Langerhans cell histiocytosis
Conditions
Interventions
Experimental group:Patients with BRAF V600E mutation receive dabrafenib orally, and the patients with other mutations receive trametinib orally. The treatment duration was 6 months for patients with n
Control group:All patients received vindesine and steroid combination chemotherapy. The treatment duration was 6 months for patients with non CNS-risk multifocal bone involvement and 12 months for oth
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: 1 Patients aged < 18 years of both genders; 2 Patients with confirmed diagnosis of LCH by lesion biopsy and without prior LCH-directed therapy (including MAPK inhibitors, vinca alkaloids, cytarabine, cladribine, clofarabine, and etoposide); 3 Patients with multisystem involvement, multifocal bone involvement, central nervous system(CNS)-risk unifocal bone involvement (CNS-risk bone lesions including in the mastoid, sphenoid, orbit, clivus, or temporal bone lesions) or unifocal vertebral bone involvement with intraspinal occupying lesion compressing the spinal cord, and all of which were assessed by standardized evaluation protocol of the Histiocyte Society. 4 Patients with confirmed mutations in MAPK pathway associated genes in lesions or (and) plasma. ? .
Exclusion criteria
Exclusion criteria: 1 Patients with evidence of malignancy; 2 Patients who complicated with severe organ dysfunction, including: a. Severe liver dysfunction, defined as one of the following: serum total bilirubin >= 1.5×upper limit of normal range based on age, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase (AKP) >= 2.5×upper limit of normal range; b. Severe renal dysfunction: creatinine clearance rate 0.47 seconds in 12- lead body surface electrocardiogram; congestive heart failure with New York Heart Association classification >= grade 2; clinically significant arrhythmias, including but not limited to complete left bundle branch block, second-degree atrioventricular block; clinically significant coronary heart disease, cardiomyopathy, severe valvular disease; left ventricular ejection fraction (LVEF) < 50% shown by echocardiography; 3 Presence of central nervous system involvement, defined as one of the following: a. Pituitary involvement: pituitary magnetic resonance imaging (MRI) presents with thickening of the pituitary stalk and/or disappearance of the high signal in the posterior pituitary lobe, and/or clinical onset of diabetes insipidus; b. Space-occupying lesions in CNS: MRI indicates nodules or space-occupying lesions with low (isointense) T1 signal and high T2 signal in the meninges, choroid plexus or brain parenchyma; c. Neurodegeneration (Prosch diagnostic criteria): abnormal signals in the cerebellum, basal ganglia or pons on MRI T2 - weighted images, and/or progressive cerebellar atrophy; 4 Patients with active massive bleeding (including massive gastrointestinal bleeding, alveolar bleeding, intracranial bleeding, etc.); 5 Patients with severe allergic reactions to the drugs in the therapy; 6 Patients who are unable to comply during the trial and/or follow-up; 7 Patients participating in other clinical trials simultaneously; 8 Patients which the investigators consider are not suitable to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 24-months event-free survival rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall response;Relapse rate;24-months overall survival rate;adverse events; | — |
Countries
China
Contacts
Public ContactRui Zhang
Beijing Children's Hospital, Capital Medical University
Outcome results
None listed