Laber Damian A, Mushtaq Mian
Division of Hematology and Medical Oncology, University of Louisville, J.G. Brown Cancer Center, Louisville, KY 40202, USA.
Clin Genitourin Cancer. 2009 Jan;7(1):34-8. doi: 10.3816/CGC.2009.n.006.
In 2005, a phase III trial demonstrated a significant increase in progression-free survival in patients with renal cell cancer (RCC) treated with sorafenib versus placebo. While awaiting the full review by the US Federal Drug Administration, we initiated a treatment protocol as a mechanism for providing sorafenib to patients with advanced RCC but who were ineligible for other sorafenib clinical trials, also known as "compassionate use." In December 2005, sorafenib became commercially available, and this protocol was closed. Herein, we report our single-institution experience with this study.
Eligibility criteria included adults with advanced RCC with adequate organ function and performance status (PS). Treatment consisted of sorafenib 400 mg orally twice a day.
We enrolled 14 patients. The median age was 64 years, and PS was 2. All had metastatic RCC that had progressed after a median of 2 therapies. One patient (7%) had a partial response, and 3 (21%) had stable disease, for a clinical benefit rate of 29%. Severe toxicities included 1 patient with each of grade 4 thrombocytopenia, grade 3 warfarin-induced coagulopathy (drug-to-drug interaction), hypertension, diarrhea, anorexia, nausea, rash, and headache. Five subjects received concomitant radiation therapy without unexpected toxicities.
Sorafenib has modest activity in patients with advanced RCC who have progressed after multiple therapies.
2005年，一项III期试验表明，与安慰剂相比，索拉非尼治疗肾细胞癌（RCC）患者的无进展生存期显著延长。在美国联邦药物管理局进行全面审查期间，我们启动了一项治疗方案，作为向晚期RCC患者提供索拉非尼的一种机制，这些患者不符合其他索拉非尼临床试验的条件，也被称为“同情用药”。2005年12月，索拉非尼上市，该方案结束。在此，我们报告我们单机构的这项研究经验。
入选标准包括器官功能和体能状态（PS）良好的晚期RCC成年患者。治疗方案为索拉非尼400mg口服，每日两次。
我们纳入了14例患者。中位年龄为64岁，PS为2。所有患者均有转移性RCC，在接受中位2次治疗后病情进展。1例患者（7%）部分缓解，3例（21%）病情稳定，临床获益率为29%。严重毒性反应包括1例4级血小板减少症、1例3级华法林诱导的凝血病（药物相互作用）、高血压、腹泻、厌食、恶心、皮疹和头痛患者。5名受试者接受了同步放疗，未出现意外毒性反应。
索拉非尼对多线治疗后病情进展的晚期RCC患者有一定活性。
