Wenyi Yuan, Mian Liu, Dapeng Yang, Yirui Shi, Zhikang Wang, Xuan Cao, Jun Liang, Linyu Geng, Huayong Zhang, Xuebing Feng, Ziyi Jin, Dandan Wang, Lingyun Sun
{"title":"Improvement in long-term survival with mesenchymal stem cell transplantation in systemic sclerosis patients: a propensity score-matched cohort study.","authors":"Wenyi Yuan, Mian Liu, Dapeng Yang, Yirui Shi, Zhikang Wang, Xuan Cao, Jun Liang, Linyu Geng, Huayong Zhang, Xuebing Feng, Ziyi Jin, Dandan Wang, Lingyun Sun","doi":"10.1186/s13287-025-04237-1","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>The intricate and varied clinical presentations of systemic sclerosis (SSc) pose significant challenges for treatment. While several studies have investigated the therapeutic potential of mesenchymal stem cell transplantation (MSCT), the clarity of its long-term outcomes for SSc patients is still lacking.</p><p><strong>Methods: </strong>Data on MSCT were extracted from the medical records of inpatients at Nanjing Drum Tower Hospital between January 2013 and December 2022. Additionally, Baseline characteristics and survival outcomes were ascertained from medical records and telephone follow-up. Propensity score matching (PSM) was employed to equalize the baseline characteristics of the patient groups, while survival analysis and multivariate Cox regression assessed the relationship between received MSCT and all-cause mortality and disease-specific survival rates in SSc patients.</p><p><strong>Results: </strong>Of the 333 hospitalized SSc patients, 113 patients underwent MSCT. The log-rank test revealed significantly higher survival rates in the MSCT group compared to the control group (10-year survival rate 89.4% vs. 73.4%, P = 0.002). In the PSM cohort, receiving MSCT significantly reduced mortality (10-year survival 88.0% vs. 79.9%, P = 0.032). Multivariate Cox regression analysis indicated that MSCT was linked to a reduced mortality risk during the follow-up period (HR 0.38, 95% CI 0.19-0.75, P = 0.005). This finding was further confirmed in the matched cohort (HR 0.38, 95% CI 0.18-0.82, P = 0.014). Subgroup analyses revealed that treated with MSCT was correlated with reduced mortality in patients of various demographics, including younger age at diagnosis (≤ 47 years), female, diffuse cutaneous systemic sclerosis (dcSSc) subtype, concurrent arthritis, pulmonary arterial hypertension (PAH), and interstitial lung disease (ILD).</p><p><strong>Conclusion: </strong>MSCT significantly enhances the survival rate of patients with SSc, with outcomes related to the age at diagnosis. MSCT is particularly indicated for patients with comorbid conditions, including PAH, ILD, digital ulcers, and arthritis, as well as those with severe disease presentations associated with the dcSSc subtype.</p>","PeriodicalId":21876,"journal":{"name":"Stem Cell Research & Therapy","volume":"16 1","pages":"128"},"PeriodicalIF":7.1000,"publicationDate":"2025-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11892273/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem Cell Research & Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s13287-025-04237-1","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CELL & TISSUE ENGINEERING","Score":null,"Total":0}
引用次数: 0
Abstract
Background: The intricate and varied clinical presentations of systemic sclerosis (SSc) pose significant challenges for treatment. While several studies have investigated the therapeutic potential of mesenchymal stem cell transplantation (MSCT), the clarity of its long-term outcomes for SSc patients is still lacking.
Methods: Data on MSCT were extracted from the medical records of inpatients at Nanjing Drum Tower Hospital between January 2013 and December 2022. Additionally, Baseline characteristics and survival outcomes were ascertained from medical records and telephone follow-up. Propensity score matching (PSM) was employed to equalize the baseline characteristics of the patient groups, while survival analysis and multivariate Cox regression assessed the relationship between received MSCT and all-cause mortality and disease-specific survival rates in SSc patients.
Results: Of the 333 hospitalized SSc patients, 113 patients underwent MSCT. The log-rank test revealed significantly higher survival rates in the MSCT group compared to the control group (10-year survival rate 89.4% vs. 73.4%, P = 0.002). In the PSM cohort, receiving MSCT significantly reduced mortality (10-year survival 88.0% vs. 79.9%, P = 0.032). Multivariate Cox regression analysis indicated that MSCT was linked to a reduced mortality risk during the follow-up period (HR 0.38, 95% CI 0.19-0.75, P = 0.005). This finding was further confirmed in the matched cohort (HR 0.38, 95% CI 0.18-0.82, P = 0.014). Subgroup analyses revealed that treated with MSCT was correlated with reduced mortality in patients of various demographics, including younger age at diagnosis (≤ 47 years), female, diffuse cutaneous systemic sclerosis (dcSSc) subtype, concurrent arthritis, pulmonary arterial hypertension (PAH), and interstitial lung disease (ILD).
Conclusion: MSCT significantly enhances the survival rate of patients with SSc, with outcomes related to the age at diagnosis. MSCT is particularly indicated for patients with comorbid conditions, including PAH, ILD, digital ulcers, and arthritis, as well as those with severe disease presentations associated with the dcSSc subtype.
背景:系统性硬化症(SSc)复杂多样的临床表现给治疗带来了重大挑战。虽然有几项研究调查了间充质干细胞移植(MSCT)的治疗潜力,但其对SSc患者的长期结果的明确性仍然缺乏。方法:从2013年1月至2022年12月南京鼓楼医院住院患者病历中提取MSCT数据。此外,通过医疗记录和电话随访确定基线特征和生存结果。采用倾向评分匹配(PSM)来平衡患者组的基线特征,而生存分析和多变量Cox回归评估接受MSCT与SSc患者全因死亡率和疾病特异性生存率之间的关系。结果:333例SSc住院患者中,113例接受了MSCT。log-rank检验显示,MSCT组的生存率显著高于对照组(10年生存率89.4% vs. 73.4%, P = 0.002)。在PSM队列中,接受MSCT可显著降低死亡率(10年生存率为88.0%对79.9%,P = 0.032)。多因素Cox回归分析显示,MSCT与随访期间死亡风险降低有关(HR 0.38, 95% CI 0.19-0.75, P = 0.005)。这一发现在匹配队列中得到进一步证实(HR 0.38, 95% CI 0.18-0.82, P = 0.014)。亚组分析显示,MSCT治疗与各种人口统计学患者的死亡率降低相关,包括诊断时年龄较小(≤47岁)、女性、弥漫性皮肤系统性硬化症(dcSSc)亚型、并发关节炎、肺动脉高压(PAH)和间质性肺疾病(ILD)。结论:MSCT可显著提高SSc患者的生存率,其预后与诊断年龄有关。MSCT特别适用于有合并症的患者,包括PAH、ILD、数字溃疡和关节炎,以及与dcSSc亚型相关的严重疾病。
期刊介绍:
Stem Cell Research & Therapy serves as a leading platform for translational research in stem cell therapies. This international, peer-reviewed journal publishes high-quality open-access research articles, with a focus on basic, translational, and clinical research in stem cell therapeutics and regenerative therapies. Coverage includes animal models and clinical trials. Additionally, the journal offers reviews, viewpoints, commentaries, and reports.