Fu Shengyuan, Guo Yao, Peng Zhiyong, Zhang Dengyang, Chang Zhiguang, Xiao Yan, Zhang Qi, Yu Liuting, Chen Chun, Chen Yun, Zhao Yuming
Edmond H. Fischer Translational Medical Research Laboratory, Scientific Research Center, The Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, 518107, Guangdong, China.
Nanfang-Chunfu Children's Institute of Hematology, Taixin Hospital, Dongguan, Guangdong, China.
Med Oncol. 2024 Dec 9;42(1):25. doi: 10.1007/s12032-024-02549-5.
Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative neoplasm occurring in infants and young children. JMML has been shown to be resistant to all conventional cytotoxic chemotherapy drugs, and current curative therapies still rely on hematopoietic stem cell transplantation, which carries a high risk of relapse post-transplantation. This underscores the urgent need for novel treatment strategies. However, the rarity of JMML poses a major limitation for research, as it is difficult to collect substantial primary research material. To gain a deeper insight into the underlying biological mechanisms of JMML, researchers are continuously improving and developing preclinical research models to better emulate the disease. Therefore, this review aims to delineate the various experimental models currently employed in JMML, including patient-derived cell-based models, cell models, and animal models. We will discuss the characterization of these models in the context of JMML, hoping to provide a valuable reference for researchers in this field.
青少年粒单核细胞白血病（JMML）是一种发生于婴幼儿的罕见骨髓增殖性肿瘤。已证明JMML对所有传统细胞毒性化疗药物均耐药，目前的治愈性疗法仍依赖造血干细胞移植，而移植后复发风险很高。这凸显了对新型治疗策略的迫切需求。然而，JMML的罕见性对研究构成了重大限制，因为难以收集大量的原发性研究材料。为了更深入地了解JMML的潜在生物学机制，研究人员不断改进和开发临床前研究模型，以更好地模拟该疾病。因此，本综述旨在描述目前在JMML中使用的各种实验模型，包括患者来源的细胞模型、细胞模型和动物模型。我们将在JMML的背景下讨论这些模型的特征，希望为该领域的研究人员提供有价值的参考。
