Department of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), University of Texas M.D. Anderson Cancer Center, 1515Holcombe Boulevard, Houston, TX 77030, USA.
Clin Cancer Res. 2010 Aug 1;16(15):4031-7. doi: 10.1158/1078-0432.CCR-10-0672. Epub 2010 Jun 4.
To compare clinical outcomes of metastatic head and neck cancer patients treated in phase I clinical trials with clinical outcomes of those patients who had their last Food and Drug Administration (FDA)-approved therapy in the setting of metastatic disease.
We retrospectively reviewed the outcomes of 61 consecutive patients with head and neck tumors treated in 36 phase I trials at The University of Texas M.D. Anderson Cancer Center between July 2004 and September 2009.
The most common histology was head and neck squamous cell carcinoma (62%). Median age was 55 years (range, 26-80). Eastern Cooperative Oncology Group performance status was 0 to 1 for 95% of patients. Fifty-nine patients had received FDA-approved drugs as the backbone of their last systemic therapy before inclusion in phase I trials (median, 2 systemic therapies). Progression-free survival (PFS) on phase I trials was not inferior to PFS on their last FDA-approved therapies (12 versus 10.7 weeks, log-rank P = 0.87). Fifty-three patients were evaluable for response by Response Evaluation Criteria in Solid Tumors criteria. Four (7%) had partial responses and 16 (26%) had stable disease for > or =4 months. In univariate analysis, number of metastatic sites, lactate dehydrogenase (LDH) levels at baseline, and Royal Marsden Hospital prognosis scores were significant predictors of survival. Only LDH was significant in multivariate analysis (hazard ratio, 6.35; P < or = 0.0001).
For patients with heavily pretreated advanced head and neck tumors, PFS on phase I trials is not inferior to PFS with their last FDA-approved therapy. The only significant predictor of survival in the multivariate analysis was baseline LDH.
比较接受 I 期临床试验治疗的转移性头颈部癌患者的临床结局与在转移性疾病背景下接受最后一次美国食品药品监督管理局（FDA）批准的治疗的患者的临床结局。
我们回顾性分析了 2004 年 7 月至 2009 年 9 月期间，在德克萨斯大学 MD 安德森癌症中心的 36 项 I 期试验中连续治疗的 61 例头颈部肿瘤患者的结果。
最常见的组织学类型是头颈部鳞状细胞癌（62%）。中位年龄为 55 岁（范围，26-80）。95%的患者的东部合作肿瘤组体能状态为 0-1。59 例患者在入组 I 期试验前接受了 FDA 批准的药物作为其最后一次全身治疗的基础（中位数，2 种系统治疗）。I 期试验的无进展生存期（PFS）并不逊于其最后一次 FDA 批准治疗的 PFS（12 周与 10.7 周，对数秩检验 P=0.87）。53 例患者可根据实体瘤反应评估标准评估反应。4 例（7%）有部分缓解，16 例（26%）有≥4 个月的稳定疾病。单因素分析中，转移部位数量、基线乳酸脱氢酶（LDH）水平和皇家马斯登医院预后评分是生存的显著预测因素。只有 LDH 在多因素分析中具有统计学意义（风险比，6.35；P<0.0001）。
对于接受过多线治疗的晚期头颈部肿瘤患者，I 期试验的 PFS 并不逊于其最后一次 FDA 批准的治疗。多因素分析中唯一有统计学意义的生存预测因素是基线 LDH。
