Predictive value of TMB for ICI treatment response and survival
EVENTS, METASTASIS, REMISSIONS
The median follow-up duration for PFS and OS at data cut-off (March 15, 2022) was 7.6 and 14.9 months, respectively. 25 PFS and 24 OS events were documented. Among all included patients, the one-year PFS and OS rates were 18% and 26%, respectively. Median PFS was 2.5 months (95% confidence interval [CI] 1.77–3.32), while the median OS was 3.8 months (95% CI 3.3–8.8) (Supplementary Fig. Dual checkpoint blockade led to two complete (6.5%) and three partial remissions (9.7%) according to RECIST v1.1, summing up to an ORR of 16.2% (95% CI 5.5-33.7%). Among these, three of five patients (60%; 95% CI 14.7–94.7%) with high TMB and two of 26 patients (7.7%; 95% CI 0.9-25.1%) with low TMB achieved an objective response (Fisher´s exact test Treatment responseSource data are provided as a Source Data file.No treatment-related mortality occurred. Safety data are shown in Table Incidence of treatment-related adverse eventsSource data are provided as a Source Data file.Survival analysis in patients with high vs. low TMB revealed a clinically meaningful longer PFS in the TMBBoth PFS and OS were significantly longer in patients with low compared to patients with intermediate or high metastasis burden scores (median PFS 18.3 vs. 1.45 vs. 2.3 months, log-rank 