肿瘤细胞治疗研究
英文原题：The emerging role of immunotherapy in improving outcomes in Down syndrome - associated acute lymphoblastic leukemia: an insightful review.
The emerging role of immunotherapy in improving outcomes in Down syndrome - associated acute lymphoblastic leukemia: an insightful review.
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唐氏综合征相关急性淋巴细胞白血病（DS-ALL）是一种生物学上独特的亚型，具有较高的治疗相关死亡率和复发率。
唐氏综合征相关急性淋巴细胞白血病（DS-ALL）是一种生物学上独特的亚型，具有较高的治疗相关死亡率和复发率。基因组分析显示，CRLF2 重排、JAK-STAT 通路突变以及 BCR::ABL1 样疾病等高危亚型频繁出现。传统化疗可实现缓解，但在 DS 患者中受限于毒性。近年来免疫治疗的进展，包括 blinatumomab、inotuzumab ozogamicin 和靶向 CD19 的 CAR-T 细胞疗法，在儿童和成人 DS-ALL 中显示出显著疗效和良好的耐受性。Blinatumomab 和 inotuzumab 可实现微小残留病清除，并作为减少化疗或桥接策略，而 CAR-T 细胞疗法具有治愈潜力，并可能减少对移植的依赖。纳入早期免疫治疗、MRD 指导治疗和无化疗方案的新兴方法可能改善生存率和生活质量。针对 DS 的前瞻性试验对于优化治疗和缩小这一高危人群的结局差距至关重要。
Down syndrome-associated acute lymphoblastic leukemia (DS-ALL) is a biologically distinct subtype with high rates of treatment-related mortality and relapse. Genomic profiling reveals frequent CRLF2 rearrangements, JAK-STAT pathway mutations, and high-risk subtypes such as BCR::ABL1-like disease. Conventional chemotherapy achieves remission but is limited by toxicity in DS patients. Recent advances in immunotherapy, including blinatumomab, inotuzumab ozogamicin, and CD19-directed CAR T-cell therapy, have shown significant efficacy and favorable tolerability in pediatric and adult DS-ALL. Blinatumomab and inotuzumab enable minimal residual disease clearance and serve as chemotherapy-sparing or bridging strategies, while CAR T-cell therapy offers curative potential and may reduce transplant reliance. Emerging approaches incorporating early immunotherapy, MRD-guided treatment, and chemotherapy-free regimens may improve survival and quality of life. Prospective DS-specific trials are essential to optimize therapy and close the outcome gap in this high-risk population.
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