Background: Certain stem cell transplantation procedures might slow down inflammatory pathology in multiple sclerosis (MS). Aims: To halt disease progression in aggressive MS by a bone marrow transplantation (BMT) pro...Background: Certain stem cell transplantation procedures might slow down inflammatory pathology in multiple sclerosis (MS). Aims: To halt disease progression in aggressive MS by a bone marrow transplantation (BMT) protocol aimed at maximum T cell suppression. Methods: Autologous BMT was performed in 14 patients with rapid secondary progressive MS (median EDSS score at baseline, 6; median disease duration, five years). To accomplish rigorous T cell ablation, a strong conditioning protocol was chosen-cyclophosphamide, total body irradiation, and antithymocyte globulin. To minimise the possibility of reinfusing mature T cells in the graft, bone marrow, not peripheral blood, was used as the CD34+ stem cell source. Results: Median follow up was 35 months (range, 23- 50). Post-transplant haemopoietic recovery was successful in all patients. Early toxicity included Epstein-Barr virus related post-transplantation lymphoproliferative disorder. Longterm effects were development of antithyroid antibodies (three) and myelodysplastic syndrome (one). One patient died of progressive disease five years after transplantation. Treatment failure, defined by EDSS increase sustained for six months or more, was seen in nine patients and stabilisation or improvement in five. Other clinical parameters generally showed the same outcome. No gadolinium enhanced lesions were seen on post-treatment magnetic resonance imaging, in either cerebral or spinal cord scans. However, cerebrospinal fluid oligoclonal bands remained positive in most cases. Conclusions: This strong immunosuppressive regimen did not prevent clinical progression in patients with aggressive secondary MS. The lack of efficacy, together with some serious side effects, does not favour the use of similar rigorous T cell depleting protocols in the future.展开更多
目的:探讨神经干细胞移植术在治疗痉挛性脑性瘫痪的应用价值。方法33例痉挛性脑性瘫痪患儿,均行靶点刺激加神经干细胞移植手术,靶点为内囊前肢。对病例行术前,术后及半年均表面肌电图检查,并采用当前国际上通用的残疾评价方法——粗大...目的:探讨神经干细胞移植术在治疗痉挛性脑性瘫痪的应用价值。方法33例痉挛性脑性瘫痪患儿,均行靶点刺激加神经干细胞移植手术,靶点为内囊前肢。对病例行术前,术后及半年均表面肌电图检查,并采用当前国际上通用的残疾评价方法——粗大运动评估量表(Gross motor function measure GMFM)及Berg平衡量表(Berg Balance Scale,BBS)进行评估。结果术后表面肌电图复查158组肌肉肌电,改善及显著改善为80组,术后半年与术后3天50组比较,改善及显著改善为10组。术后与术前GFMF(粗大运动评估)比较,T=10.317,P<0.05,具有统计学意义,术后患者粗大运动较术前改善。术后与术前Berg(平衡量表)评估比较,T=6.44,P<0.05,具有统计学意义,术后患者平衡力较术前改善。结论神经干细胞移植可以一定程度地改善脑瘫患者的后遗症,可提高脑瘫患者的运动功能和生活质量。展开更多
文摘Background: Certain stem cell transplantation procedures might slow down inflammatory pathology in multiple sclerosis (MS). Aims: To halt disease progression in aggressive MS by a bone marrow transplantation (BMT) protocol aimed at maximum T cell suppression. Methods: Autologous BMT was performed in 14 patients with rapid secondary progressive MS (median EDSS score at baseline, 6; median disease duration, five years). To accomplish rigorous T cell ablation, a strong conditioning protocol was chosen-cyclophosphamide, total body irradiation, and antithymocyte globulin. To minimise the possibility of reinfusing mature T cells in the graft, bone marrow, not peripheral blood, was used as the CD34+ stem cell source. Results: Median follow up was 35 months (range, 23- 50). Post-transplant haemopoietic recovery was successful in all patients. Early toxicity included Epstein-Barr virus related post-transplantation lymphoproliferative disorder. Longterm effects were development of antithyroid antibodies (three) and myelodysplastic syndrome (one). One patient died of progressive disease five years after transplantation. Treatment failure, defined by EDSS increase sustained for six months or more, was seen in nine patients and stabilisation or improvement in five. Other clinical parameters generally showed the same outcome. No gadolinium enhanced lesions were seen on post-treatment magnetic resonance imaging, in either cerebral or spinal cord scans. However, cerebrospinal fluid oligoclonal bands remained positive in most cases. Conclusions: This strong immunosuppressive regimen did not prevent clinical progression in patients with aggressive secondary MS. The lack of efficacy, together with some serious side effects, does not favour the use of similar rigorous T cell depleting protocols in the future.
文摘目的:探讨神经干细胞移植术在治疗痉挛性脑性瘫痪的应用价值。方法33例痉挛性脑性瘫痪患儿,均行靶点刺激加神经干细胞移植手术,靶点为内囊前肢。对病例行术前,术后及半年均表面肌电图检查,并采用当前国际上通用的残疾评价方法——粗大运动评估量表(Gross motor function measure GMFM)及Berg平衡量表(Berg Balance Scale,BBS)进行评估。结果术后表面肌电图复查158组肌肉肌电,改善及显著改善为80组,术后半年与术后3天50组比较,改善及显著改善为10组。术后与术前GFMF(粗大运动评估)比较,T=10.317,P<0.05,具有统计学意义,术后患者粗大运动较术前改善。术后与术前Berg(平衡量表)评估比较,T=6.44,P<0.05,具有统计学意义,术后患者平衡力较术前改善。结论神经干细胞移植可以一定程度地改善脑瘫患者的后遗症,可提高脑瘫患者的运动功能和生活质量。
