难治性朗格汉斯细胞组织细胞增生症治疗进展
Research advances in the treatment of refractory Langerhans cell histiocytosis
Received date: 2024-09-12
Accepted date: 2024-11-22
Online published: 2025-09-29
郎格汉斯细胞组织细胞增生症（LCH）是一种炎症性髓系肿瘤，目前治疗依危险度分层化疗，但存在疾病再激活率高、难治性病例多等难点。难治性LCH目前无标准治疗方案，以核苷类似物化疗为主，但高强度治疗伴随高并发症风险；靶向治疗起效快但易复发，联合治疗策略及优化疗程仍需进一步研究。未来，随着对LCH发病机制的深入研究以及更多新型治疗方法的探索，有望进一步提高难治性LCH的治疗效果，改善患者的预后。
关键词： 郎格汉斯细胞组织细胞增生症; 化疗; 难治性; 靶向治疗; 儿童
泥永安 , 孙立荣 . 难治性朗格汉斯细胞组织细胞增生症治疗进展[J]. 临床儿科杂志, 2025 , 43(10) : 798 -802 . DOI: 10.12372/jcp.2025.24e0974
Langerhans cell histiocytosis (LCH) is an inflammatory myeloid neoplasm. At present, the primary therapeutic strategy is risk-stratified chemotherapy. Nevertheless, significant clinical challenges persist, including high rates of disease reactivation and a considerable number of refractory cases. Currently, no standardized treatment protocol exists for refractory LCH. Nucleoside analog-based chemotherapy remains the mainstay of therapy, although it is associated with a substantial risk of complications. While targeted therapies demonstrate rapid efficacy, disease recurrence remains a common issue. Therefore, further investigation into combination treatment strategies and optimization of therapeutic regimens is warranted. With ongoing advances in understanding the pathogenesis of LCH and the development of novel therapeutic modalities, there is optimism that the management of refractory LCH will improve significantly, ultimately leading to better patient outcomes.
Key words： Langerhans cell histiocytosis; chemotherapy; refractory; targeted therapy; child
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