Yang Xiao-feng, Xu Yi-feng, Zhang Yi-bin, Wang Hong-mei, Lü Nai-wu, Wu Yan-xiang, Lü Xin, Cui Ji-ping, Shan Hong, Yan Yang, Zhou Jin-xu
Cell Treatment Center, PLA No.463 Hospital, Shenyang, China.
Zhonghua Yi Xue Za Zhi. 2009 Sep 29;89(36):2552-6.
To investigate the feasibility of employing double transplantations of autologous bone marrow mesenchymal stem cells (BMSC) and umbilical cord mesenchymal stem cells (UMSC) in the treatment of progressive muscular dystrophy (PMD).
A total of 82 cases were treated by the double transplantations of BMSC and CB-MSC. They were diagnosed by clinical manifestations, CK, LDH, genetic analysis, electromyography, MRI and pathologic examination of biopsied muscle specimens from July 2007 to July 2008. Control group was self-made at before and after treatment and cases were followed up for 3 - 12 months. treatment method: Eighty-two patients underwent the double transplantations of bone mesenchymal stem cell (BMSC) and human umbilical cord blood MSC (CB-MSC). (1) BMSC: 80 - 150 ml bone marrow sample was collected through a puncture at bilateral posterior superior iliac spine. Ficoll density gradient centrifuge was employed to separate individual monocyte for induced differentiation. (2) CB-MSC: 80 - 160 ml umbilical cord blood was harvested and processed likewise as above. (3) Stem cell transplantation: Both BMSC and CB-MSC were collected and prepared into 1 x 10(8)/ml and 1 x 10(7)/ml cell suspension respectively. They were transplanted in divided does into the extremity muscle and vein. The clinical and laboratory parameters were monitored at 3, 6, 9 and 12 months.
It was found that 31 cases (37.8%) obtained a remarkable efficacy, 37 cases (45.1%) were effective and 14 cases (17.1%) had no change. Total effective rate was 82.9%. Seventy patients (85.4%) felt limbs warmly, appetite improved, gained weight, had better appetite and action were nimble. Activity of daily living scale (ADL) in 72 patients (87.8%) increased as compared with pre-treatment (P < 0.01). LDH decreased at post-treatment [(475 +/- 223) u/L vs (410 +/- 216) u/L, P < 0.05, t = 6.650]. Creatine kinase [(2952 +/- 2259) u/L vs (2841 +/- 2092) u/L, P = 0.223, t = 1.094] and creatine [(26 +/- 12) micromol/L vs (25 +/- 11) micromol/L, P = 0.306, t = 1.029] decreased slightly. Adherence to therapy among Children and no adverse reaction was reported during the course of treatment.
The double transplantation of BMSC and CB-MSC is convenient, safe and effective in the treatment of progressive muscular dystrophy and can be considered as a new therapy of PMD. MSC represents a possible tool of cellular therapeutics for PMD.
探讨自体骨髓间充质干细胞（BMSC）与脐带间充质干细胞（UMSC）双重移植治疗进行性肌营养不良（PMD）的可行性。
选取2007年7月至2008年7月间82例经临床表现、肌酸激酶（CK）、乳酸脱氢酶（LDH）、基因分析、肌电图、磁共振成像（MRI）及肌肉活检病理检查确诊的患者，采用BMSC与脐带血间充质干细胞（CB - MSC）双重移植治疗。治疗前后设自身对照，随访3 - 12个月。治疗方法：82例患者接受骨髓间充质干细胞（BMSC）与人脐带血间充质干细胞（CB - MSC）双重移植。（1）BMSC：经双侧髂后上棘穿刺采集80 - 150 ml骨髓样本，采用Ficoll密度梯度离心法分离单个核细胞进行诱导分化。（2）CB - MSC：采集80 - 160 ml脐带血，处理方法同上。（3）干细胞移植：将采集制备好的BMSC和CB - MSC分别制成1×10⁸/ml和1×10⁷/ml的细胞悬液，分剂量移植至肢体肌肉及静脉内。于3、6、9及12个月监测临床及实验室指标。
显效31例（37.8%），有效37例（45.1%），无效14例（17.1%），总有效率82.9%。70例（85.4%）患者自觉肢体温热，食欲改善，体重增加，食欲及行动较前灵活。72例（87.8%）患者日常生活活动量表（ADL）评分较治疗前升高（P < 0.01）。治疗后LDH降低[（475±223）U/L vs（410±216）U/L，P < 0.05，t = 6.650]。肌酸激酶[（2952±2259）U/L vs（2841±2092）U/L，P = 0.223，t = 1.094]及肌酸[（26±１2）μmol/L vs（25±11）μmol/L，P = 0.306，t = 1.029]略有降低。患儿治疗依从性好，治疗过程中未报告不良反应。
BMSC与CB - MSC双重移植治疗进行性肌营养不良方便、安全、有效，可作为PMD的一种新的治疗方法。间充质干细胞是PMD细胞治疗的一种可能手段。
