人源肿瘤异种移植模型在罕见癌症中的应用进展

Application of patient-derived xenografts model in rare cancers

随着早期诊疗、靶向药物和免疫疗法的普遍应用,大部分常见癌症的5年生存率都有较明显的提升。相比于常见癌症,罕见癌症由于其发病率较低,长期缺乏足够的关注度。然而,基于庞大的人口总数,罕见癌症的发病数依然是不容忽视的,但有效治疗手段的缺失使得罕见癌症患者的预后不佳。罕见癌症的研究难点之一在于癌种群体分散,样本量较小,难以进行相关的临床研究。而患者来源的异种移植模型可有效保存患者样本,高度模拟原发肿瘤,是研究罕见癌症的有利工具。就罕见癌症的概念和人源肿瘤异种移植模型在不同类别的罕见癌症如腺样囊性癌、胆管癌、肛管癌、恶性间皮瘤、罕见妇科癌症、睾丸癌、黑色素瘤、恶性胚胎肿瘤、脊索瘤、胃肠胰神经内分泌肿瘤、肾上腺皮质癌、胶质母细胞瘤、血液系统恶性肿瘤中的应用进展进行综述,以期为探索罕见癌症发病机制及开发更具有前景的抗罕见癌症药物提供参考。

With the prevalence of early diagnosis and treatment,targeted drugs and immunotherapy,the 5-year survival rate of most common cancers has improved more significantly.Compared with common cancers,rare cancers have long lacked sufficient attention due to their lower incidence.However,based on the large population,the incidence number of rare cancers is still not negligible,but the lack of effective treatments makes the prognosis of patients with rare cancers poor.One of the difficulties in the research of rare cancers is the dispersed cancer population and small sample size,which makes it difficult to conduct relevant clinical studies.Patient-derived xenograft models,on the other hand,can effectively preserve patient samples and highly mimic primary tumors,making them a favorable tool for studying rare cancers.In this paper,we review the concept of rare cancers and the progress of the application of patient-derived xenograft models in different categories of rare cancers,such as adenoid cystic carcinoma,cholangiocarcinoma,squamous cell carcinoma of the anal canal,malignant mesothelioma,rare gynecological cancers,testicular cancer,melanoma,malignant embryonal tumors,chordoma,gastroenteropancreatic neuroendocrine neoplasms,adrenocortical carcinoma,glioblastoma,and hematologic malignancy,with the aim of providing an opportunity to explore the pathogenesis of rare cancers and to develop more promising and effective models for the study of rare cancers.We hope to provide reference for exploring the pathogenesis of rare cancers and developing more promising anti-rare cancer drugs.