目的:大量数据表明,早发性胃肠道肿瘤的发病率上升,预后不良,但是关于胆道癌(BTC)的数据很少。我们的目的是分析临床病理,分子,早发性BTC患者的治疗特征和预后(EOBTC,诊断时年龄≤50岁),对老年人。
方法:我们分析了诊断为肝内胆管癌的患者,肝外胆管癌,和胆囊腺癌在2003年1月1日至2021年6月30日之间。描述各组的基线特征和治疗并进行比较。无进展生存,使用Kaplan-Meier方法估计每组的总生存期和无病生存期.
结果:总体而言,纳入1256例患者,188(15%)与EOTC。EOBTC患者的合并症较少(63.5%vs.84.5%,p<.0001),更高的肿瘤分期(cT3-4:50.0%vs.32.3%,p=.0162),双叶肝受累(47.8%vs.32.1%,p=.0002),和转移性疾病(67.6%vs.57.5%,p=.0097)与老年人相比。EOBTC患者接受二线治疗的频率更高(89.5%vs.81.0%非EOTC,p=.0224)。对于不可切除的BTC患者,中位总生存期为17.0vs.16.2个月(p=.0876),中位无进展生存期为5.8vs.6.0个月(p=.8293),在EOBTCvs.老了。在高级阶段,在EOBTC中发现了较少的可操作的改变(例如,IDH1突变[7.8%vs.16.6%];FGFR2融合[11.7%vs.8.9%];p=.029)。
结论:EOBTC患者在诊断时具有更晚期的疾病,在晚期接受更多治疗,但表现出相似的存活率。发现了富含FFRF2融合物的独特分子谱。
OBJECTIVE: Accumulating data has shown the rising incidence and poor prognosis of early-onset gastrointestinal cancers, but few data exist on biliary tract cancers (BTC). We aimed to analyse the clinico-pathological, molecular, therapeutic characteristics and prognosis of patients with early onset BTC (EOBTC, age ≤50 years at diagnosis), versus olders.
METHODS: We analysed patients diagnosed with intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, and gallbladder adenocarcinoma between 1 January 2003 and 30 June 2021. Baseline characteristics and treatment were described in each group and compared. Progression-free survival, overall survival and disease-free survival were estimated in each group using the Kaplan-Meier method.
RESULTS: Overall, 1256 patients were included, 188 (15%) with EOBTC. Patients with EOBTC demonstrated fewer comorbidities (63.5% vs. 84.5%, p < .0001), higher tumour stage (cT3-4: 50.0% vs. 32.3%, p = .0162), bilobar liver involvement (47.8% vs. 32.1%, p = .0002), and metastatic disease (67.6% vs. 57.5%, p = .0097) compared to older. Patients with EOBTC received second-line therapy more frequently (89.5% vs. 81.0% non-EOBTC, p = .0224). For unresectable patients with BTC, median overall survival was 17.0 vs. 16.2 months (p = .0876), and median progression-free survival was 5.8 vs. 6.0 months (p = .8293), in EOBTC vs. older. In advanced stages, fewer actionable alterations were found in EOBTC (e.g., IDH1 mutations [7.8% vs. 16.6%]; FGFR2-fusion [11.7% vs. 8.9%]; p = .029).
CONCLUSIONS: Patients with EOBTC have a more advanced disease at diagnosis, are treated more heavily at an advanced stage but show similar survival. A distinctive molecular profile enriched for FGRF2 fusions was found.