Vemurafenib provides a rapid and robust clinical response in pediatric Langerhans cell histiocytosis with the BRAF V600E mutation but does not eliminate low-level minimal residual disease per ddPCR using cell-free circulating DNA.
Dmitry A EvseevIrina KalininaElena RaykinaDaria OsipovaZalina AbashidzeAnna IgnatovaAnna MitrofanovaAlexey MaschanGalina NovichkovaMichael MaschanPublished in: International journal of hematology (2021)
Vemurafenib is effective in children with BRAF V600E + LCH. However, treatment with vemurafenib does not eradicate the disease and its long-term toxicity has not been established.