New Insights into the Treatment of Nasopharyngeal Carcinoma in Children, Adolescents, and Young Adults: A Retrospective Study

Mengwei Ren¹Jing Tian²Mengyuan Han¹Shiran Sun¹Kai Wang¹Runye Wu¹Meng Yuan³Junlin Yi^1*Fei Ma^1*Sidan Li^1*

• ¹Center for National Cancer, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China
• ²Beijing Shijitan Hospital Capital Medical University, Beijing, China
• ³Beijing Children's Hospital Capital Medical University, Beijing, China

Objective To evaluate the efficacy, safety, and survival impacts of diverse induction chemotherapy regimens (including combination therapy with immune checkpoint inhibitors [ICIs]), radiotherapy modalities, and consolidation therapy in children, adolescents, and young adults (CAYA) with locally advanced or metastatic nasopharyngeal carcinoma (NPC). Methods This multicenter retrospective study analyzed 102 CAYA NPC patients (aged 6–24 years; stage III–IVB) from two Chinese centers (January 2011–October 2024). All received induction therapy followed by concurrent chemoradiotherapy (CCRT), radiotherapy combined with concurrent anti-EGFR therapy, or radiotherapy alone, with select cases receiving consolidation (median follow-up: 22 months). Results GP and TPF induction achieved higher objective response rates (ORR) vs. TP (GP: 68.0% vs. TP: 31.6%, P = 0.005; TPF: 89.5% vs. TP: 68.0%, P < 0.001), though no significant difference in long-term survival was observed. ICIs + chemotherapy (n=15) improved ORR (93.3% vs. 53.3%, P=0.013) though without a demonstrable difference in survival metrics at this follow-up. In patients achieving partial response (PR) post-induction, CCRT/anti-EGFR therapy + radiotherapy improved 1-year progression-free survival (PFS: 94.5% vs. 50.0%, P < 0.001) and distant metastasis-free survival (DMFS: 97.4% vs. 50.0%, P < 0.001). For stable disease (SD) patients, multimodal therapy increased 5-year overall survival (OS: 100% vs. 66.7%, P = 0.046). Consolidation therapy (n=24) in locally advanced NPC was associated with clinical stage (P = 0.001) but not survival (P > 0.05). Conclusion TPF/GP regimens improved short-term responses with manageable toxicity. The addition of ICIs enhanced objective response rates, though survival benefits were not assessable within the limited follow-up period. CCRT demonstrated survival advantages over radiotherapy alone, especially in PR patients, while consolidation therapy showed limited benefit except in advanced subgroups. These findings, generated from a retrospective analysis, highlight the need for personalized strategies and warrant validation in larger prospective trials.