Background: Programmed death receptor 1 PD-1 inhibitors have shown durable response and mild adverse events in adult malignancies. However, study on PD-1 inhibitors in pediatric patients remains limited, and a direct comparison of distinct PD-1 inhibitors in pediatric tumors is lacking.
Conclusions: Sintilimab demonstrated favorable tolerability and efficacy in pediatric patients with malignancies, with a safety and efficacy profile comparable to Pembrolizumab. For pediatric patients with advanced or recurrent malignancies receiving immune checkpoint inhibitor therapy, long-term monitoring of thyroid and cardiac function is recommended. Unfortunately, the prognosis for children with advanced or recurrent malignancies remains poor, with a year overall survival OS and progression free survival PFS of only There is an urgent need for safe and effective novel therapeutic strategies to improve outcomes for pediatric patients with advanced or recurrent malignancies.
Over the past decade, programmed cell death-1 PD-1 inhibitors have demonstrated durable responses and mild adverse events AEs in adult patients with advanced or recurrent cancers across numerous clinical trials. However, study regarding the safety and efficacy of PD-1 inhibitors in pediatric patients is limited 2 , 4. Results from an interim analysis of the KEYNOTE trial indicated that Pembrolizumab exhibited anti-tumor activity in pediatric patients with advanced melanoma and PD-L1-positive, advanced, relapsed, or refractory solid tumor or lymphoma 5.
Sintilimab, a fully humanized immunoglobulin G4 anti-PD-1 monoclonal antibody, has demonstrated promising tolerability and anti-tumor activity in pediatric patients with advanced or recurrent malignancies in a phase I study NCT 6. Previous studies have shown that PD-1 inhibitors are more effective in pediatric patients with lymphoma than other malignant solid tumors 7 , 8. These findings highlight the potential of Sintilimab as a promising therapeutic option for pediatric malignancies. To date, no direct comparisons of Sintilimab and Pembrolizumab in pediatric patients have been conducted.
Given the limited data, we performed a retrospective study to compare the safety and efficacy of Sintilimab and Pembrolizumab, both in combination with chemotherapy, radiotherapy, or targeted therapy, in pediatric patients with advanced or recurrent malignancies in a real-world setting.
