CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Pediatric adrenocortical carcinoma.
Pediatric adrenocortical carcinoma.
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肾上腺皮质癌(ACC)是一种罕见的肾上腺内分泌恶性肿瘤,预后不良。在儿童中尤其罕见,20岁以下人群发病率为每百万人0.2–0.3例。儿童ACC主要与Li-Fraumeni和Beckwith-Wiedemann肿瘤易感综合征相关。由于该人群中Li-Fraumeni综合征相关TP53突变更常见,巴西南部儿童ACC发病率是其他地区的10至15倍。目前的治疗方案主要借鉴成人ACC,包括手术,和/或依托泊苷、多柔比星、顺铂(EDP)联合米托坦化疗。关于儿童ACC其他疗法的研究有限,包括米托坦、帕博利珠单抗、卡博替尼和自体CAR-T 细胞治疗。
Adrenocortical carcinoma (ACC) is a rare endocrine malignancy of the adrenal gland with an unfavorable prognosis. It is rare in the pediatric population, with an incidence of 0. 2-0. 3 patients per million in patients under 20 years old. It is primarily associated with Li-Fraumeni and Beckwith-Wiedemann tumor predisposition syndromes in children. The incidence of pediatric ACC is 10-15fold higher in southern Brazil due to a higher prevalence of TP53 mutation associated with Li-Fraumeni syndrome in that population.
Current treatment protocols are derived from adult ACC and consist of surgery and/or chemotherapy with etoposide, doxorubicin, and cisplatin (EDP) with mitotane. Limited research has been reported on other treatment modalities for pediatric ACC, including mitotane, pembrolizumab, cabozantinib, and chimeric antigen receptor autologous cell (CAR-T) therapy.
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