Development and validation of MRI-derived deep learning score for non-invasive prediction of PD-L1 expression and prognostic stratification in head and neck squamous cell carcinoma

Abstract

Background

Immunotherapy has revolutionized the treatment landscape for head and neck squamous cell carcinoma (HNSCC) and PD-L1 combined positivity score (CPS) scoring is recommended as a biomarker for immunotherapy. Therefore, this study aimed to develop an MRI-based deep learning score (DLS) to non-invasively assess PD-L1 expression status in HNSCC patients and evaluate its potential effeciency in predicting prognostic stratification following treatment with immune checkpoint inhibitors (ICI).

Methods

In this study, we collected data from four patient cohorts comprising a total of 610 HNSCC patients from two separate institutions. We developed deep learning models based on the ResNet-101 convolutional neural network to analyze three MRI sequences (T1WI, T2WI, and contrast-enhanced T1WI). Tumor regions were manually segmented, and features extracted from different MRI sequences were fused using a transformer-based model incorporating attention mechanisms. The model’s performance in predicting PD-L1 expression was evaluated using the area under the curve (AUC), sensitivity, specificity, and calibration metrics. Survival analyses were conducted using Kaplan-Meier survival curves and log-rank tests to evaluate the prognostic significance of the DLS.

Results

The DLS demonstrated high predictive accuracy for PD-L1 expression, achieving an AUC of 0.981, 0.860 and 0.803 in the training, internal and external validation cohort. Patients with higher DLS scores demonstrated significantly improved progression-free survival (PFS) in both the internal validation cohort (hazard ratio: 0.491; 95% CI, 0.270–0.892; P = 0.005) and the external validation cohort (hazard ratio: 0.617; 95% CI, 0.391–0.973; P = 0.040). In the ICI-treated cohort, the DLS achieved an AUC of 0.739 for predicting durable clinical benefit (DCB).

Conclusions

The proposed DLS offered a non-invasive and accurate approach for assessing PD-L1 expression in patients with HNSCC and effectively stratified HNSCC patients to benefit from immunotherapy based on PFS.

Supplementary Information

The online version contains supplementary material available at 10.1186/s40644-025-00837-5.