Real-World Data as External Control in Assessing the Efficacy of Allogeneic Adipose-Derived Stem Cells Therapy for Advanced Chronic Kidney Disease

真实世界数据作为外部对照,用于评估异体脂肪干细胞疗法治疗晚期慢性肾病的疗效

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Abstract

Chronic kidney disease is a progressive condition with limited therapeutic options in its advanced stages. Adipose-derived stem cell therapy has shown potential in preclinical studies for renal repair. This study evaluated the short-term stability of renal function in patients with moderate to severe chronic kidney disease who received adipose-derived stem cell therapy, using a matched control group derived from real-world clinical data for comparison. A total of 34 treated patients were matched in a one-to-five ratio with 170 control patients based on key clinical characteristics. The primary outcomes included the mean percentage change in estimated glomerular filtration rate and the incidence of renal function decline exceeding defined thresholds. To enhance the robustness of treatment effect estimation, real-world data were utilized to construct an external control group that closely resembled the clinical trial population. This approach allowed indirect treatment comparisons and strengthened the internal validity of findings in the absence of randomization. Results demonstrated that the treated group exhibited a more stable renal function trajectory and a significantly lower risk of deterioration compared to the control group, particularly in patients with more advanced disease. Among dose groups, the low-dose group showed the greatest stability in renal function. These findings support the feasibility of using real-world data to construct external comparators and suggest that stem cell therapy may offer a short-term stabilizing effect on renal function. Further research is needed to validate these findings and explore their long-term clinical implications. Trial Registration: ClinicalTrials.gov identifier: NCT02933827 (registered October 13, 2016. https://clinicaltrials.gov/study/NCT02933827).

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