Treatment Outcomes With Novel Targeted and Immunotherapeutic Regimens in CAYA Hodgkin Lymphoma: A Retrospective Study

ABSTRACT Background To evaluate the efficacy and safety of novel chemotherapy regimens, including brentuximab vedotin (BV) and immune checkpoint inhibitors (ICIs), in children, adolescents, and young adults (CAYA) with Hodgkin lymphoma (HL). Methods A retrospective analysis was conducted on untreated Stage IIB (bulky), III, or IV HL patients (≤ 24 years) who were admitted to Cancer Hospital of the Chinese Academy of Medical Sciences and Beijing Children's Hospital, Capital Medical University, from 2017 to 2024. The cohort compared conventional regimens with BV‑based therapies (BV‑AVD ± R). Furthermore, the efficacy and safety of ICIs were evaluated in relapsed/refractory cHL patients receiving second‑line or later therapy. Results In this comparison between BV and conventional regimens, 182 patients were enrolled. The median follow‐up was 38 months. No significant differences were observed in objective response rate (ORR) (96.4% vs 95.4%, p = 0.927) or progression‐free survival (PFS) rate (92.8% vs 95.3%, p = 0.466).However, the incidence of Grade 4 hematologic toxicity was significantly higher with conventional therapy compared to BV‐AVD±R (61.2% vs 25.6%, p < 0.001). The BV‐AVD group alone achieved a significantly higher ORR than the BV ‐AVD+R group (100% vs. 93.9%, p = 0.047), while there was no significant difference in PFS rate. However, the BV‐AVD + R group exhibited a significantly higher incidence of Grade 3–4 hematologic toxicities (78.8% vs. 20.0%, p = 0.023). Immune checkpoint inhibitors was well‐tolerated with infrequent adverse events, and 18/19 cases (94.7%) demonstrated treatment efficacy. Conclusion For patients with stage II Hodgkin lymphoma presenting with bulky disease, as well as those with stage III–IV disease, the BV‐AVD regimen is recommended as first‐line therapy, given its more favorable toxicity profile.Although ICIs represent a promising therapeutic approach, existing studies are constrained by limited sample sizes. Future research should focus on expanding patient cohorts to refine treatment strategies and ultimately improve clinical outcomes.