Current State of Targeted Therapy in Adult Langerhans Cell Histiocytosis and Erdheim-Chester Disease.

IF 4.4 3区 医学 Q2 ONCOLOGY
Targeted Oncology Pub Date : 2024-09-01 Epub Date: 2024-07-11 DOI:10.1007/s11523-024-01080-x
He Lin, Xin-Xin Cao
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引用次数: 0

Abstract

The mitogen-activated protein kinase (MAPK) pathway is a key driver in many histiocytic disorders, including Langerhans cell histiocytosis (LCH) and Erdheim-Chester disease (ECD). This has led to successful and promising treatment with targeted therapies, including BRAF inhibitors and MEK inhibitors. Additional novel inhibitors have also demonstrated encouraging results. Nevertheless, there are several problems concerning targeted therapy that need to be addressed. These include, among others, incomplete responsiveness and the emergence of resistance to BRAF inhibition as observed in other BRAF-mutant malignancies. Drug resistance and relapse after treatment interruption remain problems with current targeted therapies. Targeted therapy does not seem to eradicate the mutated clone, leading to inevitable relapes, which is a huge challenge for the future. More fundamental research and clinical trials are needed to address these issues and to develop improved targeted therapies that can overcome resistance and achieve long-lasting remissions.

Abstract Image

成人朗格汉斯细胞组织细胞增生症和埃尔德海姆-切斯特病的靶向治疗现状。
有丝分裂原激活蛋白激酶(MAPK)通路是许多组织细胞疾病(包括朗格汉斯细胞组织细胞增生症(LCH)和埃尔德海姆-切斯特病(ECD))的关键驱动因素。因此,包括 BRAF 抑制剂和 MEK 抑制剂在内的靶向疗法取得了成功,前景广阔。其他新型抑制剂也取得了令人鼓舞的效果。尽管如此,靶向治疗仍有一些问题需要解决。这些问题包括,除其他外,在其他BRAF突变恶性肿瘤中观察到的对BRAF抑制剂的不完全反应性和耐药性的出现。耐药性和治疗中断后的复发仍然是目前靶向疗法面临的问题。靶向治疗似乎无法根除突变克隆,导致不可避免的复发,这是未来面临的巨大挑战。要解决这些问题,开发出能够克服耐药性并实现长期缓解的改良型靶向疗法,还需要更多的基础研究和临床试验。
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来源期刊
Targeted Oncology
Targeted Oncology 医学-肿瘤学
CiteScore
8.40
自引率
3.70%
发文量
64
审稿时长
>12 weeks
期刊介绍: Targeted Oncology addresses physicians and scientists committed to oncology and cancer research by providing a programme of articles on molecularly targeted pharmacotherapy in oncology. The journal includes: Original Research Articles on all aspects of molecularly targeted agents for the treatment of cancer, including immune checkpoint inhibitors and related approaches. Comprehensive narrative Review Articles and shorter Leading Articles discussing relevant clinically established as well as emerging agents and pathways. Current Opinion articles that place interesting areas in perspective. Therapy in Practice articles that provide a guide to the optimum management of a condition and highlight practical, clinically relevant considerations and recommendations. Systematic Reviews that use explicit, systematic methods as outlined by the PRISMA statement. Adis Drug Reviews of the properties and place in therapy of both newer and established targeted drugs in oncology.
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