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目的观察骨髓间充质干细胞(BMSC)移植治疗杜氏型肌营养不良(DMD)治疗前后血清酶学变化及临床效果。方法选择2007年6月至2009年6月解放军第四六三医院515例DMD患者,其中男性502例,女性13例;年龄1～20岁,平均年龄13.3岁。阳性家族史121例。经解放军第四六三医院伦理委员会同意,并征求患者本人及家人签字同意后,进行自体BMSC移植治疗,设自身治疗前后血清酶学变化对照方法进行疗效观察,入组患者均皮下注射粒细胞集落刺激因子,动员4d后采集骨髓,Percoll梯度离心,提取的单个核细胞总量为2.0×109,培养骨髓单个核细胞7~10d,最终得到BMSC总数4.0×108~1.5×109/mL。应用四肢肌内注射的方式进行BMSC移植。结果 BMSC移植治疗12个月肌力增加者占患者总数的79.2%。临床升级人数占27.5%。血清肌酸激酶(CK)降低率81.0%,血清乳酸脱氢酶(LDH)降低率52.8%。结论 BMSC移植治疗DMD,对肌肉组织有一定的修复作用,使DMD患者的运动功能得到改善,肌力有所提高,血清酶学较移植前下降。BMSC移植无不良反应,患者依从性好,是治疗DMD的新手段。
Objective To observe the changes of serum enzyme and clinical effect of bone marrow mesenchymal stem cells (BMSCs) transplantation before and after treatment of Duchenne muscular dystrophy (DMD). Methods From June 2007 to June 2009, 515 DMD patients in the 463 Hospital of PLA, including 502 males and 13 females, aged from 1 to 20 years with a mean age of 13.3 years. Positive family history of 121 cases. With the approval of the People’s Liberation Army’s 463 Hospital Ethics Committee and seeking the consent of patients and their families, autologous BMSC transplantation was performed. The changes of serum enzymatic changes before and after treatment were observed. The patients were injected subcutaneously with granulocyte colony Bone marrow cells were collected 4 days after mobilization. Percoll gradient centrifugation was performed. Total amount of extracted mononuclear cells was 2.0 × 109. Bone marrow mononuclear cells were cultured for 7 ~ 10 days. The total number of BMSCs was 4.0 × 108 ~ 1.5 × 109 / mL. Application of intramuscular injection of limbs for BMSC transplantation. Results BMSC transplantation at 12 months of muscle strength accounted for 79.2% of the total number of patients. The number of clinical upgrades accounted for 27.5%. Serum creatine kinase (CK) decreased 81.0%, serum lactate dehydrogenase (LDH) decreased 52.8%. Conclusion BMSC transplantation for the treatment of DMD has a certain role in the repair of muscle tissue, which can improve the motor function of DMD patients, increase the muscle strength and decrease the serum enzyme level before transplantation. BMSC transplantation without adverse reactions, patients with good compliance, is a new means of treatment of DMD.