Table 2.

Univariate and multivariate analysis of factors affecting outcome for all patients treated with immunotherapy agents (TMB low or intermediate vs. high; N = 151)a

VariableGroup (N)PR/CR N (%)OR (95% CI)bP univariate (PR/CR)cP multivariate (PR/CR)Median PFS (mos)dHR (95% CI) (PFS)bP univariate (PFS)dP multivariate (PFS)Median OS (mos)dHR (95% CI) (OS)aPunivariate (OS)ePmultivariate (OS)
Age≤60 years (n = 78)20 (26%)0.66 (0.32–1.33)0.28734.01.12 (0.77–1.63)0.539628.41.03 (0.61–1.72)0.9165
>60 years (n = 73)25 (34%)1.51 (0.75–3.14)5.70.89 (0.61–1.29)25.40.97 (0.58–1.63)
GenderMen (n = 93)33 (36%)2.11 (0.97–4.57)0.06750.2355.80.70 (0.47–1.03)0.05720.36228.40.88 0.51–1.50)0.6204
Women (n = 58)12 (21%)0.47 (0.22–1.03)3.61.44 (0.97–2.12)16.31.14 (0.67–1.95)
EthnicityCaucasian (n = 111)38 (34%)2.45 (0.99–6.29)0.06850.6045.90.58 (0.36–0.92)0.00660.98328.40.65 (0.35–1.20)0.1192
Hispanic (n = 18)3 (17%)0.43 (0.13–1.57)0.27452.61.38 (0.73–2.61)0.254315.61.46 (0.64–3.30)0.2927
Asian (n = 9)1 (11%)0.28 (0.02–1.94)0.28082.02.61 (0.86–7.90)0.00630.083Not reached (median f/u 3.4 mos)1.84 (0.49–7.01)0.2265
African American (n = 9)3 (33%)1.19 (0.31–4.45)1.00003.91.30 (0.55–3.07)0.5002Not reached (median f/u 6.5 mos)1.26 (0.35–4.60)0.6916
Other (n = 4)0 (9%)0 (0–2.40)0.31814.11.54 (0.45–5.23)0.390238.331.02 (0.25–4.27)0.9730
Tumor typeMelanoma (n = 52)26 (50%)3.68 (1.71–7.82)0.00070.5629.30.36 (0.25–0.51)<0.00010.035Not reached (median f/u of 15.6 mos)0.26 (0.16–0.43)<0.00010.006
NSCLC (n = 36)7 (19%)0.49 (0.19–1.23)0.14602.82.00 (1.21–3.27)0.00070.9928.01.90 (0.97–3.72)0.02130.794
Other tumors5 (n = 63)12 (19%)0.39 (0.18–5.51)0.01870.3012.91.67 (1.12–2.50)0.00560.99211.22.13 (1.23–3.69)0.00220.794
TMBLow to intermediate (n = 113)23 (20%)0.19 (0.09–0.41)0.0001<0.0013.32.98 (2.02–4.41)<0.0001<0.00116.33.03 (1.72–5.33)0.00360.016
High (n = 38)22 (58%)5.38 (2.44–11.58)12.80.34 (0.23–0.50)Not reached (median f/u of 10.5 mos)0.33 (0.19–0.58)
Type of immunotherapyAnti-PD-1/PD-L1 monotherapy (n = 102)21 (21%)0.27 (0.13–0.58)0.00060.7433.32.41 (1.65–3.50)<0.00010.58815.72.74 (1.63–4.59)0.00050.820
Anti-CTLA4 alone (n = 15)2 (13%)0.33 (0.07–1.40)0.23294.71.08 (0.58–2.01)0.795728.40.87 (0.39–1.95)0.7529
High dose IL2 (n = 9)5 (56%)3.19 (0.88–10.72)0.127037.00.40 (0.23–0.71)0.01460.070Not reached (median f/u of 34.6 mos)0.37 (0.17–0.80)0.06140.284
Anti-CTLA4/anti-PD-1/PD-L1 (n = 17)13 (77%)10.36 (3.05–30.18)<0.00010.004Not reached (median f/u 9.2 mos)0.27 (0.16–0.44)0.00060.024Not reached (median f/u of 16.3 mos)0.20 (0.10–0.40)0.01070.172
Other immunotherapyg (N = 8)4 (50%)2.49 (0.69–8.82)0.23928.90.77 (0.32–1.87)0.6044Not reached (median f/u of 5.5 mos)0.81 (0.23–2.92)0.7694
  • NOTE: Significant P values were bolded.

  • Abbreviations: CI, confidence interval; CR, complete response; PD, progressive disease; PR, partial response; SD, stable disease.

  • Abbreviations: CI, confidence interval; CR, complete response; PR, partial response.

  • aAll univariate P values of ≤0.1 were included in the multivariate analysis. For a similar analysis by TMB low vs. intermediate to high, see Supplementary Table S4.

  • bOR >1.0 implies higher chance of response; HR <1.0 implies less chance of progression or death.

  • cCalculated using Fisher exact test.

  • dAll medians for PFS and OS calculated by Kaplan–Meier.

  • eCalculated using log-rank (Mantel-Cox) test.

  • fOther tumors: Adrenal carcinoma (n = 1), appendix adenocarcinoma (n = 1), basal cell carcinoma (n = 2), bladder transitional cell carcinoma (n = 4), breast cancer (n = 3), cervical cancer (n = 2), colon adenocarcinoma (n = 5), cutaneous squamous cell carcinoma (n = 8), hepatocellular carcinoma (n = 3), head and neck (n = 13), Merkel cell carcinoma (n = 2), ovarian carcinoma (n = 2), pleural mesothelioma (n = 1), prostate cancer (n = 1), renal cell carcinoma (n = 6), sarcoma (n = 3), thyroid cancer (n = 3), unknown primary squamous cell carcinoma (n = 2), and urethral squamous cell carcinoma (n = 1).

  • gOther immunotherapy: OX40 (n = 3), anti-CD73 (n = 1), Talimogene laherparepvec (n = 2), OX40+anti-PD-L1 (n = 1), and IDO+anti-PD-1/PD-L1 (n = 1).