Neoadjuvant treatment with immune checkpoint inhibitors in patients with melanoma: A real‐life retrospective study

Summary Background Neoadjuvant treatment with ipilimumab and nivolumab has shown efficacy in melanoma patients with nodal metastases in clinical trials. Real world data on neoadjuvant therapies is lacking. Patients and Methods This study investigates the effectiveness of neoadjuvant therapy in a real‐world setting and included all melanoma patients who received combined anti‐CTLA4/PD1 immunotherapy prior to resection. Pathologic and radiologic responses as well as treatment‐related adverse events were assessed, and recurrence‐free survival (RFS) was compared between patients with major pathologic response (mPR) and patients without mPR. Results In total, 24 patients were analyzed, including patients with distant metastases and patients with prior adjuvant treatment. Median follow‐up was 21.5 months. Upon histologic assessment, mPR was achieved in 50% (12/24) of the patients, including two patients with lung metastases and three patients who progressed after prior adjuvant anti‐PD1 therapy. Radiologic response after neoadjuvant treatment correlated with mPR. No patient with mPR relapsed during follow‐up (median RFS not reached) compared to six out of 12 patients without mPR (median RFS = 13 months, p = 0.005). Conclusions Neoadjuvant treatment with ipilimumab and nivolumab is effective in real‐world patients with different melanoma subtypes, different stages of disease and even advanced primary tumor.