异基因间充质干细胞移植治疗多发性肌炎/皮肌炎的临床研究

目的 探讨异基因间充质干细胞(MSC)移植治疗多发性肌炎/皮肌炎(PM/DM)的临床疗效及安全性.方法 8例难治性PM/皮肌炎患者经静脉输注异基因骨髓或脐带来源MSC,评价患者移植前后血清肌酸激酶(CK)、手工肌力评分(MMT8)等实验室及临床指标,采用配对t检验.结果 异基因MSC移植后随访6～12个月.5例患者移植后1周血清CK值由移植前(1681±430)U/L下降至(886±248)U/L(P＜0.05),移植后2周(474±61)U/L、1个月(293±89)U/L、3个月(202±70)和6个月(175±46)U/L继续下降,与移植前比,差异均有统计学意义(P＜0.05).2例患者在随访9个月时病情复发,血清CK上升.MMT8在移植后1个月67±3、3个月64±10、6个月64±4均显著上升,与移植前45±14比,差异有统计学意义(P＜0.05).所有患者移植后2周口服糖皮质激素(甲泼尼龙)用量较移植前显著减少[(18±6)mg与(34±15)mg,P＜0.05].2例以肺间质病变为主的患者,移植后临床症状缓解、肺部高分辨率CT显示间质性肺炎明显改善.1例以皮肤难治性溃疡为主的皮肌炎患者,移植后溃疡明显愈合.所有患者均无移植相关并发症.结论 异基因MSC移植治疗PM/皮肌炎安全有效,其长期疗效有待进一步观察.

Abstract：

Objective To explore the clinical efficacy of allogenic mesenchymal stem cells transplantation (MSCT) in patients with refractory polymyositis/dermatomyositis (PM/DM). Methods Bone marrow derived mesenchymal stem cells (BM-MSC) or umbilical cord derived mesenchymal stem cells (UC-MSC)were infused intravenously in 8 PM/DM patients. The clinical manifestations and laboratory parameters,including serum creatin in kinase (CK) and manual muscle test 8 (MMT8) score, were compared before and after MSCT. Staistically andlyzed by paired t-test. Results The eight patients were followed up for six to twelve months after MSCT. The level of serum CK decreased from (1681±430) to (886±248) U/L one week after MSCT (P＜0.05) and further decreased at week 2 (474±61) U/L, 1 month (293±89) U/L, 3 month (202±70) U/L and 6 month (175±46) U/L, respectively (all P＜0.05). MMT8 score increased to 1 month [(67±3) vs (45±14), P＜0.05], 3 month [(64±10) vs (45±14), P＜0.05], 6 month [(64±4) vs (45±14),P＜0.05] after MSCT. The dosage of glucocorticoid steroid were tapered in all patients 2 weeks after MSCT [(18±6) mg vs (34±15) mg, P＜0.05]. Clinical symptoms of interstitial pneumonia of both patients were relieved after MSCT, which was confirmed by the result of high resolution CT (HRCT) of the lung.The skin ulcers tended to be recovered after the transplantation in one DM patient. All patients did not develop transplantation related complications. Conclusion Allogenic MSCT is an effective and safe approach for the treatment of refractory PM/DM. However, extensive follow-up study is needed for long-term benefit evaluation.

Clinical effect evaluation of allogenic mesenchymal stem cells transplantation for patients with polymyositis/dermatomyositis

Objective To explore the clinical efficacy of allogenic mesenchymal stem cells transplantation (MSCT) in patients with refractory polymyositis/dermatomyositis (PM/DM). Methods Bone marrow derived mesenchymal stem cells (BM-MSC) or umbilical cord derived mesenchymal stem cells (UC-MSC)were infused intravenously in 8 PM/DM patients. The clinical manifestations and laboratory parameters,including serum creatin in kinase (CK) and manual muscle test 8 (MMT8) score, were compared before and after MSCT. Staistically andlyzed by paired t-test. Results The eight patients were followed up for six to twelve months after MSCT. The level of serum CK decreased from (1681±430) to (886±248) U/L one week after MSCT (P＜0.05) and further decreased at week 2 (474±61) U/L, 1 month (293±89) U/L, 3 month (202±70) U/L and 6 month (175±46) U/L, respectively (all P＜0.05). MMT8 score increased to 1 month [(67±3) vs (45±14), P＜0.05], 3 month [(64±10) vs (45±14), P＜0.05], 6 month [(64±4) vs (45±14),P＜0.05] after MSCT. The dosage of glucocorticoid steroid were tapered in all patients 2 weeks after MSCT [(18±6) mg vs (34±15) mg, P＜0.05]. Clinical symptoms of interstitial pneumonia of both patients were relieved after MSCT, which was confirmed by the result of high resolution CT (HRCT) of the lung.The skin ulcers tended to be recovered after the transplantation in one DM patient. All patients did not develop transplantation related complications. Conclusion Allogenic MSCT is an effective and safe approach for the treatment of refractory PM/DM. However, extensive follow-up study is needed for long-term benefit evaluation.