Abstract
Objective
To assess the long-term safety and efficacy of umbilical cord mesenchymal stem cells transplantation (UMSCT) in patients with systemic sclerosis (SSc).
Methods
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.
Results
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.
Conclusion
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.
Clinical Trial Registration
ClinicalTrials.gov Identifier: NCT00962923.
Key Point • UMSCT is safe and effective for SSc patients. |
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Data availability
The raw/processed data required to reproduce these findings cannot be shared at this time as the data also forms part of an ongoing study.
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Acknowledgements
This work was supported by the National Key R&D Program of China (2020YFA0710800), the Jiangsu Provincial Key Research and Development Program (BE2020621), Jiangsu Provincial Health Department Foundation (H2023087) and National Natural Science Foundation of China (81401353, 81871282). We thank all the participants who were involved in this study..
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LS, DW contributed to study design. Alip, DW and SZ contributed equally to this work and share first authorship. SL, DW, Alip, SZ, DW, XD, DZ, SL, SW, BH, HW, HZ and XF contributed to data acquisition, data interpretation, and manuscript writing. LS, DW and Mihribangvl analyzed the data and revised the manuscript. All authors read and approved the final manuscript. All authors were involved in drafting and revising the article. There are no competing interests for any author.
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Key message
This retrospective longitudinal single-arm study completed a 5-year follow-up for 41 SSc patients who received umbilical cord mesenchymal stem cells transplantation (UMSCT), and the 5-year overall survival rate is 92.7% (38/41).The mean mRSS showed a decrease six months after the initiation of therapy that combined patients' original treatment with UMSCT, and this reduction was maintained throughout the 5-year follow-up period. Notably, no adverse events related to UMSCT were observed.
Mihribangvl Alip, Dandan Wang, and Shengnan Zhao are co-senior authors.
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Alip, M., Wang, D., Zhao, S. et al. Umbilical cord mesenchymal stem cells transplantation in patients with systemic sclerosis: a 5-year follow-up study. Clin Rheumatol 43, 1073–1082 (2024). https://doi.org/10.1007/s10067-024-06865-z
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DOI: https://doi.org/10.1007/s10067-024-06865-z