小柯机器人

自体巨噬细胞疗法治疗肝硬化的安全性获得评估，这一成果由英国爱丁堡大学Stuart J. Forbes研究小组取得。该项研究成果在线发表在2019年10月7日的《自然—医学》上。

研究人员在9名肝硬化成人患者中进行了一项自体巨噬细胞疗法在人类中的第一阶段剂量递增试验，其终末期肝病模型（MELD）评分为10–16（ISRCTN 10368050）。三名一组的参与者接受了10⁷、10⁸或最多10⁹个细胞的单次外周输注。白细胞分离术和巨噬细胞输注耐受良好，无输血反应、剂量限制性毒性或巨噬细胞活化综合征。所有参与者均在一年后存活并且没有移植，只有一个临床事件记录在案，即发生最小程度的腹水。这获得了安全性和可行性的主要结果。这项研究为肝硬化和其他纤维化疾病的功效研究提供了相关信息和理论依据。

据介绍，目前迫切需要减少肝纤维化和刺激器官再生的疗法。

附：英文原文

Title: Safety profile of autologous macrophage therapy for liver cirrhosis

Author: Francesca Moroni, Benjamin J. Dwyer, Catriona Graham, Chloe Pass, Laura Bailey, Lisa Ritchie, Donna Mitchell, Alison Glover, Audrey Laurie, Stuart Doig, Emily Hargreaves, Alasdair R. Fraser, Marc L. Turner, John D. M. Campbell, Neil W. A. McGowan, Jacqueline Barry, Joanna K. Moore, Peter C. Hayes, Diana J. Leeming, Mette J. Nielsen, Kishwar Musa, Jonathan A. Fallowfield, Stuart J. Forbes

Issue&Volume: 2019-10-07

Abstract: Therapies to reduce liver fibrosis and stimulate organ regeneration are urgently needed. We conducted a first-in-human, phase 1 dose-escalation trial of autologous macrophage therapy in nine adults with cirrhosis and a Model for End-Stage Liver Disease (MELD) score of 1016 (ISRCTN 10368050). Groups of three participants received a single peripheral infusion of 107, 108 or up to 109 cells. Leukapheresis and macrophage infusion were well tolerated with no transfusion reactions, dose-limiting toxicities or macrophage activation syndrome. All participants were alive and transplant-free at one year, with only one clinical event recorded, the occurrence of minimal ascites. The primary outcomes of safety and feasibility were met. This study informs and provides a rationale for efficacy studies in cirrhosis and other fibrotic diseases. A first-in-human, phase 1 dose-escalation trial demonstrates the safety and feasibility of autologous macrophage therapy in adults with liver cirrhosis.

DOI: 10.1038/s41591-019-0599-8

Source:https://www.nature.com/articles/s41591-019-0599-8

Nature Medicine：《自然—医学》，创刊于1995年。隶属于施普林格·自然出版集团，最新IF：87.241
官方网址：https://www.nature.com/nm/
投稿链接：https://mts-nmed.nature.com/cgi-bin/main.plex