Langerhans cell histological hyperplasia (LCH) is a histocellular disease formerly known as "histocellular hyperplasia X". Its characteristics are clonal proliferation of histocytes and excessive accumulation of pathological Langerhans cells. The most common involved organs include: bone (80%), skin (33%), pituitary (25%), etc. When dangerous organs are involved, such as the blood system, liver, and spleen, the patient is at risk of dying, has a poor response to treatment, and has a poor prognosis.
Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histological hyperplasia, formerly known as "lipid granuloma". ECD lesions can involve in all systems throughout the body. The most common affected systems include: bones, cardiovascular, respiratory, central nervous system , etc. General symptoms include fever, fatigue, night sweats, weight loss, etc.
Langerhans cell histiocytic hyperplasia (LCH) and Erdheim-Chester disease are caused by mutations in the MAPK pathway ( is most commonly BRAF V600E) in myeloid dendritic cells, thus leading to both overlap and their own unique manifestations.
Due to the rareness of the above two diseases, there are certain challenges in their diagnosis and treatment. MEK inhibitor and BRAF inhibitor are important progress in recent years in application.
Free Clinical Project
BRAF Inhibitor HLX208
Trial Title: A single-arm, open, multicenter Phase II clinical study of rare diseases that evaluate the effectiveness, safety, and pharmacokinetics (PK) in adult Langerhans cell histiocytosis (LCH) and Erdheim-Chester disease (ECD) with BRAF V600E mutations.
Test type: Single-arm test
Indications: Langerhans cell histiocytic hyperplasia and Erdheim-Chester disease (first line and above)
Registration number: CTR2021245 9
Research Center: Beijing, Zhengzhou, Henan, Changsha, Hunan, Shanghai, Chengdu, Sichuan, Tianjin, Hangzhou, Zhejiang,
Main entry standards:
1, Histology confirmed adult LCH or (and) ECD patients.
2. The research center or central laboratory test clearly shows that BRAF V600E molecular mutation : Patients who used PCR or NGS methods to detect BRAF V600E mutation in the research center can be enrolled; if patients with LCH or (and) ECD who entered the screening period do not have a BRAF V600E report, the center laboratory test is required; if there is a BRAF V600E test report of the existing BRAF V600E test report of the research center, there is no need for unified retest in the central laboratory.
3, involves multi-system (greater than one system) or patients with involves single-system multi-lesion (greater than one lesion).
4. There are lesions that can be evaluated based on the PERCIST 1.0 standard. Adult LCH or (and) ECD patients with initial treatment or relapse or refractory can be included.
6. Age ≥18 years old, gender is not limited; expected survival time is at least 3 months; ECOG physical strength score 0-2 points.
7. You should receive any other anti-cancer treatments except this study within 2 weeks before the first use of the research drug (such as chemotherapy, other targeted treatments, other experimental drugs, radiotherapy, traditional Chinese medicine treatment, etc.) cannot participate in .
8, do not patients who have received BRAF V600E inhibitors (vimofenib, darafenib , etc.) or MEK inhibitors (smetinib, bimetinib, etc.) (except sorafenib ).
9. Previous or currently there are ventricular or atrial arrhythmia cannot participate in .
10. Refractory nausea and vomiting, malabsorption, bile drainage or hindering full absorption, intestinal segment resection cannot participate in .
[Important Tips] This official account [Global Good Medicine Information] All article information is for reference only. For specific treatment , please follow the doctor's advice !
