Autologous transplantation of P63⁺ lung progenitor cells in patients with bronchiectasis: A randomized, single-blind, controlled trial.

IF 11.7 1区医学 Q1 CELL BIOLOGY

Cell Reports Medicine Pub Date : 2024-11-19 DOI:10.1016/j.xcrm.2024.101819

Jiayang Yan, Weipan Zhang, Yun Feng, Xuefei Liu, Lingyun Niu, Yi Guo, Ling Zhou, Mengmeng Shi, Caixia Di, Qiurui Zhang, Xiaofei Wang, Jianping Zhou, Ranran Dai, Lei Ni, Zhiyao Bao, Tianli Yan, Yun Hu, Ping Wang, Ting Zhang, Min Zhou, Wei Zuo, Jieming Qu

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引用次数: 0

Abstract

Non-cystic fibrosis bronchiectasis is a progressive respiratory disease with limited treatment options, prompting the exploration of regenerative therapies. This study investigates the safety and efficacy of autologous P63⁺ progenitor cell transplantation in a randomized, single-blind, controlled, phase 1/2 trial. Thirty-seven patients receive bronchoscopic airway clearance (B-ACT) (n = 19) or B-ACT plus P63⁺ progenitor cells (n = 18). Results show that compared to the control group, the change in D_LCO levels from baseline to 24 weeks post therapy is significantly higher in the cell treatment group (p value = 0.039). Furthermore, the patients in the cell treatment group demonstrate significantly reduced lung damaged area, improved SGRQ score, and ameliorated BSI and FACED scores within 4-12 weeks post therapy. Transcriptomic analysis reveals that progenitor cells with higher expression of P63 gene have better therapeutic efficacy. These findings suggest that P63⁺ progenitor cells may offer a promising therapeutic approach for bronchiectasis. This study was registered at ClinicalTrials.gov(NCT03655808).

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支气管扩张症患者的 P63+ 肺祖细胞自体移植：随机、单盲、对照试验。

非囊性纤维化支气管扩张症是一种进展性呼吸系统疾病，治疗方案有限，因此人们开始探索再生疗法。本研究通过一项随机、单盲、对照、1/2 期试验，探讨了自体 P63+祖细胞移植的安全性和有效性。37名患者接受支气管镜气道清除术（B-ACT）（n = 19）或B-ACT加P63+祖细胞（n = 18）。结果显示，与对照组相比，细胞治疗组的 DLCO 水平从基线到治疗后 24 周的变化明显更高（P 值 = 0.039）。此外，在治疗后 4-12 周内，细胞治疗组患者的肺损伤面积明显缩小，SGRQ 评分提高，BSI 和 FACED 评分改善。转录组分析表明，P63 基因表达较高的祖细胞具有更好的疗效。这些研究结果表明，P63+祖细胞可为支气管扩张症提供一种前景广阔的治疗方法。本研究已在ClinicalTrials.gov（NCT03655808）上注册。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Cell Reports Medicine Biochemistry, Genetics and Molecular Biology-Biochemistry, Genetics and Molecular Biology (all)

CiteScore

15.00

自引率

1.40%

发文量

231

审稿时长

40 days

期刊介绍： Cell Reports Medicine is an esteemed open-access journal by Cell Press that publishes groundbreaking research in translational and clinical biomedical sciences, influencing human health and medicine. Our journal ensures wide visibility and accessibility, reaching scientists and clinicians across various medical disciplines. We publish original research that spans from intriguing human biology concepts to all aspects of clinical work. We encourage submissions that introduce innovative ideas, forging new paths in clinical research and practice. We also welcome studies that provide vital information, enhancing our understanding of current standards of care in diagnosis, treatment, and prognosis. This encompasses translational studies, clinical trials (including long-term follow-ups), genomics, biomarker discovery, and technological advancements that contribute to diagnostics, treatment, and healthcare. Additionally, studies based on vertebrate model organisms are within the scope of the journal, as long as they directly relate to human health and disease.