Table 3.
Factors affecting the outcome of patients treated with immunotherapy agents.
| Complete or partial response N (%) |
Stable or progressive disease N (%) |
P – value univariateg | P-value multivariateh |
OR [95% CI]i | ||
|---|---|---|---|---|---|---|
| Tumor type | NSCLCj | 7 (19%) | 29 (81%) | > 0.9999 | ||
| Other tumorsk | 12 (19%) | 51 (81%) | ||||
| TMBl | Low | 2 (4%) | 44 (96%) | 0.0006 | 0.006 | 0.09 (0.017–0.51) |
| Intermediate | 8 (24%) | 25 (76%) | 0.4211 | |||
| High | 9 (45%) | 11 (55%) | 0.0027 | 0.651 | 1.34 (0.37–4.85) | |
| APOBEC-related mutagenesism | Low | 1 (3%) | 28 (97%) | 0.0106 | 0.031 | 10.99 (1.25–100) |
| High | 18 (26%) | 52 (74%) | ||||
| Type of immunotherapy | Anti-PD-1/PD-L1 monotherapy | 16 (18%) | 75 (82%) | 0.1780 | ||
| Other immunotherapyn | 3 (38%) | 5 (62%) |
Abbreviations: CI = confidence interval; CTLA4 = cytotoxic T-lymphocyte associated protein 4; Mb = megabase; N = number; NSCLC = non-small cell lung cancer; OR = odds ratio; PD-1 = programmed death receptor-1; PD-L1 programmed death receptor-ligand 1; PFS = progression free survival; TMB = tumor mutational burden.
a Calculated using Fisher’s exact test. Supplemental Table 4 shows the univariate analysis with all demographic factors.
b All univariate p-values of ≤ 0.05 were included in the multivariate analysis, using a binomial logistic regression model.
c OR > 1.0 implies higher chance of response.
d Histologies of NSCLC included adenocarcinoma (N = 30) and squamous cell carcinoma (N = 6).
e Other tumors include 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).
f TMB low = 1–5 mutations/Mb; TMB intermediate = 6–19 mutations/Mb; TMB high ≥ 20 mutations/Mb.
g An estimate of the APOBEC-related mutagenesis was obtained for each patient using the AMSE tool, available at https://github.com/KwatME/mutational_signature. Patients were classified in APOBEC-related mutagenesis LOW phenotype for scores ≤ 0.727 and in APOBEC-related mutagenesis HIGH phenotype for scores > 0.727.
h Other immunotherapy included OX40 (n = 2), anti-CD73 (n = 1), anti-CTLA4 (n = 2), OX40+ IDO (n = 1), anti-PD-1+ anti-CTLA4 (n = 1), and IDO+ anti-PD-1 (n = 1).