儿童朗格汉斯细胞组织细胞增多症的临床特征及预后因素：一项单中心回顾性研究
Clinical features and prognostic factors of pediatric Langerhans cell histiocytosis: a single-center retrospective study.
作者信息
Lu Yunfeng, Liu Liying, Wang Qi, Liu Bingju, Zhao Ping, Guan Guotao, Dai Yunpeng
机构信息
Department of Pediatric Hematology and Oncology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, China.
出版信息
Front Med (Lausanne). 2025 Jan 15;11:1452003. doi: 10.3389/fmed.2024.1452003. eCollection 2024.
PURPOSE
To retrospectively evaluate the clinical features and prognostic factors of pediatric LCH patients treated in a single center of China.
METHODS
Pediatric LCH cases were treated following the SD-LCH protocol at the Affiliated Provincial Hospital of Shandong First Medical University in Jinan, China. An analysis was conducted on 82 recently identified LCH cases to retrospectively evaluate the initial symptoms, therapeutic alternatives, and extended results. Follow-ups were conducted until July 31, 2023.
RESULTS
The median age at diagnosis was 2 (0.25-12) years. 42 (51.2%) were SS-LCH, and 40 (48.8%) were MS-LCH. The most common organ involved was bone (82.9%). Over the 16-year follow-up period, the 5-year EFS and OS rates were 75.2 ± 5% and 90.9 ± 3.3%, respectively. The cumulative reactivation rate was 23.2%. The 5-year EFS rate in SS-LCH and MS-LCH patients were 90.2 ± 4.6% and 58.8 ± 8.3%, and the 5-year OS rate in SS-LCH and MS-LCH patients were 90.2 ± 4.6% and 81.2 ± 6.5%, respectively. The 5-year OS and EFS rate in RO+ LCH and RO- LCH patients were 79.5 ± 7.5%, 53.8 ± 9.6% and 87.5 ± 11.7%, 76.2 ± 14.8%, insignificantly. Multivariate Cox regression showed that liver involvement predicted poor EFS and hematological system involvement was an independent prognostic factor for OS. Detection of the mutation and targeted therapy significantly improved the prognosis post-2017.
CONCLUSION
Liver or hematological system involvement indicates a poor prognosis, and the SD-LCH protocol improves prognosis for pediatric LCH patients.
目的
回顾性评估在中国单一中心接受治疗的儿童朗格汉斯细胞组织细胞增多症（LCH）患者的临床特征和预后因素。
方法
中国济南山东第一医科大学附属省立医院按照SD-LCH方案治疗儿童LCH病例。对最近确诊的82例LCH病例进行分析，以回顾性评估初始症状、治疗选择和远期结果。随访至2023年7月31日。
结果
诊断时的中位年龄为2（0.25 - 12）岁。42例（51.2%）为单系统LCH（SS-LCH），40例（48.8%）为多系统LCH（MS-LCH）。最常受累的器官是骨骼（82.9%）。在16年的随访期内，5年无事件生存率（EFS）和总生存率（OS）分别为75.2±5%和90.9±3.3%。累积复发率为23.2%。SS-LCH和MS-LCH患者的5年EFS率分别为90.2±4.6%和58.8±8.3%，SS-LCH和MS-LCH患者的5年OS率分别为90.2±4.6%和81.2±6.5%。RO + LCH和RO - LCH患者的5年OS和EFS率分别为79.5±7.5%、53.8±9.6%和87.5±11.7%、76.2±14.8%，差异无统计学意义。多因素Cox回归显示肝脏受累预示EFS不良，血液系统受累是OS的独立预后因素。2017年后检测到该突变并进行靶向治疗显著改善了预后。
结论
肝脏或血液系统受累提示预后不良，SD-LCH方案可改善儿童LCH患者的预后。
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