成人朗格汉斯细胞组织细胞增生症的治疗进展

Progress in the Treatment of Adult Langerhans Cell Histiocytosis ——Review

朗格汉斯细胞组织细胞增生症(LCH)是一种罕见的以朗格汉斯细胞增生为主的增生性疾病,属于炎性髓样肿瘤。其临床表现不一,好发于任何年龄、任何部位,成人较儿童少见。诊断的金标准是组织病理活检。由于成人LCH的罕见性及疾病的异质性,成人LCH的治疗应根据疾病程度和危险分层制定治疗方案。随着MAPK、PI3K、c-KIT信号通路激活的发现,尤其是BRAF V600E、MAP2K1突变,靶向治疗已成为治疗的研究热点。同时,M2极化的巨噬细胞和Foxp3+调节性T细胞(Treg)在LCH中高表达的发现,为免疫治疗的探索提供了重要依据。本文将重点综述近年成人LCH治疗的最新研究进展,为临床上成人LCH患者的治疗研究提供参考。

Langerhans cell histiocytosis(LCH) is a rare proliferative disease dominated by the proliferation of Langerhans cells, which is inflammatory myeloid neoplasms. Its clinical manifestations are variable, occurring at any age and at any site, and it is rarer in adults than in children. The gold standard for diagnosis is histopathological biopsy. Due to the rarity of adult LCH and the heterogeneity of this disease, treatment of adult LCH should be developed according to the extent of the disease and risk stratification. With the discovery of MAPK, PI3K and c-KIT signaling pathway activation, especially BRAF V600 E and MAP2K1 mutations, targeted therapy has become a hot spot for therapeutic research. Meanwhile, the discovery of high expression of M2-polarized macrophages and Foxp3;regulatory T cells(Treg) in LCH has provided an important basis for the immunotherapy. In this article, we will focus on reviewing the latest research progress in the treatment of adult LCH in recent years, and provide a reference for clinical research on the treatment of adult LCH patients.