RT Journal Article SR Electronic T1 Nivolumab serum concentration in metastatic melanoma patients could be related to outcome and enhanced immune activity: a gene profiling retrospective analysis JF Journal for ImmunoTherapy of Cancer JO J Immunother Cancer FD BMJ Publishing Group Ltd SP e005132 DO 10.1136/jitc-2022-005132 VO 10 IS 11 A1 Domenico Mallardo A1 Diana Giannarelli A1 Maria Grazia Vitale A1 Domenico Galati A1 Giusy Trillò A1 Assunta Esposito A1 Maria Antonietta Isgrò A1 Grazia D'Angelo A1 Lucia Festino A1 Vito Vanella A1 Claudia Trojaniello A1 Andrew White A1 Teresa De Cristofaro A1 Michael Bailey A1 Sandro Pignata A1 Corrado Caracò A1 Antonella Petrillo A1 Paolo Muto A1 Piera Maiolino A1 Alfredo Budillon A1 Sarah Warren A1 Ernesta Cavalcanti A1 Paolo Antonio Ascierto YR 2022 UL http://jitc.bmj.com/content/10/11/e005132.abstract AB Background Nivolumab is an anti-PD-1 antibody approved for treating metastatic melanoma (MM), for which still limited evidence is available on the correlation between drug exposure and patient outcomes.Methods In this observational retrospective study, we assessed whether nivolumab concentration is associated with treatment response in 88 patients with MM and if the patient’s genetic profile plays a role in this association.Results We observed a statistically significant correlation between nivolumab serum concentration and clinical outcomes, measured as overall and progression-free survival. Moreover, patients who achieved a clinical or partial response tended to have higher levels of nivolumab than those who reached stable disease or had disease progression. However, the difference was not statistically significant. In particular, patients who reached a clinical response had a significantly higher concentration of nivolumab and presented a distinct genetic signature, with more marked activation of ICOS and other genes involved in effector T-cells mediated proinflammatory pathways.Conclusions In conclusion, these preliminary results show that in patients with MM, nivolumab concentration correlates with clinical outcomes and is associated with an increased expression of ICOS and other genes involved in the activation of T effectors cells.Data are available in a public, open access repository.