Pediatric Melanoma: Emerging Therapies and Ongoing Clinical Trials.
This narrative review summarizes current pediatric melanoma management, available evidence for checkpoint inhibitors and targeted therapies, and ongoing trials of CAR T cells, NK-cell infusions, Wnt/β-catenin inhibitors, and cancer vaccines.
Open original publication →What the AI sees
This narrative review summarizes current pediatric melanoma management, available evidence for checkpoint inhibitors and targeted therapies, and ongoing trials of CAR T cells, NK-cell infusions, Wnt/β-catenin inhibitors, and cancer vaccines.
Research significance
The supplied record supports that adult-derived immunotherapies and targeted therapies are already being considered for pediatric melanoma and that several novel approaches are under clinical investigation; it is reasonable—but not demonstrated here—to hypothesize that molecularly stratified, pediatric-specific use of these therapies could improve efficacy or safety.
Source abstract
Pediatric melanoma is a rare but clinically distinct malignancy, accounting for 1-4% of all melanoma cases. It encompasses unique subtypes-Spitzoid melanoma, melanoma arising in congenital melanocytic nevi, and conventional melanoma-each with specific biological behavior and therapeutic implications. While surgical excision remains the primary treatment, systemic therapies are often adapted from adult protocols and used off-label, with limited pediatric-specific data on efficacy, dosing, and safety. This narrative review summarizes current treatment approaches, including the use of immune checkpoint inhibitors and targeted therapies, and highlights outcomes from available prospective and retrospective studies. Furthermore, it reviews ongoing clinical trials investigating novel strategies such as chimeric antigen receptor (CAR) T-cell therapy, natural killer (NK) cell infusions, Wnt/β-catenin pathway inhibitors, and cancer vaccines. Despite emerging interest, challenges persist due to the rarity of the disease, lack of long-term toxicity data, and limited biomarker-driven stratification. Expanding pediatric-focused clinical research, integrating molecular profiling, and addressing developmental and psychosocial needs are essential for advancing care. Ongoing trials may help pave the way for safer and more effective therapies tailored to this vulnerable population.