Kim Hyung-Sik, Lee Ji Hyun, Roh Kyoung-Hwan, Jun Hee Jin, Kang Kyung-Sun, Kim Tae-Yoon
Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul, Republic of Korea.
Pusan National University School of Medicine, Busan, Republic of Korea.
Stem Cells. 2017 Jan;35(1):248-255. doi: 10.1002/stem.2401. Epub 2016 Jun 28.
Mesenchymal stem cells (MSCs) have been proven to be therapeutically effective against atopic dermatitis (AD) in preclinical studies. However, the safety and efficacy of MSCs against AD have not yet been investigated in a clinical study. To establish the safety and efficacy of human umbilical cord blood-derived MSCs (hUCB-MSCs) in AD, 34 adult patients with moderate-to-severe AD were enrolled in two phase trials with a follow-up for 1 month and 3 months, respectively. Patients were randomly allocated to receive low dose (2.5 × 10 ) or high dose (5.0 × 10 ) of hUCB-MSCs subcutaneously. An Eczema Area and Severity Index (EASI) score, Investigator's Global Assessment (IGA) score, Severity Scoring for Atopic Dermatitis (SCORAD) score, adverse effect assessments, and serum biomarker levels were evaluated as end points. A single treatment of hUCB-MSCs resulted in dose-dependent improvements in AD manifestation. Fifty-five percent of patients in high dose hUCB-MSC-treated group showed a 50% reduction in the EASI score. The IGA score and SCORAD score decreased by 33% and 50%, respectively, in high dose-treated group. Particularly, the administration of high dose hUCB-MSCs reduced the pruritus score by 58%. The serum IgE levels and number of blood eosinophils were downregulated by the treatment. No serious adverse events occurred, and none of the patients discontinued the trial due to adverse events. This is the first report to demonstrate a marked improvement of AD features with cell therapeutics. These data suggest that the infusion of hUCB-MSCs might be an effective therapy for patients with moderate-to-severe AD. Stem Cells 2017;35:248-255.
间充质干细胞（MSCs）在临床前研究中已被证明对特应性皮炎（AD）具有治疗效果。然而，MSCs治疗AD的安全性和有效性尚未在临床研究中得到调查。为了确定人脐带血来源的间充质干细胞（hUCB-MSCs）治疗AD的安全性和有效性，34例中重度AD成年患者分别参加了两项为期1个月和3个月随访的临床试验。患者被随机分配皮下接受低剂量（2.5×10 ）或高剂量（5.0×10 ）的hUCB-MSCs。以湿疹面积和严重程度指数（EASI）评分、研究者整体评估（IGA）评分、特应性皮炎严重程度评分（SCORAD）、不良反应评估和血清生物标志物水平作为终点指标进行评估。单次给予hUCB-MSCs可使AD症状呈剂量依赖性改善。高剂量hUCB-MSC治疗组55%的患者EASI评分降低了50%。高剂量治疗组的IGA评分和SCORAD评分分别下降了33%和50%。特别是，高剂量hUCB-MSCs的给药使瘙痒评分降低了58%。治疗使血清IgE水平和血液嗜酸性粒细胞数量下调。未发生严重不良事件，也没有患者因不良事件而中止试验。这是第一份证明细胞疗法可显著改善AD症状的报告。这些数据表明，输注hUCB-MSCs可能是中重度AD患者的一种有效治疗方法。《干细胞》2017年；35卷：248 - 255页
