Department of Rheumatology and Immunology, Nanjing Drum Tower Hospital, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, 210008, China.
Clin Rheumatol. 2024 Mar;43(3):1073-1082. doi: 10.1007/s10067-024-06865-z. Epub 2024 Jan 11.
To assess the long-term safety and efficacy of umbilical cord mesenchymal stem cells transplantation (UMSCT) in patients with systemic sclerosis (SSc).
Forty-one patients with moderate to severe SSc underwent UMSCT at the Affiliated Drum Tower Hospital of Nanjing University Medical School from 2009 to 2017. In this study, we conducted a longitudinal and retrospective analysis and compared the clinical and laboratory manifestations before and after UMSCT. The main outcome of the study was overall survival. We evaluated changes in the modified Rodnan Skin Score (mRSS), as well as the changes in the pulmonary examination by using high-resolution computed tomography (HRCT) and ultrasound cardiogram (UCG). Additionally, we assessed the Health Assessment Questionnaire-Disability Index (HAQ-DI) and the severity of peripheral vascular involvement during the first year after treatment.
The overall 5-year survival rate was 92.7% (38 out of 41 patients). Following UMSCT, the mean mRSS significantly decreased from 18.68 (SD = 7.26, n = 41) at baseline to 13.95 (SD = 8.49, n = 41), 13.29 (SD = 7.67, n = 38), and 12.39 (SD = 8.49, n = 38) at 1, 3, and 5 years, respectively. Improvement or stability in HRCT images was observed in 72.0% of interstitial lung disease (ILD) patients. Pulmonary arterial hypertension (PAH) remained stable in 5 out of 8 patients at the 5-year follow-up. No adverse events related to UMSCT were observed in any of the patients during the follow-up period.
UMSCT may provide a safe and feasible treatment option for patients with moderate to severe SSc based on long-term follow-up data. The randomized controlled study will further confirm the clinical efficacy of UMSCT in SSc.
ClinicalTrials.gov Identifier: NCT00962923. Key Point • UMSCT is safe and effective for SSc patients.
评估脐带间充质干细胞移植（UMSCT）治疗系统性硬化症（SSc）患者的长期安全性和疗效。
2009 年至 2017 年，南京医科大学附属鼓楼医院共收治 41 例中重度 SSc 患者行 UMSCT。本研究进行了纵向回顾性分析，比较 UMSCT 前后的临床和实验室表现。主要研究终点为总生存率。我们评估了改良 Rodnan 皮肤评分（mRSS）的变化，以及高分辨率计算机断层扫描（HRCT）和超声心动图（UCG）评估的肺检查变化。此外，我们还评估了治疗后第 1 年的健康评估问卷残疾指数（HAQ-DI）和周围血管受累的严重程度。
41 例患者的 5 年总生存率为 92.7%（38/41）。UMSCT 后，平均 mRSS 从基线时的 18.68（SD=7.26，n=41）显著下降至 13.95（SD=8.49，n=41）、13.29（SD=7.67，n=38）和 12.39（SD=8.49，n=38），分别为 1 年、3 年和 5 年。72.0%的间质性肺病（ILD）患者的 HRCT 图像得到改善或稳定。8 例肺动脉高压（PAH）患者中有 5 例在 5 年随访时保持稳定。在随访期间，未观察到任何与 UMSCT 相关的不良事件。
根据长期随访数据，UMSCT 可能为中重度 SSc 患者提供一种安全可行的治疗选择。随机对照研究将进一步证实 UMSCT 在 SSc 中的临床疗效。
ClinicalTrials.gov 标识符：NCT00962923。关键点• UMSCT 对 SSc 患者安全有效。
