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Neoadjuvant immunotherapy in melanoma: pathological response as a surrogate endpoint?
Michel Meyers1, Oumnia Mouna1, Mireille Langouo1,2
1Université Libre de Bruxelles, Hôpital Universitaire de Bruxelles, Instiut Jules Bordet, Departement of Medical Oncology.
Current Opinion in Oncology
|January 27, 2025
Summary
Pathological complete response (pCR) is a key indicator for targeted melanoma therapy success. For immunotherapy, durable survival benefits are seen even with partial responses, suggesting pCR is not universally reliable for melanoma.
Area of Science:
- Oncology
- Immunotherapy
- Melanoma Research
Background:
- Pathological complete response (pCR) is a critical prognostic biomarker in various cancers, including breast cancer.
- Its role as a surrogate marker in melanoma, particularly with neoadjuvant therapies, is under increasing investigation.
Purpose of the Study:
- To evaluate the reliability of pathological complete response (pCR) as a surrogate marker for overall survival (OS) in melanoma patients receiving neoadjuvant immunotherapy.
- To compare the predictive value of pCR in melanoma treated with neoadjuvant immunotherapy versus targeted therapy.
Main Methods:
- Systematic review and analysis of recent clinical trials and studies on neoadjuvant therapy for melanoma.
- Comparative analysis of outcomes, including pCR and overall survival (OS), between immunotherapy and targeted therapy regimens.
Main Results:
- Targeted therapies for melanoma show pCR is crucial for long-term success (e.g., Neo-Combi, Neo-Trio).
- Immunotherapy offers durable survival benefits even with partial responses (e.g., OpACIN-neo, SWOG S1801).
- Immunotherapy demonstrates higher pathologic response rates and improved survival outcomes compared to pCR-dependent targeted therapies (e.g., NADINA, INMC analysis).
Conclusions:
- pCR is a validated prognostic biomarker in breast cancer, predicting long-term survival, especially in aggressive subtypes.
- In melanoma, pCR's predictive value differs between targeted treatments and immunotherapies.
- Further melanoma-specific studies are needed to validate pCR as a universal surrogate endpoint for neoadjuvant immunotherapy.

