隨著早期診療、靶向藥物和免疫療法的普遍應用,大部分常見癌癥的5年生存率都有較明顯的提升。相比于常見癌癥,罕見癌癥由于其發(fā)病率較低,長期缺乏足夠的關注度。然而,基于龐大的人口總數(shù),罕見癌癥的發(fā)病數(shù)依然是不容忽視的,但有效治療手段的缺失使得罕見癌癥患者的預后不佳。罕見癌癥的研究難點之一在于癌種群體分散,樣本量較小,難以進行相關的臨床研究。而患者來源的異種移植模型可有效保存患者樣本,高度模擬原發(fā)腫瘤,是研究罕見癌癥的有利工具。就罕見癌癥的概念和人源腫瘤異種移植模型在不同類別的罕見癌癥如腺樣囊性癌、膽管癌、肛管癌、惡性間皮瘤、罕見婦科癌癥、睪丸癌、黑色素瘤、惡性胚胎腫瘤、脊索瘤、胃腸胰神經(jīng)內(nèi)分泌腫瘤、腎上腺皮質(zhì)癌、膠質(zhì)母細胞瘤、血液系統(tǒng)惡性腫瘤中的應用進展進行綜述,以期為探索罕見癌癥發(fā)病機制及開發(fā)更具有前景的抗罕見癌癥藥物提供參考。

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.

R979.1

上海市浦江人才計劃(D類)資助項目(2021PJD091)