Although patients treated with immune checkpoint inhibitors (ICIs) in the real world generally had worse overall survival (OS) for most tumor indications compared with patients in pivotal clinical trials, their outcomes were often better than that of patients who received the standard of care, according the a retrospective study in JCO Clinical Cancer Informatics.

Using data from electronic health records for patients cared for in the Veterans Affairs health care system, study researchers identified 11,888 patients who received ICIs for various cancer indications.

The most common tumor type was non-small cell lung cancer (NSCLC; 51.1%), followed by melanoma (14.4%), renal cell carcinoma (8.1%), squamous cell carcinoma of the head and neck (6.8%), urothelial cancer (6.4%), and hepatocellular carcinoma (4.5%).

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Patients who received ICIs had worse OS compared with patients treated in pivotal clinical trials, except for patients treated with nivolumab for melanoma in the first-line setting and pembrolizumab or nivolumab for NSCLC in the second or later line setting.

The study authors explained that the worse survival is “not unexpected” given that patients in these cohorts tend to be older and have more comorbidities. The average age of starting treatment was 69 years and 68.9% of  patients had “substantial” comorbidities.

The analysis also showed that when patients were stratified by frailty, patients who were not frail generally had survival outcomes closer to those seen in the pivotal clinical trials compared with patients who were frail.

Outcomes from real-world patients were also compared with historical controls for select indications, revealing that patients who received ICIs lived significantly longer than historical controls. The greatest survival benefit was seen for patients with melanoma who received ICIs in the first-line setting compared with historical controls (adjusted hazard ratio, 0.278; P <.001).

When outcomes from real-world patients were compared with concurrent controls, survival tended to be longer for patients who received ICIs.

“The generally favorable survival outcome observed among patients receiving ICIs relative to both historic and concurrent controls suggests a treatment benefit for ICIs,” the study authors wrote.


La J, Cheng D, Brophy MT, et al. Real-world outcomes for patients treated with immune checkpoint inhibitors in the Veterans Affairs system. JCO Clin Cancer Inform. 2020;4:918-928. doi:10.1200/CCI.20.00084