Is targeted therapy more effective than chemotherapy for patients with NSCLC?

This study compared the effectiveness and safety of nivolumab (Opdivo) plus ipilimumab (Yervoy) versus chemotherapy for non-small cell lung cancer (NSCLC). The authors concluded that this combination treatment improved survival outcomes more than chemotherapy.

Treatment for NSCLC often includes chemotherapy or biological therapies such as nivolumab and ipilimumab. These agents work by binding to a molecule called PD-L1. This molecule helps cancer cells evade the body's immune system. So, blocking PD-L1 allows immune cells to attack cancer cells. 

Previous studies have shown that nivolumab plus ipilimumab are more effective than chemotherapy alone. However, the long-term outcomes of this treatment combination for patients with NSCLC are unclear. This study examined the effectiveness and safety of nivolumab and ipilimumab compared with chemotherapy.

This study had 1739 patients with advanced NSCLC. Patients were divided into two groups based on how much PDL-1 was present in their tumors. In Group 1, 1189 patients had PD-L1 in 1% or more of tumor cells. These patients received nivolumab plus ipilimumab (NI; 396 patients), nivolumab (396), or chemotherapy only (397). In Group 2, 550 patients had PD-L1 in less than 1% of tumor cells. These patients received NI (187 patients), nivolumab plus chemotherapy (NC; 177), or chemotherapy only (186). Patients were followed for an average of 29.3 months.

Overall, more patients treated with nivolumab plus ipilimumab were still alive 2 years later (40.4%) compared to chemotherapy (29.7%).

In Group 1, NI was the most effective treatment. 40% of patients who received NI were still alive 2 years later compared to 36.2% (nivolumab) and 32.8% (chemotherapy). NI treatment lowered mortality risk by 21% compared to chemotherapy alone.

In Group 2, NI was the most effective treatment. 40.4% of patients who received NI were still alive 2 years later compared to 34.7% (NC) and 23% (chemotherapy). NI treatment lowered mortality risk by 38% compared to chemotherapy alone.

Overall, slightly fewer patients treated with NI had side effects (76.7%) compared to chemotherapy (81.9%). Diarrhea and rash were most common (17% each). In the chemotherapy group, nausea (36.1%) and low red blood cell count (33.0%) were most common. Serious side effects were more common with NI treatment than chemotherapy (24.5% vs. 13.9%).

The authors concluded that NI was more effective than chemotherapy for patients with NSCLC. However, more patients treated with NI had serious side effects.

The manufacturer of nivolumab and ipilimumab, Bristol Myers Squibb, funded this study.  This study did not include an ipilimumab-only group, which may limit these results.