目的：本研究的目的是评估人脐带血间充质干细胞(hUC-MSC)治疗狼疮肾炎(LN)的有效性。既往的研究提示hUC-MSC具有非常显著的疗效。

        方法：共纳入18例III或IV型LN的患者，随机分为hUC-MSC(剂量2×108细胞)及安慰剂组。所有患者均接受标准免疫抑制剂治疗，初始应用静脉甲强龙及环磷酰胺，随后应用维持量口服强的松及吗替麦考纷酯。

        结果：hUC-MSC组12例患者中9例(75%)缓解，安慰剂组6例患者5例缓解(83%)。缓解定义为病情稳定或肾功能改善或尿红细胞、尿蛋白减少。hUC-MSC组合安慰剂组达到完全缓解的比例类似。血清白蛋白、补体、肾功能、SLEDAI评分、BILAG评分的改善在两组间无差别。安慰剂组中一人发生卒中，另一人发生腹腔积液。hUC-MSC组中一人发生白细胞减低，肺炎及亚急性脓肿形成，另一人死于重症肺炎。因观察过程中发现本实验无阳性结果出现的提示故而终止了试验。

        结论：与标准免疫抑制剂治疗相比,hUC-MSC并无显著疗效。

        附原文

        Abstract：OBJECTIVE:We evaluate the efficacy of human umbilical cord-derived mesenchymal stem cell (hUC-MSC) for the treatment oflupus nephritis(LN). Previous reports showed hUC-MSC could have dramatic treatment effect.METHODS:Eighteen patients with WHO class III or IV LN were randomly assigned to hUC-MSC (dose 2×108cells) or placebo. All patients received standard immunosuppressive treatment, which consisted of intravenous methylprednisolone and cyclophosphamide, followed by maintenance oral prednisolone and mycophenolate mofetil.RESULTS:Remission occurred in 9 of 12 patients (75%) in the hUC-MSC group and 5 of 6 patients (83%) in the placebo group. Remission was defined as stabilisation or improvement in renal function, reduction in urinary red cells and protein. A similar proportion of patients on hUC-MSC and placebo achieved complete remission. Improvements in serum albumin, complement, renal function, SystemicLupusErythematosus Disease Activity Index and British IslesLupusAssessment Group scores were similar in both groups. One patient on placebo had a stroke and another had ascites. One patient on hUC-MSC had leucopenia, pneumonia and subcutaneous abscess and another died of severe pneumonia. The trial was abandoned after 18 patients were enrolled when it had become obvious it would not demonstrate a positive treatment effect.CONCLUSION:hUC-MSC has no apparent additional effect over and above standard immunosuppression.

        引自

        Deng D,Zhang P,Guo Y,Lim TO.A randomised double-blind, placebo-controlled trial of allogeneic umbilical cord-derived mesenchymal stem cell forlupus nephritis.Ann Rheum Dis.2017 Aug;76(8):1436-1439.