肿瘤细胞治疗研究
英文原题:Case Report: CD19 CAR-T therapy induces durable remission in a pediatric patient with TP53-mutated, refractory Burkitt lymphoma: a 30-month follow-up.
Case Report: CD19 CAR-T therapy induces durable remission in a pediatric patient with TP53-mutated, refractory Burkitt lymphoma: a 30-month follow-up.
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TP53突变的伯基特淋巴瘤(BL)具有显著化疗耐药性和不良预后,给临床治疗带来重大挑战。CAR-T 细胞疗法在复发/难治性BL中显示出希望,但其对TP53突变病例的疗效仍需进一步验证。本病例报告介绍一名16岁男性青少年,确诊为TP53突变BL;患者最初因反复腹痛就诊,被误诊为高级别B细胞淋巴瘤。疾病对两周期化疗(DA-EPOCH-R和AZA+R-CDOP)无应答,接受三线R-CODOX-M后仍进展。重新评估确诊BL后,患者接受CD19靶向CAR-T 细胞治疗(阿基仑赛注射液,Axi-cel),达到完全代谢缓解(CMR)。基线乳酸脱氢酶(LDH)显著升高,为1,343 U/L。截至2025年3月最近一次随访,CAR-T 输注后30个月患者仍处于缓解状态,功能完全恢复并重返正常学业生活。本病例属于已报道的较年轻商业化Axi-cel治疗BL病例之一,凸显青少年淋巴瘤诊断的复杂性,并显示CD19 CAR-T 可能克服青少年BL中TP53相关的化疗耐药。病例还提示,将分子谱分析与免疫治疗结合,可能为青少年及青年高危难治病例提供新的管理策略。
Burkitt lymphoma (BL) with TP53 mutations is characterized by strong chemoresistance and poor prognosis, posing significant challenges in clinical treatment. Chimeric antigen receptor T-cell (CAR-T) therapy has shown promise in refractory/relapsed (r/r) BL, but its efficacy in TP53-mutated cases remains to be further validated. This case report describes a 16-year-old male adolescent diagnosed with TP53-mutated BL, whose initial presentation with recurrent abdominal pain led to a misdiagnosis of high-grade B-cell lymphoma. The disease was refractory to two cycles of chemotherapy (DA-EPOCH-R and AZA + R-CDOP) and even progression after third-line R-CODOX-M therapy. Following reevaluation confirming BL, the patient received CD19-directed CAR-T cell therapy (Axicabtagene Ciloleucel infusion, Axi-cel) and achieved complete metabolic response (CMR).
Baseline lactate dehydrogenase (LDH) was markedly elevated at 1,343 U/L. As of the latest follow-up in March 2025, the patient remains in remission at 30 months after CAR-T infusion with full functional recovery, including resumption of normal academic life.
This case, among the youngest reported uses of commercial Axi-cel for BL, highlights the diagnostic complexity in adolescent lymphoma and demonstrates that CD19 CAR-T therapy can overcome TP53-associated chemoresistance in adolescent BL. It also suggests that integrating molecular profiling and immunotherapy may provide new strategies for managing high-risk, treatment-refractory cases in the adolescent and young adult population.
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