Langerhans cell histiocytosis
Conditions
Interventions
Dabrafenib group:Dabrafenib
Chemotherapy group:Cytarabine, clatrabine, vindesine, dexamethasone, et al.
Sponsors
Beijing Children's Hospital, Capital Medical University, National Children's Medical Center
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: All the following criteria must be met: 1. Children with relapsed/refractory LCH admitted to our hospital; Relapsed/refractory LCH was defined as meeting one of the following conditions: (1)Patients with high-risk (RO+) were evaluated as no organ improvement or AD-progression or AD-intermediate after the first-line therapy; (2)Patients with bone marrow involvement and/or secondary hemophagocytic syndrome (HLH); (3)Patients with central nervous system (including pituitary) involvement; (4)Patients who relapsed after the first-line therapy. (5)Patients with plasma cfBRAF-V600E mutation-positive at week 11/25/52 of the first -line therapy; 2. Patients were diagnosed as LCH by clinical and pathological examination; 3. The BRAF-V600E mutation was positive in biopsy tissue or plasma before treatment with Dabrafenib; 4. Patients were less than 18 years of age; 5. Informed consent were obtained from guardians of the patients and the patients (more than 8 years old).
Exclusion criteria
Exclusion criteria: If one of the following conditions exists, he/she will not be included in this study: 1. Patient with BRAF-V600E mutation-negative in biopsy tissue; 2. Patient who had received other BRAF inhibitors, hematopoietic stem cell transplantation or an investigational agent; 3. Patient who had a history of myocardial infarction, unstable angina, peripheral vascular disease, familial QTc prolongation, abnormal cardiac valve morphology, or other cardiac issues; and had a history of leukemia or another malignancy; 4. Patient who was unable to comply during the trial and / or follow-up phase.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Progression-free Survival, PFS;overall response (OR) rate;Conversion rate of plasma cfBRAF-V600E;Cumulative incidence of relapse; | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall survival;Cumulative incidence of permanent consequences;Dynamic changes of key biomarkers during treatment;Plasma concentrations of Dabrafenib; | — |
Countries
China
Contacts
Public ContactRui Zhang
Beijing Children's Hospital, Capital Medical University, National Children's Medical Center
Outcome results
None listed