Efficacy of allogeneic mesenchymal stem cell transplantation in patients with drug-resistant polymyositis and dermatomyositis
Efficacy of allogeneic mesenchymal stem cell transplantation in patients with drug-resistant polymyositis and dermatomyositis
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DOI：
10.1136/ard.2010.141804
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发表时间：
2011-07-01
影响因子：
27.4
通讯作者：
Sun, Lingyun
中科院分区：
文献类型：
--
作者：
Wang, Dandan;Zhang, Huayong;Sun, Lingyun
目的探讨异基因间充质干细胞移植（MSCT）治疗10例耐药多发性肌炎（PM）或皮肌炎（DM）的安全性和临床疗效。所有患者均同意接受同种异体MSCT检查。结果所有患者的血清肌酸激酶（CK）、肌酸激酶同工酶同工酶（CK-MB）、视觉模拟评分（VAS）、肌力等指标均较术前明显改善，部分患者的肺间质病变改善。在1例患者中观察到慢性不愈合皮肤溃疡的改善。结论多层螺旋CT对耐药DM/PM患者是安全有效的。需要更大规模的研究，包括接受标准治疗的对照组，以评估同种异体MSCT在难治性DM/PM患者中的长期疗效。
Objective To assess the safety and clinical efficacy of allogeneic mesenchymal stem cell transplantation (MSCT) in a small-scale pilot study with 10 patients with drug-resistant polymyositis (PM) or dermatomyositis (DM).Methods A single-arm trial involving 10 patients with DM/PM who were either refractory to standard treatment, or had severe systemic involvement. All patients consented and underwent allogeneic MSCT. Clinical and laboratory manifestations were compared before and after MSCT.Results Improvements were seen in serum creatine kinase (CK), CK-MB, patient global assessment by visual analogue scale and muscle strength by manual muscle test in all patients, as well as improvement in interstitial lung disease in selected patients. Improvement in chronic non-healing skin ulcers was noted in one patient. Clinical responses were also seen in patients undergoing a second MSCT for recurrence of disease.Conclusion MSCT appears safe and effective in drug-resistant patients with DM/PM. Larger-scale studies including a control group receiving standard treatment are needed to assess the long-term efficacy of allogeneic MSCT in refractory patients with DM/PM.