional multiple sclerosis (CMS) during disease progressive stage in a Chinese population. Thirty-six secondary progressive MS patients, among whom 21 were with OSMS and 15 with CMS, underwent APBSCT and were followed up for an average of 48.92 months (range, 10-91 months). Peripheral blood stem cells were obtained by leukapheresis after mobilization with granulocyte colony-stimulating factor. Modified BEAM conditioning regimen (Tiniposide, melphalan, carmustin, and cytosine arabinoside) were administered. Outcomes were evaluated using the expanded disability status scale (EDSS). No maintenance treatment was administered if there was no disease progression. No treatment-related mortality occurred. Among the 36 patients, one OSMS patient dropped during the follow-up. Among the 22 relapse-free patients, 20 were with continuous neurological improvement without any relapse events, and two remained in neurologically stable states. Among the 13 relapse patients, seven had experienced of neurological relapse, but with no progression during the follow-up period; and six experienced neurological deterioration after transplantation and needed further immunosuppressant treatment. The confirmed relapse-free survival rate was 62.9% and progression-free survival rate was 83.3% after 91 months according to Kaplan and Meier survival curves. Eleven of the 20 OSMS patients (55%) and two of the 15 CMS patients (13.3%) stayed in disease active group (P = 0.014). For the 20 OSMS patients, the overall EDSS score decreased significantly after transplantation (P = 0.016), while visual functions had no significant improvement (P = 0.716). Progressive OSMS has a higher relapse rate than CMS following APBSCT.
authors.affiliation: Department of Hematology, Xuan Wu Hospital, Capital Medical University, No. 45 Changchun Street, Xuan Wu District, Beijing, China. xujuandail@x263.net
authors.forename: Juan
authors.initials: J
authors.lastname: Xu
authors.forename: Bing-Xin
authors.initials: BX
authors.lastname: Ji
authors.forename: Li
authors.initials: L
authors.lastname: Su
authors.forename: Hui-Qing
authors.initials: HQ
authors.lastname: Dong
authors.forename: Wan-Ling
authors.initials: WL
authors.lastname: Sun
authors.forename: Sui-Gui
authors.initials: SG
authors.lastname: Wan
authors.forename: Ya-Ou
authors.initials: YO
authors.lastname: Liu
authors.forename: Pu
authors.initials: P
authors.lastname: Zhang
authors.forename: Cong-Yan
authors.initials: CY
authors.lastname: Liu
mesh_terms: D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002330:Carmustine / Q000627:therapeutic use; D002681:China; D003561:Cytarabine / Q000627:therapeutic use; D018572:Disease-Free Survival; D005047:Etoposide / Q000627:therapeutic use; D005260:Female; D019650:Hematopoietic Stem Cell Mobilization; D006801:Humans; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D009471:Neuromyelitis Optica / Q000473:pathology / Q000503:physiopathology / Q000628:therapy; D036102:Peripheral Blood Stem Cell Transplantation; D012008:Recurrence; D012074:Remission Induction; D013116:Spinal Cord / Q000473:pathology; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D003561:Cytarabine; D005047:Etoposide; D008558:Melphalan; D002330:Carmustine