title: Long-term efficacy of autologous haematopoietic stem cell transplantation in multiple sclerosis at a single institution in China.
abstract: Autologous haematopoietic stem cell transplantation (AHSCT) is a promising treatment for multiple sclerosis (MS) patients who have not adequately responded to conventional therapies. We retrospectively evaluated the safety and long-term clinical outcome of AHSCT in MS patients in China. Twenty-five patients with various types of MS were treated with AHSCT. Peripheral blood stem cells were derived by leukapheresis after mobilized with granulocyte colony-stimulating factor. Then CD34+ cell selection of the graft was performed and anti-thymocyte globulin was given for T-cell depletion, with the conditioning regimen BEAM adopted and early and late toxicities recorded. Long-term responses were evaluated by the expanded disability status scale (EDSS), progression-free survival and gadolinium-enhanced magnetic resonance imaging scans. 10, 7 and 8 patients experienced neurological improvement, stabilization and progression, respectively. The median EDSS scores observed over 1-year follow-up after transplantation (5.5-7.0) were consistently lower than the baseline (8.0). The progression-free survival rate was 74, 65 and 48% at 3, 6 and 9 years post-transplant. 58% cases (7/12) had active lesions at baseline and all turned to inactive status in the years of follow-up. 25% cases (3/12) experienced progression after transplantation but had no active lesions in MRI over the whole follow-up period. 17% cases (2/12) without active lesions at baseline progressed active lesions in MRI. The major early toxicity resulted in fever and late toxicity caused transplantation-related mortality due to severe pneumonia and varicella-zoster virus hepatitis, respectively. AHSCT is a feasible treatment for severe MS and its long-term efficacy is favorable.
authors.affiliation: Department of Hematology, The Affiliated Drum Tower Hospital of Nanjing University Medical School, 321 Zhongshan Road, Nanjing 210008, People's Republic of China.
authors.forename: Bing
authors.initials: B
authors.lastname: Chen
authors.forename: Min
authors.initials: M
authors.lastname: Zhou
authors.forename: Jian
authors.initials: J
authors.lastname: Ouyang
authors.forename: Rongfu
authors.initials: R
authors.lastname: Zhou
authors.forename: Jingyan
authors.initials: J
authors.lastname: Xu
authors.forename: Qiguo
authors.initials: Q
authors.lastname: Zhang
authors.forename: Yonggong
authors.initials: Y
authors.lastname: Yang
authors.forename: Yong
authors.initials: Y
authors.lastname: Xu
authors.forename: Xiaoyan
authors.initials: X
authors.lastname: Shao
authors.forename: Li
authors.initials: L
authors.lastname: Meng
authors.forename: Jing
authors.initials: J
authors.lastname: Wang
authors.forename: Yun
authors.initials: Y
authors.lastname: Xu
authors.forename: Xiushi
authors.initials: X
authors.lastname: Ni
authors.forename: Xueguang
authors.initials: X
authors.lastname: Zhang
mesh_terms: D000293:Adolescent; D000328:Adult; D018952:Antigens, CD34 / Q000378:metabolism; D002681:China; D004185:Disability Evaluation; D018572:Disease-Free Survival; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000097:blood; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D053208:Kaplan-Meier Estimate; D008137:Longitudinal Studies; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000097:blood / Q000601:surgery*; D009460:Neurologic Examination; D060168:Thymocytes / Q000378:metabolism; D055815:Young Adult
chemical_list: D018952:Antigens, CD34; D016179:Granulocyte Colony-Stimulating Factor