Development of thrombocytopenia is associated with improved survival in patients treated with immunotherapy

Future Sci OA. 2020 Jun 25;6(7):FSO581. doi: 10.2144/fsoa-2020-0021.


Background: Immune-related adverse events are associated with efficacy of immune checkpoint inhibitors (ICIs). We hypothesize that immune-mediated thrombocytopenia could be a biomarker for response to ICIs.

Materials & methods: This retrospective study included 215 patients with metastatic malignancies treated with ICIs. Patients were stratified by nadir platelet count. Outcomes of interest were progression-free survival and overall survival.

Results: On multivariate analysis, grade 1 thrombocytopenia was positively associated with overall survival compared with patients who did not develop thrombocytopenia (hazard ratio [HR]= 0.28 [95% CI: 0.13-0.60]; p = 0.001), while grade 2-4 thrombocytopenia was not (HR= 0.36 [95% CI: 0.13-1.04]; p = 0.060). There was no association between degree of thrombocytopenia and progression-free survival.

Conclusion: Follow-up studies are warranted to substantiate the predictive significance of thrombocytopenia in patients receiving ICIs.

Keywords: PD1/PDL-1 inhibitors; checkpoint inhibitors; immune-mediated; immunotherapy; platelets; prognosis; survival; thrombocytopenia.