Mekinist for Langherhan’s Hystiocytosis – pro
BRAF and MEK inhibition are becoming widely recognized as a very effective approach to the treatment of Langherhan’s Histiocytosis. There are multiple reports of the successful treatment of BRAFV600-mutant LCH with BRAF inhibitors (vemurafenib and dabrafenib) as monotherapyas well as blockade of the MEK pathway and reports that dual blockade of the MAPK pathway using a BRAF plus MEK inhibitor results in higher efficacy and less toxicity compared with monotherapy with a BRAF inhibitor. The FDA is reviewing an accelerated approval of this therapy.
Matina Papapanagiotou, Klaus G. Griewank, Uwe Hillen, Tobias T. Schimming, Lars C. Moeller, Dagmar Führer, Lisa Zimmer, Alexander Roesch, Antje Sucker, Dirk Schadendorf, Elisabeth Livingstone, and Bastian SchillingTrametinib-Induced Remission of an MEK1-Mutated Langerhans Cell Histiocytosis.JCO Precision Oncology 2017 :1, 1-5
Brown NA, Furtado LV, Betz BL, et al: High prevalence of somatic MAP2K1 mutations in BRAF V600E-negative Langerhans cell histiocytosis. Blood 124:1655-1658, 2014
Awada G, Seremet T, Fostier K, Everaert H, Neyns B. Long-term disease control of Langerhans cell histiocytosis using combined BRAF and MEK inhibition. Blood Adv. 2018;2(16):21562158. doi:10.1182/bloodadvances.2018021782
Awada G, Seremet T, Fostier K, Everaert H, Neyns B. Long-term disease control of Langerhans cell histiocytosis using combined BRAF and MEK inhibition. Blood Adv. 2018;2(16):2156–2158. doi:10.1182/bloodadvances.2018021782