Langerhans cell histiocytosis
Conditions
Interventions
experimental group:vemurafenib sequential cytarabine
Sponsors
West China Second University Hospital, Sichuan University
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1) LCH was confirmed by histopathology (typical cell morphology and immunohistochemistry CD1a or / and langerin positive); 2) BRAF-V600E mutation was positive in pathological biopsy; 3) LCH patients with risk organ positive(RO +, including liver, spleen and hematopoietic system failed) failed to the first-line chemotherapy of LCH children. Failure of first-line therapy was characterized by disease progression in one or more ROs after six or more vinblastine (VBL) doses (one per week) and 28 days of prednisolone (minimum dose, 40 mg/m2/d), with or without the adjunction of a third drug. 4) LCH patients without risk organ involvement (RO -) failed to receive first-line chemotherapy (the definition is the same as above), and the disease still progressed after chemotherapy with high dose cytarabine; 5) No heart disease; 6) The age is from 1 month to 18 years old, and the gender is not limited; 7) Voluntarily sign informed consent form and agree to use all data for clinical research.
Exclusion criteria
Exclusion criteria: 1)Receive any form of radiotherapy within the past 3 months; 2) Patients with sclerosing cholangitis or central neurodegeneration at the time of diagnosis; 3) QTc > 0.5s or long QT interval syndrome; 4) Oral antiarrhythmic drugs; 5) Refractory electrolyte disorder; 6) Those who fail to understand and cooperate with the subject after being fully informed; 7) Those who intend to participate or are participating in other clinical studies during the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response rate;Progression free survival;Reactivation rate; | — |
Countries
China
Contacts
Public ContactJu Gao
West China Second University Hospital, Sichuan University
Outcome results
None listed