Data Sheet 1_Efficacy of chimeric antigen receptor T-cell therapy in testicular relapse of pediatric acute lymphoblastic leukemia: a multicenter retrospective study.pdf

IntroductionTesticular relapse constitutes one of the most frequent extramedullary recurrences in pediatric acute lymphoblastic leukemia (ALL), yet its clinical management remains incompletely characterized.
MethodsThis study assessed treatment outcomes and long-term survival in children with testicular relapse following initial therapy under the CCCG-ALL-2015 study (ChiCTR-IPR-14005706, http://www.chictr.org.cn). In total, 66 patients from 13 medical centers were retrospectively analyzed. Clinical characteristics and survival outcomes were compared across salvage treatment modalities.
ResultsThe median interval from initial diagnosis to testicular relapse was 37 months. Among 59 patients who received post-relapse therapy, the 2-year overall survival (OS) rate was 86.1% after a median follow-up of 33 months. Patients treated with chimeric antigen receptor T-cell (CAR-T) therapy showed a 2-year OS of 90.7%, compared to 81.7% in those managed with conventional regimens, such as chemotherapy, orchiectomy, or hematopoietic stem-cell transplantation (P > 0.05). Among 37 children with isolated testicular relapse, 18 underwent CAR-T therapy and 10 underwent orchiectomy, achieving 2-year OS rates of 92.3% and 100%, respectively (P > 0.05).
DiscussionTesticular relapse typically emerged approximately 3 years after initial diagnosis. CAR-T therapy proved to be both safe and effective, providing survival comparable to conventional regimens and offering potential advantages in preserving life quality among long-term survivors.

引言
睾丸复发是儿童急性淋巴细胞白血病（Acute Lymphoblastic Leukemia, ALL）最常见的髓外复发类型之一，但其临床管理策略仍未得到充分阐明。
方法
本研究针对CCCG-ALL-2015研究（ChiCTR-IPR-14005706，http://www.chictr.org.cn）中初始治疗后发生睾丸复发的儿童患者，评估其挽救治疗结局与长期生存情况。本研究回顾性分析了来自13家医疗中心的66例患者，并对比了不同挽救治疗方案下的临床特征与生存结局。
结果
从初始诊断至睾丸复发的中位间隔时间为37个月。在59例接受复发后治疗的患者中，中位随访33个月时的2年总生存（Overall Survival, OS）率为86.1%。接受嵌合抗原受体T细胞（Chimeric Antigen Receptor T-cell, CAR-T）治疗的患者2年总生存率为90.7%，而采用化疗、睾丸切除术或造血干细胞移植等常规治疗方案的患者2年总生存率为81.7%（P > 0.05）。在37例单纯睾丸复发的儿童患者中，18例接受了CAR-T治疗，10例接受了睾丸切除术，二者的2年总生存率分别为92.3%与100%（P > 0.05）。
讨论
睾丸复发通常在初始诊断后约3年出现。嵌合抗原受体T细胞治疗被证实兼具安全性与有效性，其生存获益与常规治疗方案相当，且可为长期生存患者保留更高生活质量提供潜在优势。