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Umbilical cord mesenchymal stem cells transplantation in patients with systemic sclerosis: a 5-year follow-up study

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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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Authors and Affiliations

  1. Department of Rheumatology and Immunology, Nanjing Drum Tower Hospital, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, 210008, China

    Mihribangvl Alip, Dandan Wang, Shengnan Zhao, Siqi Li, Dongdong Zhang, Xiaoxiao Duan, Shiying Wang, Bingzhu Hua, Hong Wang, Huayong Zhang, Xuebing Feng & Lingyun Sun

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  1. Mihribangvl Alip
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  2. Dandan Wang
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  4. Siqi Li
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  5. Dongdong Zhang
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  7. Shiying Wang
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  12. Lingyun Sun
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Contributions

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.

Corresponding author

Correspondence to Lingyun Sun.

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Ethics approval

This study was approved by the local Ethics Committee (No. 2009004).

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Obtained.

Patient and public involvement

Patients or the public WERE NOT involved in the design, or conduct, or reporting, or dissemination plans of our research.

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None.

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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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