■纤维板层肝细胞癌(FLHCC)是一种罕见的原发性肝脏恶性肿瘤,通常在晚期诊断。虽然关于特定药物疗效的数据有限,我们旨在报告接受系统治疗的患者的结局,并探讨预后因素.
■回顾了2010年至2022年期间接受治疗的患者的病历。在多学科评估后定义治疗。描述性统计用于基线人口统计学。使用Kaplan-Meier方法估计事件发生时间结果,通过对数排序进行比较,并通过回归模型进行调整。放射学特征(包括大小、形状,和纹理)的原发性病变被提取和维度减少。进行了无监督高斯混合模型(GMM)聚类,并在集群之间比较生存率。
■我们确定了23名患者:12名男性,平均年龄为23.6岁。诊断时,82.6%有转移,最常见的是肺部(39.1%),淋巴结(39.1%),和腹膜(21.7%)。患者接受了三行(1-8)治疗的中位数,包括不同的方案。索拉非尼(39.1%),卡培他滨(30.4%),卡培他滨/干扰素(13%)是最常用的一线方案.中位失败时间为3.8个月(95%CI:3.2-8.7)。卡培他滨+干扰素(42.1%)和铂类联合(39.1%)是最常用的二线方案,失效时间为3.5个月(95%CI:1.5-11.6)。中位总生存期为26.7个月(95%CI:15.1-40.4)。高基线中性粒细胞与淋巴细胞比率(NLR)与较差的生存率相关(p=0.02)。放射学特征确定了三个簇,一组(n=6)具有更好的生存期(40.4vs22.6个月,p=0.039)。动脉期的肿瘤球形是与更好预后相关的最相关特征(准确性=0.93)。
■与传统HCC相比,FLHCC具有独特的功能,包括年轻的发病,性别平衡,没有肝病.系统疗法可以提供令人鼓舞的生存,但缺乏一致性排除了定义一个更好的方案。建议影像组学和NLR与预后相关,并需要进一步验证。
UNASSIGNED: Fibrolamellar hepatocellular carcinoma (FLHCC) is a rare primary liver malignancy often diagnosed at advanced stages. While there are limited data on the efficacy of specific agents, we aim to report outcomes of patients treated with systemic therapies and explore prognostic factors.
UNASSIGNED: Medical records of patients treated between 2010 and 2022 were reviewed. Treatments were defined after multidisciplinary assessment. Descriptive statistics were used for baseline demographics. Time-to-event outcomes were estimated using the Kaplan-Meier method, compared by log-rank and adjusted by a regression model. Radiomic features (including size, shape, and texture) of the primary lesion were extracted and dimensionality reduced. An unsupervised Gaussian Mixture Model (GMM) clustering was performed, and survival was compared between clusters.
UNASSIGNED: We identified 23 patients: 12 males, with a median age of 23.6 years. At diagnosis, 82.6% had metastases, most frequently to the lungs (39.1%), lymph nodes (39.1%), and peritoneum (21.7%). Patients received a median of three lines (1-8) of treatment, including different regimens. Sorafenib (39.1%), capecitabine (30.4%), and capecitabine/interferon (13%) were the most used first-line regimens. The median time-to-failure was 3.8 months (95% CI: 3.2-8.7). Capecitabine + interferon (42.1%) and platinum combinations (39.1%) were the most used second-line regimens, with a time-to-failure of 3.5 months (95% CI: 1.5-11.6). Median overall survival was 26.7 months (95% CI: 15.1-40.4). A high baseline neutrophil-to-lymphocyte ratio (NLR) was associated with worse survival (p=0.02). Radiomic features identified three clusters, with one cluster (n=6) having better survival (40.4 vs 22.6 months, p=0.039). Tumor sphericity in the arterial phase was the most relevant characteristic associated with a better prognosis (accuracy=0.93).
UNASSIGNED: FLHCC has unique features compared to conventional HCC, including young onset, gender balance, and absence of hepatopathy. Systemic therapies can provide encouraging survival, but lack of uniformity precludes defining a preferable regimen. Radiomics and NLR were suggested to correlate with prognosis and warrant further validation.