Journal: Journal of clinical oncology : official journal of the American Society of Clinical Oncology
This phase III randomized superiority trial compared ultra-low-dose nivolumab with standard single-agent chemotherapy (docetaxel or paclitaxel) in 500 patients with advanced solid tumors who had progressed on at least one prior systemic therapy and had ECOG performance status 0–1.
The study population was predominantly:
- Head and neck cancers: 52%
- Lung cancers: 36%
- Two or more prior lines of therapy: 29%
Patients were randomized 1:1 to:
- Nivolumab: 20 mg IV every 2 weeks
- Standard chemotherapy: docetaxel or paclitaxel
Treatment was continued until progression or intolerable toxicity. The primary endpoint was overall survival (OS).
Efficacy outcomes:
- Overall survival (OS): Ultra-low-dose nivolumab significantly improved median OS to 5.88 months (95% CI, 4.99–7.13) versus 4.70 months with chemotherapy (95% CI, 3.91–5.65), with a hazard ratio of 0.80 (95% CI, 0.66–0.97; p = .022).
- One-year OS: Higher with nivolumab (27.3% vs 16.9%).
- Progression-free survival (PFS): Similar between arms—2.04 months (95% CI, 2.00–2.10) with nivolumab versus 2.09 months (95% CI, 2.04–2.17) with chemotherapy (HR 1.03; 95% CI, 0.86–1.23; p = .77).
Safety and quality of life:
- Grade ≥3 treatment-related adverse events: Less frequent with nivolumab (42.5% vs 60.8%; p < .001).
- Quality of life: Reported as significantly better with nivolumab.
Overall, ultra-low-dose nivolumab provided a statistically significant OS benefit over standard chemotherapy in previously treated solid tumors, with reduced toxicity and improved quality of life, supporting reconsideration of dosing strategies for immune checkpoint inhibitors and potential implications for broader global access.