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支气管扩张症患者中 P63 肺祖细胞的自体移植:一项随机、单盲、对照试验。

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

机构信息

Department of Pulmonary and Critical Care Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China; Institute of Respiratory Diseases, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China; Shanghai Key Laboratory of Emergency Prevention, Diagnosis and Treatment of Respiratory Infectious Diseases, Shanghai 200025, China.

Institute for Regenerative Medicine, State Key Laboratory of Cardiology and Medical Innovation Center, Shanghai East Hospital, School of Medicine, Tongji University, Shanghai 200092, China.

出版信息

Cell Rep Med. 2024 Nov 19;5(11):101819. doi: 10.1016/j.xcrm.2024.101819.

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 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).

摘要

非囊性纤维化支气管扩张症是一种进展性呼吸系统疾病,治疗选择有限,因此需要探索再生疗法。本研究采用随机、单盲、对照、1/2 期临床试验,评估自体 P63 祖细胞移植的安全性和疗效。37 例患者接受支气管镜气道清除术(B-ACT)(n=19)或 B-ACT 联合 P63 祖细胞治疗(n=18)。结果显示,与对照组相比,细胞治疗组治疗后 24 周时 D 水平的变化明显更高(p 值=0.039)。此外,细胞治疗组患者在治疗后 4-12 周内肺损伤面积明显减少,SGRQ 评分改善,BSI 和 FACED 评分改善。转录组分析显示,P63 基因表达较高的祖细胞具有更好的治疗效果。这些发现表明,P63 祖细胞可能为支气管扩张症提供一种有前途的治疗方法。本研究在 ClinicalTrials.gov(NCT03655808)注册。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4ad1/11604513/4c38440f28c4/fx1.jpg

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