OPTIM: a randomized phase II trial of nivolumab followed by nivolumab-ipilimumab or docetaxel at progression in recurrent/metastatic squamous cell carcinoma of the head and neck (OPTIM; AIO-KHT-0117)
Mené sur 31 patients atteints d'un carcinome épidermoïde de la tête et du cou récidivant ou de stade métastatique, cet essai de phase II évalue l'efficacité, du point de vue du taux de réponse objective, et la toxicité du nivolumab suivi d'un traitement par nivolumab-ipilimumab ou docétaxel
Background: Treatment options after PD-1 inhibition for recurrent/metastatic squamous cell carcinoma of the head and neck (R/M-SCCHN) remain limited. We investigated whether staggered immune checkpoint inhibition could improve outcomes compared with docetaxel in nivolumab-refractory disease.
Methods: In this randomized phase II trial, adults with platinum-refractory R/M-SCCHN received nivolumab and were randomized at progression to nivolumab 3 mg/kg every 2 weeks plus ipilimumab 1 mg/kg every 6 weeks (NIVO-IPI) or docetaxel 75 mg/m² every 3 weeks (DOCE) until progression or intolerance. The primary endpoint was objective response rate (ORR) per RECIST 1.1; progression-free survival (PFS), overall survival (OS), and safety were secondary endpoints. PD-L1 expression was assessed in all patients.
Results: Among 14 patients in the NIVO-IPI arm and 17 in the DOCE arm, ORR was 0% versus 17.6%, median PFS was 1.97 versus 3.66 months (P = 0.036), and median OS was 3.97 versus 11.9 months (P = 0.356), respectively. Twelve-month OS rates were 28.6% and 44.6%. Outcomes were similar irrespective of PD-L1 status. Treatment-emergent adverse events occurred in 84.6% versus 81.3% of patients, with grade ≥3 events in 38.5% and 68.8%.
Conclusions: NIVO-IPI did not improve efficacy over docetaxel and showed numerically inferior survival, whereas docetaxel was associated with greater toxicity.
British Journal of Cancer , résumé, 2026