Shuhang Wang1, Peiwen Ma1, Ning Jiang1, Yale Jiang1, Yue Yu1, Yuan Fang1, Huilei Miao1, Huiyao Huang2, Qiyu Tang1, Dandan Cui1, Hong Fang1, Huishan Zhang2, Qi Fan1, Yuning Wang1, Gang Liu3, Zicheng Yu4, Qi Lei1, Ning Li1()
1. Clinical Trial Center, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100021, China 2. Phase I Clinical Trial Center, Fujian Medical University Cancer Hospital/Fujian Cancer Hospital, Fuzhou 350014, China 3. Key Laboratory of Molecular Epigenetics of the Ministry of Education, Northeast Normal University, Changchun 130024, China 4. GenePlus-Shenzhen, Shenzhen 518118, China
Advances in novel drugs, therapies, and genetic techniques have revolutionized the diagnosis and treatment of cancers, substantially improving cancer patients’ prognosis. Although rare tumors account for a non-negligible number, the practice of precision medicine and development of novel therapies are largely hampered by many obstacles. Their low incidence and drastic regional disparities result in the difficulty of informative evidence-based diagnosis and subtyping. Sample exhaustion due to difficulty in diagnosis also leads to a lack of recommended therapeutic strategies in clinical guidelines, insufficient biomarkers for prognosis/efficacy, and inability to identify potential novel therapies in clinical trials. Herein, by reviewing the epidemiological data of Chinese solid tumors and publications defining rare tumors in other areas, we proposed a definition of rare tumor in China, including 515 tumor types with incidences of less than 2.5/100 000 per year. We also summarized the current diagnosis process, treatment recommendations, and global developmental progress of targeted drugs and immunotherapy agents on the status quo. Lastly, we pinpointed the current recommendation chance for patients with rare tumors to be involved in a clinical trial by NCCN. With this informative report, we aimed to raise awareness on the importance of rare tumor investigations and guarantee a bright future for rare tumor patients.
Systemic therapy for alveolar soft part sarcoma, solitary fibrous tumor
PazoPanib, anlotinib
Advanced/metastatic soft tissue sarcoma: first line (patients ineligible for IV systemic therapy); subsequent lines
Tab.2
Fig.5
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