Schott Anne F, Welch John J, Verschraegen Claire F, Kurzrock Razelle
Department of Medicine, Division of Hematology-Oncology, University of Michigan, Ann Arbor, MI.
National Cancer Institute Center for Global Health , Bethesda, MD.
Semin Oncol. 2015 Oct;42(5):731-9. doi: 10.1053/j.seminoncol.2015.07.010. Epub 2015 Jul 10.
Rare cancers account for 27% of neoplasms diagnosed each year, and 25% of cancer-related deaths in the United States. However, rare cancers show some of the highest response rates to targeted therapies, probably due to identification of oncogenic drivers with little interpatient variability. Although the low incidence of rare cancers makes large-scale randomized trials involving single histologies difficult to perform, drugs have been successfully developed in rare cancers using clinical trial designs that combine microscopic histologies. Such trials are being pursued within the National Clinical Trials Network (NCTN), which possesses unique qualifications to perform widespread molecular screening of tumors for patient enrollment onto therapeutic clinical trials. When larger clinical trials are needed to determine optimum treatment strategies in rare cancers, the NCTN's broad reach in North America and internationally, and their ability to partner with both United States-based and international research organizations, can make these challenging studies feasible.
罕见癌症占美国每年确诊肿瘤的27%，以及癌症相关死亡的25%。然而，罕见癌症对靶向治疗的反应率在某些情况下是最高的，这可能是由于识别出了在患者间变异很小的致癌驱动因素。尽管罕见癌症的低发病率使得涉及单一组织学类型的大规模随机试验难以开展，但利用结合微观组织学的临床试验设计，已成功开发出针对罕见癌症的药物。此类试验正在国家临床试验网络（NCTN）中进行，该网络具备独特条件，能够对肿瘤进行广泛的分子筛查，以便将患者纳入治疗性临床试验。当需要更大规模的临床试验来确定罕见癌症的最佳治疗策略时，NCTN在北美及国际上的广泛影响力，以及它与美国本土和国际研究机构合作的能力，能够使这些具有挑战性的研究变得可行。
