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原发性骨髓纤维化的实验模型体系

韩旭^*, 刘静

中南大学生命科学学院，中南大学湘雅二医院血液科，长沙 410083

摘要

骨髓增殖性肿瘤是一类起源于造血干细胞的克隆增殖性疾病，具有转化为急性髓系白血病的风险；原发性骨髓纤维化(primary myelofibrosis, PMF)是骨髓增殖性肿瘤的一个亚型。Janus 激酶2 (Janus kinase 2, JAK2)、钙网蛋白(calreticulin,CALR)、促血小板生成素受体(thrombopoietin receptor, MPL)三个基因的突变是PMF患者主要的致病因素。目前的治疗策略(包括JAK2 抑制剂)仅对部分PMF患者有效，亟需开发新的干预手段。PMF的实验模型(包括细胞模型、动物模型和体外类器官模型等)在探索发病机理及筛选潜在治疗药物方面发挥重要作用。本文全面总结了这些实验模型体系，比较不同模型的优缺点，并讨论了当前研究的局限性及对未来方向的展望，以期为针对PMF治疗策略的开发提供帮助。

关键词： 原发性骨髓纤维化; 实验模型; 动物模型; 发病机制; 治疗策略

Experimental model systems of primary myelofibrosis

HAN Xu^*, LIU Jing

School of Life Sciences, Department of Hematology of the Second Xiangya Hospital, Central South University, Changsha 410083, China

Abstract

Myeloproliferative neoplasms are a group of clonal proliferative diseases originating from hematopoietic stem cells, with a risk of transformation to acute myeloid leukemia. Primary myelofibrosis (PMF) is a subtype of myeloproliferative neoplasms. Mutations in Janus kinase 2 (JAK2), calreticulin (CALR), and thrombopoietin receptor (MPL) genes are the main pathogenic factors in PMF patients. Current therapeutic strategies, including JAK2 inhibitors, are only effective in a subset of PMF patients, highlighting an urgent need for the development of novel interventions. Experimental models of PMF, including cellular models, animal models, and in vitro organoid models, play crucial roles in exploring pathogenesis and screening potential therapeutic agents. This review comprehensively summarizes these experimental model systems, compares the advantages and disadvantages of different models, and discusses the limitations of current research and prospects for future directions, in order to provide assistance for the development of therapeutic strategies for PMF.

Key words: primary myelofibrosis; experimental model; animal model; pathogenesis; therapeutic strategy

收稿日期：　　录用日期：

通讯作者：韩旭　　E-mail:

DOI: 10.13294/j.aps.2026.0084

引用本文：

韩旭, 刘静. 原发性骨髓纤维化的实验模型体系[J]. 生理学报 2026; 78 (4): 877-888.

HAN Xu, LIU Jing. Experimental model systems of primary myelofibrosis. Acta Physiol Sin 2026; 78 (4): 877-888 (in Chinese with English abstract).