Clinical outcome of autologous peripheral blood stem cell transplantation in opticospinal and conventional forms of secondary progressive multiple sclerosis in a Chinese population |
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Authors: | Juan Xu Bing-Xin Ji Li Su Hui-Qing Dong Wan-Ling Sun Sui-Gui Wan Ya-Ou Liu Pu Zhang Cong-Yan Liu |
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Institution: | (1) Department of Hematology, Xuan Wu Hospital, Capital Medical University, No. 45 Changchun Street, Xuan Wu District, Beijing, 100053, China;(2) Department of Neurology, Xuan Wu Hospital, Capital Medical University, Beijing, 100053, China;(3) Department of Radiology, Xuan Wu Hospital, Capital Medical University, Beijing, 100053, China |
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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. |
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