验证一种简单的评分系统，以预测索拉非尼对肝细胞癌患者的疗效。
Validation of a Simple Scoring System to Predict Sorafenib Effectiveness in Patients with Hepatocellular Carcinoma.
机构信息
Department of Transplantation - Liver Unit, Cardarelli Hospital, via A. Cardarelli 9, 80131, Naples, Italy.
Department of Medical Oncology, Istituto Scientifico Romagnolo per lo Studio e Cura dei Tumori (IRST) IRCCS, Meldola, Italy.
出版信息
Target Oncol. 2017 Dec;12(6):795-803. doi: 10.1007/s11523-017-0522-5.
BACKGROUND
Sorafenib is recommended for the treatment of advanced-stage hepatocellular carcinoma (HCC). Nonetheless, it is expensive, effective in few patients, and may cause significant adverse effects. Therefore, accurate selection of patients is needed. In a previous study, we constructed a simple scoring system to predict patients' outcomes based on the occurrence of sorafenib adverse effects.
OBJECTIVE
The present study aimed to validate this scoring system in a real-life cohort of HCC patients.
PATIENTS AND METHODS
Clinical records of 279 outpatients treated with sorafenib in eight Italian centers were retrospectively analyzed. Adverse effects considered to calculate the score were skin toxicity, diarrhea, and arterial hypertension, occurring during the first month of therapy. For each adverse effect, 1 point was assigned if present; and 0 points if absent (resulting in a total score between 0 and 3).
RESULTS
Median overall survival (OS) was 10.8 months and median time to progression (TTP) was 5.1 months. At multivariate analysis, performance status, α-fetoprotein (AFP), and Child-Pugh score were independently associated with TTP and OS. A progressive increase of OS and TTP was observed in patients with scores from 0 to 3 (p < 0.001). Six-, 12-, and 24-month survival probabilities were 55.1, 24.5, and 7.9% in score 0 patients, and 100, 80.9, and 46.2% in score 3 patients, respectively. Complete response was observed in one patient (0.4%), partial responses in 41 (15.2%), and stable disease in 117 (43.5%). The disease control rate in patients with scores of 0, 1, 2, and 3 was 34.3, 51.6, 80.9, and 96.3%, respectively (p < 0.001). Complete or partial responses were not observed in score 0 patients.
CONCLUSIONS
We have validated a useful scoring system to predict outcomes in sorafenib-treated HCC patients. This score is easy to calculate and suitable for implementation in daily clinical practice.
背景
索拉非尼被推荐用于治疗晚期肝细胞癌（HCC）。然而，它的价格昂贵，对少数患者有效，并且可能引起严重的不良反应。因此，需要准确选择患者。在之前的一项研究中，我们构建了一个简单的评分系统，根据索拉非尼不良反应的发生来预测患者的预后。
目的
本研究旨在验证该评分系统在 HCC 患者的真实队列中的有效性。
患者和方法
回顾性分析了 8 家意大利中心的 279 名接受索拉非尼治疗的门诊患者的临床记录。在治疗的第一个月内发生的皮肤毒性、腹泻和动脉高血压等不良反应被认为可用于计算评分。如果存在不良反应，则计 1 分；如果不存在，则计 0 分（总分为 0 至 3 分）。
结果
中位总生存期（OS）为 10.8 个月，中位无进展生存期（TTP）为 5.1 个月。多变量分析显示，体能状态、甲胎蛋白（AFP）和 Child-Pugh 评分与 TTP 和 OS 独立相关。在评分从 0 到 3 的患者中，OS 和 TTP 逐渐增加（p<0.001）。评分 0 患者的 6、12 和 24 个月生存率分别为 55.1%、24.5%和 7.9%，评分 3 患者的 6、12 和 24 个月生存率分别为 100%、80.9%和 46.2%。一名患者（0.4%）观察到完全缓解，41 名患者（15.2%）观察到部分缓解，117 名患者（43.5%）观察到稳定疾病。评分 0、1、2 和 3 的患者疾病控制率分别为 34.3%、51.6%、80.9%和 96.3%（p<0.001）。评分 0 的患者未观察到完全或部分缓解。
结论
我们验证了一种有用的评分系统，可用于预测索拉非尼治疗的 HCC 患者的预后。该评分易于计算，适用于日常临床实践。
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