Background and objective: Platinum-based doublet chemotherapy is still the standard first-line treatment for non-small cell lung cancer (NSCLC). Previous studies have demonstrated that pemetrexed combined with platinum had promising efficacy and safety profile in NSCLC, especially in patients with nonsquamous NSCLC. This trial was conducted to evaluate the efficacy and safety of pemetrexed made in China as first-line treatment.
Methods: The present study was a randomized, controlled, multicenter clinical trial. Patients were randomly assigned (1:1) to receive cisplatin plus pemetrexed chemotherapy (PC group) or gemcitabine plus cisplatin (GC group) every 3 weeks. The primary end point was progression free survival (PFS) and the secondary end points included 1 year survival rate, objective response rate (ORR), survival without grade 3/4 toxicity (SWT3/4) and safety profile.
Results: A total of 288 patients from 20 institutions across China were enrolled into the study. Based on the Full Analyses Set (FAS), the PFS was 168 days (5.6 months) vs 140 days (4.7 months) (P=0.16), one year survival rate was 50.0% vs 54.9% (P=0.47), ORR was 24.4% vs 14.2% (P=0.06) in the PC group and the GC group, respectively; Survival without grade 3/4 toxicity was 11.3 months in GC group vs 8.1 months in PC group (P=0.23). In terms of the safety, side effects were less observed on the PC group (81.95% vs 93.75%, P=0.003). The main side effects included leukopenia, neutropenia, emesis, anemia, thrombopenia.
Conclusions: The both regimens have similar efficacy as the treatment for advanced nonsquamous NSCLC, but pemetrexed plus cisplatin regimen has better safety profile and seems to have longer PFS, which makes it a new option as the first line setting.
背景与目的: 目前晚期非小细胞肺癌（non-small cell lung cancer, NSCLC）的标准治疗仍是含铂两药方案，国内外多项临床研究显示培美曲塞联合顺铂在晚期非鳞NSCLC具有较好的疗效及安全性，本研究拟评估国产培美曲塞二钠联合顺铂一线治疗晚期NSCLC的疗效与安全性。
方法: 本研究是一项多中心、随机、阳性药物平行对照的临床试验。入组患者按1:1随机分为两组，分别接受培美曲塞二钠联合顺铂（PC组）或吉西他滨联合顺铂（GC组）治疗，21天为一个周期。主要研究终点为无进展生存期，次要研究终点主要包括1年生存率、客观缓解率、无3度或4度毒性生存期及其安全性。
结果: 全国20家研究中心共纳入288例患者（各组144例），基于全分析集进行分析，PC组与GC组患者的中位无进展生存期分别为168天（5.6个月）和140天（4.7个月）（P=0.16）；1年生存率分别为50.0%和54.9%（P=0.47）；客观缓解率分别为24.4%和14.2%（P=0.06）；无3度/4度毒性生存期分别为11.3个月和8.1个月（P=0.23）。总体不良反应发生率PC组明显低于GC组（81.95% vs 93.75%, P=0.003）。
结论: 两种含铂方案治疗晚期非鳞NSCLC具有相似的疗效，但PC方案不良反应更轻，有望成为晚期非鳞NSCLC一线治疗的新选择。
Trial registration: ClinicalTrials.gov NCT01194453.