探讨干细胞疗法修复软骨缺损的有效性与安全性：随机对照试验的Meta分析
简介
该研究为一项纳入24项随机对照试验（共1389例）的Meta分析，评估间充质干细胞治疗骨关节炎软骨缺损的疗效与安全性。结果显示干细胞治疗较对照组显著改善疼痛（SMD=-1.31）、WOMAC评分（SMD=-0.78）及软骨体积（SMD=0.91），不良事件率无显著差异，但各研究间异质性较大，亚组分析仅为探索性。该证据主要针对骨关节炎而非运动损伤性软骨缺损，且…
英文摘要
BACKGROUND: Osteoarthritis (OA) is one of the most common degenerative joint diseases worldwide, characterized by chronic pain, functional impairment, and progressive cartilage loss. Mesenchymal stem cells (MSCs) therapy has emerged as a promising regenerative strategy due to its chondrogenic and immunomodulatory properties. Although randomized controlled trials (RCTs) have generally demonstrated favorable therapeutic effects, differences in study design, patient characteristics, and treatment protocols have resulted in substantial clinical and methodological heterogeneity. Therefore, an updated meta-analysis is needed to comprehensively evaluate the efficacy and safety of MSC therapy. OBJECTIVE: To systematically assess the efficacy and safety of stem cell therapy compared with control interventions in patients with OA with primary emphasis on pain, functional outcomes, cartilage regeneration, and adverse events, and to identify potential factors influencing therapeutic outcomes. METHODS: A comprehensive search of PubMed, Web of Science, Embase, Medline, Scopus, and the Cochrane Library was performed to identify randomized controlled trials (RCTs) published up to 1 August 2025. Clinically relevant outcomes included pain, WOMAC, KOOS subscales, cartilage volume, and adverse events, which were analyzed as key outcome domains reflecting pain, function, structural change, and safety in osteoarthritis. Meta-analysis was conducted using STATA version 15.1 (StataCorp, College Station, TX, United States) to estimate standardized mean differences (SMDs) or odds ratios (ORs) with 95% confidence intervals (CIs). Random-effects models, predefined subgroup analyses, and risk-of-bias assessments were performed. RESULTS: A total of 24 randomized controlled trials involving 1,389 patients were included. Compared with control interventions, MSC therapy significantly improved pain (SMD = -1.31, 95% CI: -1.82 to -0.81), WOMAC scores (SMD = -0.78, 95% CI: -1.09 to -0.47), cartilage volume (SMD = 0.91, 95% CI: 0.28-1.53), and KOOS subscales. No significant difference was observed in adverse event rates (OR = 1.58, 95% CI: 0.72-3.47). Subgroup analyses suggested that treatment response may vary according to disease etiology, stem cell source, tissue origin, and delivery strategy; however, these findings were exploratory and should be interpreted cautiously because substantial residual heterogeneity remained across several outcomes. CONCLUSION: MSC therapy was associated with significant improvements in pain, physical function, cartilage volume, and KOOS outcomes without an apparent increase in adverse events. However, substantial heterogeneity across studies and the exploratory nature of subgroup analyses warrant cautious interpretation of these findings. Further large-scale randomized controlled trials with standardized treatment protocols and long-term follow-up are required to confirm the optimal therapeutic strategy and long-term efficacy and safety of MSC therapy.